Oral Presentation Number: OP65 Abstract Title: Addressing System Need Through Piloting Early Value Assessment Of Digital Therapies For Mental Health Author Names: Charlotte Pelekanou ([email protected]), Lirije Hyseni, Kimberley Carter Introduction. Access to mental health services for adults, children and young people in England is limited due to increased demand and a lack of clinical capacity, leading to long waiting times for treatment. The medical technology programs at the National Institute for Health and Care Excellence (NICE) are piloting an innovative approach, known as early value assessment (EVA), to assess digital products, devices, and diagnostics, that most reflect national system need. These pilots include digital guided self-help therapies for treating adults with anxiety disorders or depression, and for treating children and young people with mild to moderate symptoms of anxiety or low mood. Methods. The aim of these EVA pilots are to assess the evidence on technologies with developing evidence bases within a short evaluation period. The process will explore whether technologies have the potential to be clinically and cost effective, and identify evidence gaps to help direct data collection. Results. During these pilots, evidence will be reviewed to evaluate whether these technologies demonstrate promising evidence of benefit with a low level of risk. This will provide challenges due to the complexity of care pathways in mental health, varying levels of evidence quantity and quality, and applicability of evidence to an National Health Service (NHS) setting. Early economic modelling will also be used to review the potential cost effectiveness of these technologies. Following the assessment, technologies may be conditionally recommended for use within the NHS provided further information is collected on key clinical outcomes. A faster full NICE assessment would be done following evidence collection before considering these for routine use in the NHS. Conclusions. A conditional recommendation for digital guided self-help therapies could increase the access to treatment in a pressurized mental health system. Trialing new methods to rapidly assess technologies in areas of unmet need will allow the service and patients to benefit from promising technologies sooner. These new methods and process will then be revised to reflect learning gained from the pilots.
Oral Presentation Number: OP66 Abstract Title: Adoption Of The World Health Organization Algorithm For Essential Medicines In The Philippine National Formulary Listing Process Author Names: Sheena Jasley Samonte ([email protected]), Princess Allyza Mondala, Lara Alyssa Liban, Patrick Wincy Reyes, Anne Julienne Marfori, Anna Melissa Guerrero, Bu Castro, Isidro Sia, Maria Minerva Calimag, Cecilia Maramba-Lazarte, Imelda Peña Introduction. The Philippine National Formulary (PNF) System preceded the health technology assessment (HTA) process in the Philippines, which was institutionalized in 2019. The transition led to previously prioritized topics of expert bodies overseeing the PNF System being endorsed to the HTA Council. However, the advent of COVID-19 forced the HTA Philippines to focus on emergency assessment needs and financing recommendations for the national government, resulting in limited capacity to assess non-public health emergency topics. To address this and improve patient access to medicines, we adopted the World Health Organization (WHO) process for evaluating and selecting medicines in the National Essential Medicines List (NEML). Methods. In assessing the pre-pandemic topics, we matched the population, intervention, comparator, and outcomes of the WHO clinical evidence reviews with those scoped with relevant stakeholders and performed local costing analyses to ensure applicability of findings to the Philippine setting. When needed, we subjected the topics to price negotiation or conducted qualitative assessments. Results. We found the method efficient in expediting the decision-making process of the HTA Council. However, given the limited internal capacity of the HTA Philippines to conduct assessments for all ongoing HTA tracks, some of the topics responsive to Universal Health Care will be outsourced to the HTA Research Network, which is yet to be established. There is also a need to improve alignment among the topics being assessed, since the priorities of the proponents, national health program, and national payer have already evolved. Conclusions. It is important to identify the priority areas for stakeholders as part of the topic nomination process, account for analytic capacity when setting the number of topics for HTA, establish mechanisms to allow proponents to conduct HTAs based on the HTA Council’s methodological standards, and proactively work with the national regulatory agency on horizon scanning and early HTA. We also recommend efficient monitoring, evaluation, and updating of the Philippine HTA guidelines so that they are more responsive to the needs of the healthcare system and the Filipino people.
Oral Presentation Number: OP67 Abstract Title: Horizon Scanning For Repurposed Medicines Author Names: Sola Akinbolade ([email protected]), Ross Fairbairn, Aoife Oliver, Amy Hussain, Sean Gill, Alex Inskip, Hannah O'Keefe, Dawn Craig, Dapo Ogunbayo Introduction. Drug repurposing, a strategy to identify new uses for approved medicines outside of their existing license, is not a new concept. However, there are perceived challenges and complexities in the regulatory and health technology assessment processes that bring these treatments to patients. The Medicines Repurposing Programme (MRP) in England identifies and progresses repurposing opportunities to improve clinical outcomes within the National Health Service (NHS). We describe a pilot project, undertaken by the National Institute for Health and Care Research (NIHR) Innovation Observatory (IO), to identify suitable candidates that meet the predefined MRP eligibility criteria and satisfy its objectives. Methods. The IO utilizes robust horizon scanning methodology to identify and collate data from clinical trial registries and company or media sources, which are then systematically stored on the IO’s internal Medicines Innovation Database (MInD). A search of trial registries and the MInD was implemented using the MRP screening criteria to identify candidates in phase I/II to IV development from non-commercial organizations, with primary completion dates between April 2021 and March 2022. A descriptive analysis of some of these data is provided here. Results. The IO’s horizon scanning process can identify and deliver intelligence on repurposed medicines. The pilot scan identified a total of 433 trials: 220 (51%) targeted oncology and 173 (40%) targeted rare diseases. Hematological cancers, lymphomas, cardiovascular system diseases, and breast cancer were the most common therapeutic areas. Of the trials, 253 (58%) were in phase II development and 127 (29%) were in phase III. Monoclonal antibodies and kinase inhibitors were the most frequently repurposed drug classes. Conclusions. This pilot scan identified potential candidate medicines that will be assessed for suitability for the MRP. If admitted to the program, they will receive tailored support toward adoption into the NHS. There is potential to expand this work to provide a more comprehensive source of intelligence on repurposed medicines, as well as to give valuable insights into innovation trends and areas of unmet medical need.
Oral Presentation Number: OP68 Abstract Title: Exploring The Diversity Of Interventions In Healthcare And Social Care: A Road To Priority Setting And Sustainability Author Names: Mireille Goetghebeur ([email protected]), Corelia Kostovic, Yannick Auclair, Brigitte Côté, Olivier Demers-Payette, Isabelle Ganache, Michèle de Guise Introduction. Horizon scanning in health technology assessment (HTA) agencies has traditionally been conducted to focus on the ongoing technological development of drugs and medical devices. While this is already a complex exercise for these types of interventions, it is even more so when it comes to understanding developments in the field of social care that could include relevant topics of assessment. Methods. The Institut National d’Excellence en Santé et Services Sociaux (INESSS) in Quebec, Canada has developed a methodology to collect and analyze interventions addressed in the international healthcare and social care technology assessment landscape, based on a conceptual approach that captures the diversity of interventions in health care and social services. Results. Insights generated by this comprehensive horizon scanning informed the Institute on existing solutions that can present the most value to the Quebec context. Conclusions. This approach feeds INESSS’ decision-making activities on topic prioritization. By the same token, it generates a reflection on the importance of diversifying interventions to develop a healthcare and social care system more adapted to the needs of the population it serves. It is envisioned that this will facilitate the most urgent reflection on carbon neutral healthcare and social care systems.
Oral Presentation Number: OP70 Abstract Title: Treating Patients With Hormone-Sensitive Cancer On Endocrine Therapy With Denosumab (Prolia®): A Systematic Review And Network Meta-Analysis Author Names: Konstance Nicolopoulos, Magdalena Ruth Moshi ([email protected]), Danielle Stringer, Ning Ma, Mathias Jenal, Thomas Vreugdenburg Introduction. Patients receiving endocrine therapy for hormone-sensitive cancers, such as men with prostate cancer (MPC) on hormone ablation therapy (HAT) and women with breast cancer (WBC) on adjuvant aromatase inhibitor therapy (AAIT), have an increased risk of developing osteoporosis. The aim of this study was to compare the safety and effectiveness of denosumab (Prolia®) with selective estrogen receptor modulators (SERMs) (raloxifene and bazedoxifene), bisphosphonates (zoledronate, ibandronate, alendronate, and risedronate), and placebo for the treatment of osteoporosis in patients receiving endocrine therapy for hormone-sensitive cancer. Methods. Systematic literature searches were conducted in three biomedical databases (PubMed, the Cochrane Library, and Embase) to identify randomized controlled trials (RCTs). Only RCTs that investigated MPC on HAT or WBC on AAIT allocated to denosumab, SERMs, bisphosphonates, or placebo were included. RCTs were appraised using the Cochrane Risk of Bias 2.0 tool. Frequentist network and pairwise meta-analyses were performed on predetermined outcomes of vertebral or nonvertebral fractures, treatment-related adverse events (AEs), bone mineral density (BMD), mortality, withdrawal due to treatment-related AEs, and serious AEs. Results. A total of 14 RCTs (15 publications, 6,463 participants) were included. Relative to placebo, denosumab was found to prevent vertebral fractures in cancer patients receiving endocrine therapy. Moreover, denosumab, alendronate, and zoledronate increased femoral neck (FN) and lumbar spine (LS) BMD in MPC on HAT, compared with placebo, whereas denosumab, risedronate, and ibandronate improved LS and total hip BMD in WBC on AAIT. Similarly, denosumab and risedronate increased trochanteric BMD in WBC on AAIT, compared with placebo. In WBC on AAIT, only denosumab increased FN BMD relative to placebo. Conclusions. Denosumab was more effective than placebo in preventing vertebral fractures and improving BMD at the LS and FN in MPC on HAT, and in preventing vertebral fractures and improving FN, trochanteric, total hip, and LS BMD in WBC on AAIT. From a policy perspective, the continued reimbursement of denosumab needs to be reviewed.
Oral Presentation Number: OP71 Abstract Title: Road To Public Funding Of Cancer Codependent Technologies In Australia In The Last Ten Years Author Names: Yuan Gao ([email protected]), Mah Laka, Tracy Merlin Introduction. In Australia, cancer codependent technologies (cCDTs) mostly comprise a biomarker targeting medicine and a companion diagnostic test (CDx). Health technology assessment (HTA) of cCDTs is carried out to inform funding deliberations on CDxs by the Medical Services Advisory Committee (MSAC) and on personalized medicine by the Pharmaceutical Benefits Advisory Committee (PBAC). To understand the strengths and weaknesses of this dual assessment mechanism, we studied the journey of cCDTs in getting funding support from the two committees since the introduction of the codependent technology evaluation framework. Methods. Public summary documents summarizing deliberations by each committee were reviewed from 2012 to 2022. Information was retrieved on the patient indication, date, biomarkers related to the tests, and PBAC or MSAC funding outcomes. The alignment of HTA decisions, time taken until dual funding approval (if approved), and the reasons for discrepant and negative decision-making were determined. Results. From 2012 to 2022, a total of 26 cCDT applications were submitted to PBAC and MSAC, corresponding with 43 paired PBAC/MSAC considerations and 11 single committee considerations. Non-small cell lung cancer and programmed cell death ligand 1 were the most frequently nominated cancer and biomarker test, respectively. When a cCDT was submitted in the same decision round to both committees, 60 percent of funding decisions were aligned, reaching 73 percent when the considerations were made separately (resubmissions). Only 9 percent of considerations received polarized, where one committee supported and the other committee rejected funding. After multiple resubmissions, 73 percent of cCDTs obtained dual funding support after an average of 34.8 weeks, with considerations by PBAC and MSAC occurring an average of 2.3 and 1.9 times, respectively. Conclusions. Most cCDTs obtain funding support, but only after multiple resubmissions to PBAC and MSAC. Polarized decisions are rare. Reasons for rejection primarily relate to uncertain clinical benefit and an unacceptably high incremental cost-effectiveness ratio.
Oral Presentation Number: OP72 Abstract Title: Innovating To Decrease Mortality And Resource Use In Surgical Inpatients: The ZERO Project Author Names: Carla Fernández-Barceló ([email protected]), Ismail Abbas, Guido Muñoz, Joan Sanchez, Ricard Mellado- Artigas, Carlos Ferrando, Laura Sampietro-Colom Introduction. Interest in early detection of complications in hospitals has increased recently. Complications after elective or urgent surgery are frequent and are associated with higher mortality rates, longer hospital stays, and more resource utilization. The ZERO project implemented an educational nursing program and developed an innovative algorithm that assesses a patient’s complication risk based on clinical parameters to prevent complications and reduce hospital burden. Our aim was to present the results from one year of implementing ZERO at the Clinic Barcelona University Hospital. Methods. A comparative effectiveness and cost study was conducted. Data from patients admitted after elective or urgent surgery were collected for one year retrospectively (n=8,844 from January 2019 to December 2019) and prospectively (ZERO) (n=8,163 from October 2021 to October 2022). Effectiveness was measured in terms of mortality, complications, and life-years gained (LYG). Length of stay (LoS) at conventional, intermediate, and intensive care units and rates of readmissions were collected for resource use. The chi-square test was used to compare categorical variables. The t-test and Wilcoxon test were used for normally and non-normally distributed continuous variables, respectively. Results. There was a significant decrease in the rate of complications (7.8%, 95% confidence interval [CI]: -8.46, -7.19; p<0.001) with ZERO. Moreover, there were statistically significant reductions in mean LoS for readmissions to conventional wards (-5.04 days, 95%CI: -9.9, -0.18; p=0.04) and to the intensive care ward within the same episode (-4.68 days, 95%CI: -9.26, -0.14; p=0.02). The mean cost per patient was EUR2,772.92 and EUR2,591.57 before and after ZERO implementation, respectively. After accounting for the cost of implementing ZERO, there was a cost saving of EUR147.76 per patient (p=0.048), which yielded a yearly impact of EUR1,206,165 for the hospital budget. Conclusions. This one-year analysis of the effect of ZERO on surgical patients shows that it decreases complication rates and all types of LoS, leading to overall cost savings for the hospital.
Oral Presentation Number: OP74 Abstract Title: Analysis Of Literature And Research Foci In Overdiagnosis Based On Citespace Author Names: Juntao Yan ([email protected]), Yan Wei, Yi Yang, Shimeng Liu, Yingyao Chen Introduction. With the rapid development of innovative health technologies, evidence increasingly shows that overdiagnosis is harmful to a person’s health and that it is a global public health issue. This study aimed to analyze the current research status and corresponding foci in the field of overdiagnosis in Chinese and English databases using bibliometric methods. Methods. A search was conducted in the English Web of Science Core Collection database and the Chinese China National Knowledge Infrastructure database for literature published from inception to 31 December 2021. CiteSpace (version 5.8 R1) software was used to perform bibliometric analysis on the countries, institutions, and keyword clusters of the included literature on overdiagnosis and to draw a corresponding visual knowledge map. Results. A total of 2,841 English and 43 Chinese publications were included. There was an increasing trend in the annual publication volume of both Chinese and English literature, with the publication volume of English research increasing significantly since 2010. In terms of countries and institutions, the top ten in English research on overdiagnosis were all from high income countries. The cooperation among these countries and institutions was close, unlike in China where the cooperation was relatively limited. Analysis of keyword clustering showed that the potential research foci for English literature on overdiagnosis included breast cancer, thyroid cancer, prostate cancer, lung cancer, and other tumor types, whereas the clustering in Chinese records was relatively scattered and mainly focused on overdiagnosis of thyroid cancer. Conclusions. The research topics in the Chinese literature on overdiagnosis lag significantly behind English research. It is suggested that more research on overdiagnosis and related fields should be actively promoted and conducted in China in the future.
Oral Presentation Number: OP75 Abstract Title: Cost-Effectiveness Of Brief Advice To Quit Smoking To Prevent Postsurgical Complications In People Living With Cancer Author Names: Nikki Mccaffrey ([email protected]), Emma Dean, Paul S. Myles, John Cunningham, Elizabeth Greenhalgh, Sally Doncovio, Nicholas Graves, Lisa Briggs, Anita Lal Introduction. People living with cancer who smoke have an increased risk of postsurgical complications. Quitting smoking significantly improves surgical outcomes, shortens hospital stays, and reduces the risk of cancer recurrence and death. Contemporary population estimates of the shorter term benefits of routinely providing smoking cessation in health services for people undergoing cancer surgery are unknown. Consequently, this analysis evaluated, for the first time, the cost effectiveness of providing brief advice to quit smoking to Australians living with cancer to reduce postsurgical complications. Methods. A decision tree model was developed to estimate the incremental cost per postsurgical complication avoided of offering brief advice to quit smoking, compared with standard care, from a hospital provider perspective. Brief advice was defined as verbal instructions from a clinician using the ask, advise, and act model. The model included the cost (2019 EUR) and effectiveness of providing brief advice (literature review and expert opinion), the cost of gastrectomy (literature review), and the incidence of postsurgical complications over 90 days in people living with cancer (systematic review). The impact of uncertainty around the model input values was estimated using probabilistic sensitivity analysis (Monte Carlo simulation). Results. Providing brief cessation advice dominated standard care (i.e., costs less and reduced complications). Brief advice dominated standard care in 99.9 percent of the 1,000 Monte Carlo iterations, suggesting a high degree of certainty in the results. Among patients undergoing cancer surgery who smoke, the expected proportion experiencing postsurgical complications was 30.9 percent (95% uncertainty interval [UI]: 29.5, 32.2) for those receiving brief advice versus 31.3 percent (95% UI: 29.9, 32.6) for standard care. Overall expected costs were slightly lower for brief advice (EUR19,816, 95% UI: 16,715, 22,392), compared with standard care (EUR20,125: 95% UI: 16,725, 22,428). Conclusions. The findings suggest that implementing brief advice to quit smoking in patients undergoing cancer surgery reduces postsurgical complications and saves healthcare costs. Communicating shorter term benefits helps promote adoption of evidence-based practice in health services by health administrators, funders, and policy makers.
Oral Presentation Number: OP77 Abstract Title: Distributional Cost-Effectiveness Analysis of Lung Cancer Screening for Indigenous and non- Indigenous Populations in Australia Author Names: Jackie Roseleur ([email protected]), Kevin ten Haaf, Harry de Koning, Tamara Mackean, Madison Shakespeare, Jonathan Karnon Introduction. Australia’s Medical Services Advisory Committee (MSAC) has recently recommended the implementation of a national lung cancer screening program. Despite Aboriginal and Torres Strait Islander (Indigenous) people experiencing a higher incidence of lung cancer at earlier ages, and poorer outcomes than the general population, no specific recommendations have been made for Indigenous peoples. To reduce health outcomes gaps, healthcare decision-making must consider equity. Distributional cost-effectiveness analysis (DCEA) is a framework for including concerns about health inequity in the economic evaluation of healthcare interventions. Our aim is to undertake a DCEA to inform decisions around the specification of differential eligibility criteria for lung cancer screening for Indigenous and non-Indigenous populations. Methods. MISCAN-Lung, a microsimulation model, was used to estimate the cost-effectiveness of alternative lung cancer screening strategies for Indigenous and non-Indigenous populations, incorporating smoking history and lung cancer incidence data specific to both populations. Equity weights will be applied to the results. These equity weights will be derived using two different methods: (i) epidemiological data on mortality and morbidity and (ii) a discrete choice experiment to elicit population preferences regarding the allocation of health gains between Indigenous and non-Indigenous populations. Consultations with Indigenous stakeholders will be undertaken to identify community-preferred screening strategies for which DCEA will be undertaken. Results. Incremental costs of AUD62,754 (USD43,601) and AUD101,130 (USD70,265) per QALY gained were estimated for Indigenous and non-Indigenous populations for the MSAC recommended screening scenario, respectively. For the Indigenous population, annual screening generated a lower incremental cost per QALY gained of AUD93,715 (AUD65,113). The community-preferred strategies identified in the consultations will be included in the model for additional analyses. The derived equity weights will be used as inputs to the DCEA to inform decisions around the value of separate criteria for Indigenous peoples. Conclusions. Application of equity weights could help decision-makers determine whether alternative eligibility criteria should be used for Indigenous peoples to reduce the inequity from lung cancer experienced by Indigenous populations.
Abstract Title: Oral Presentation Number: OP78 Abstract Title: Cost-effectiveness Of A 20-valent Pneumococcal Conjugate Vaccine To Directly Protect Adults Against Pneumococcal Disease In England Author Names: Diana Mendes ([email protected]), Ahuva Averin, Mark Atwood, Reiko Sato, Andrew Vyse, James Campling, Derek Weycker, Mary Slack, Gillian Ellsbury, Tendai Mugwagwa Introduction. Adult vaccination with 13-valent pneumococcal conjugate vaccine is currently recommended in the UK only for very high-risk individuals, with 23-valent pneumococcal polysaccharide vaccine (PPV23) being recommended to all adults 65 years or older and those 18 years or older with specified risk conditions. A 20- valent pneumococcal conjugate vaccine (PCV20) has recently become available for use in adults with potential to address a substantial proportion of the current adult pneumococcal disease burden in the UK. We evaluated the cost-effectiveness of PCV20 vaccination compared with PPV23 in adults in England currently eligible for pneumococcal vaccination. Methods. A probabilistic model with a Markov-type process was used to depict lifetime risks and costs of pneumococcal disease among a cohort of English adults. Epidemiologic parameters, serotype coverage, costs, vaccine effectiveness and coverage were based on published literature or publicly available data. The National Health Service perspective was adopted, health effects were expressed in quality-adjusted life years (QALYs), and future costs and QALYs were discounted at 3.5 percent. Results. Results suggest that under reasonable assumptions concerning disease burden, vaccine, effectiveness, and vaccine cost, PCV20 implementation of an age- and risk-based strategy targeting all adults aged 65 years or older and younger risk group adults aged 18 to 64 years would reduce a large number of pneumococcal disease hospitalizations and pneumococcal-related deaths compared to currently recommended PPV23. The incremental cost-effectiveness ratio was well below the current willingness-to-pay range of GBP20,000- GBP30,000 per QALY gained, with PCV20 being cost saving compared with PPV23 in base case and most sensitivity analyses. Probabilistic sensitivity analysis suggests high certainty in recommending PCV20 for vaccination of adults aged 18 to 64 years in risk groups and all aged 65 years or older instead of PPV23. Conclusions Our findings support replacing PPV23 with PCV20 to directly protect adults against pneumococcal disease, reducing hospitalizations and saving lives in the UK.
Abstract Title: Oral Presentation Number: OP79 Abstract Title: Gene Expression Profiling In The Diagnosis Of Aggressive Large B Cell Lymphoma: An Early Exploratory Economic Evaluation Author Names: Janet Bouttell ([email protected]), Neil Hawkins, Rachel Masson, John Goodlad Introduction. The addition of gene expression profiles (GEP) to the current clinicopathological diagnosis of aggressive large B cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempoSeq technology to distinguish Burkitt Lymphoma (BL) and Primary Mediastinal Large B Cell lymphoma (PMBCL) from Diffuse Large B Cell Lymphoma (DLBCL). This study aims to inform developers about the potential impact of the test on costs and health outcomes, and pricing and evidence generation strategies. Methods. Decision models compared current diagnosis with current plus GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from the literature and based on assumptions. Threshold estimates were made of the maximum price of the test and impact of incorrect disease classification using a threshold of GDP30,000 (USD37,155) per Quality Adjusted Life year (QALY). One way sensitivity analysis was conducted. Results. At base case values the BL signature delivers incremental QALYs of 0.0249 at an additional cost per patient of GBP508 (USD629). This results in a net monetary benefit (NMB) of GBP239 (USD296). The PMBCL signature delivers 0.0011 QALYs, a cost saving of GBP202 (USD250) and an NMB of GBP236 (USD292). The maximum threshold price for a combined test to be cost effective is GBP776 (USD961) (base case GBP400 (USD495)). Results are sensitive to cost differences in first line treatments and impact of false diagnoses. Conclusions. A combined test could be cost-effective in a UK context at a price around GBP750 (USD929). The developers can use this estimate to inform return on investment calculations. The number of patients who were reclassified as a result of the addition of GEP in our model was taken from small retrospective studies and the impact of false diagnoses was based on limited evidence. If the developers choose to proceed with the development, these aspects should be incorporated in evidence generation strategies.
Abstract Title: Oral Presentation Number: OP80 Abstract Title: Diagnostic Molecular Sequencing Of DNA (Exomes And Genomes) Is Not Perfect: Implications For HTA Author Names: Camille Schubert ([email protected]), Stephen Goodall, Tracy Merlin Introduction. The recent release of powerful next-generation sequencing platforms, which can provide whole exome sequencing (WES) or whole genome sequencing (WGS) in quicker timelines and at reduced costs, has resulted in proposals for these diagnostic testing methods to be routinely integrated into clinical practice in multiple settings. However, the complexities of these diagnostic approaches, and the minimal comparative evidence available on them, creates difficulties in the evaluation of their diagnostic performance. Novel approaches need to be developed to improve the health technology assessment (HTA) of WES and WGS. Methods. Several HTAs on genetic testing and the use of WES or WGS in fetal medicine were reviewed. Information on factors associated with this diagnostic modality that affect typical test accuracy assessment (e.g., sensitivity and specificity) was extracted. The multiple steps required for completing a WES or WGS test, and the potential for the introduction of errors (type I or type II) at each of these steps, were mapped and examples provided. The clinical and economic implications associated with imperfect and uncertain test accuracy were described. Results. Limited data on analytical and clinical validity were identified. WES and WGS are multistep processes and errors were found in sampling, molecular sequencing, bioinformatic filtering, and variant interpretation; therefore, the assumption that WES or WGS is 100 percent sensitive or specific is not reasonable. Although alternative evidence-based estimates are unlikely to be available, the inevitability of such errors, and their implications in terms of comparative effectiveness, safety, and cost effectiveness, should be described in HTAs. Conclusions. While unknown diagnostic accuracy remains an issue with WES and WGS testing, formal sensitivity analysis of test performance characteristics should be conducted as part of HTAs. A checklist has been developed to assist those involved in HTA and policy to understand the potential for inaccurate test results in clinical practice, and the risk-benefit implications of these diagnostic errors for patients.
Abstract Title: Oral Presentation Number: OP81 Abstract Title: Cost Effectiveness Of Human Papillomavirus Extended Genotyping For Cervical Cancer Screening In Singapore Author Names: Brandon Chua, Li Min Lim, Joseph Ng, Viva Ma, J. Jaime Caro, Hwee Lin Wee ([email protected]) Introduction. The World Health Organization recommends the human papillomavirus (HPV) test for cervical cancer screening. HPV partial genotyping (PGT) identifies HPV16 and HPV18 individually and the 12 other high-risk HPV genotypes (hrHPV) collectively. In contrast, HPV extended genotyping (XGT) identifies six hrHPV individually (HPV16,18,31,45,51, and 52) and the other eight in three groups (HPV33/58, HPV56/59/66, and HPV35/39/68). XGT allows better risk stratification for patient management and monitoring of persistent same-genotype infections (PSGI), which convey a higher risk for cervical cancer. This study compared the cost, quality-adjusted life-years (QALYs), and resource use of XGT with PGT when used as the primary cervical cancer screening method in Singapore. Methods. A discretely integrated condition event simulation was developed for screening 500,122 women aged 30 to 69 years over five years from the health system perspective, using a three percent annual discount. For XGT, women with HPV35/39/51/56/59/66/68 and reflex cytology of atypical squamous cells of undetermined significance were recalled for a repeat screening in one year, instead of the immediate colposcopy referral that occurs with PGT. At repeat screening, colposcopy was only provided for women with PSGI on XGT. Published data from Singapore were used for inputs and supplemented with data from international literature. Deterministic and probabilistic uncertainty analyses were conducted. Scenario analysis was conducted to simulate various HPV burdens among women. XGT was cost effective when the incremental cost-effectiveness ratio (ICER) relative to PGT was below SGD100,000 (USD118,906) (gross domestic product per capita in 2021). Results. XGT was cost effective relative to PGT (ICER SGD16,370 [USD19,465]), with fewer colposcopies (n=7,130; 19%), liquid-based cytology tests (n=6,027; 7%), and clinic consultations (n=9,787; 2%) but more HPV tests (n=2,446; 0.5%). The ICER was most sensitive to the relative cost of XGT and the cost of PGT. XGT remained cost effective in one-way sensitivity analysis in all 1,000 probabilistic uncertainty simulations and in scenario analysis with various HPV burdens. Conclusions. XGT can provide a cost effective, risk-based approach to primary cervical cancer screening, with lower resource utilization than PGT.
Oral Presentation Number: OP82 Abstract Title: Positron Emission Tomography Combined With Computed Tomography Using 18F-Sodium Fluoride Author Names: Tansolpan Aimanova, Makpal Akhmetova, Makhabbat Okesh, Andrey Avdeyev ([email protected]), Nasrulla Shanazarov, Rustam Albayev, Aigul Saduakassova, Yekaterina Lyugay, Indira Tleulessova, Olzas Turar, Gulzada Bariyeva, Introduction. Positron emission tomography combined with computed tomography (PET/CT) using 18F-sodium fluoride (18F-NaF) is used for functional imaging in diseases to detect abnormally altered osteogenic activity, such as benign and malignant bone diseases and inflammatory or traumatic changes in skeletal bones. Methods. A systematic search of literature using keywords in the MEDLINE database was conducted to identify literature on the clinical and cost effectiveness of using PET/CT with 18F-NaF-based radiopharmaceuticals in the diagnosis of bone and cartilage cancer. The search retrieved 323 publications. The analysis included 11 publications that met the selection criteria, including one meta-analysis and ten literature reviews. Results. The pooled sensitivity, specificity, diagnostic odds ratio (DOR) and area under the receiver operating characteristic curve of 18F-NaF-based PET/CT for the detection of bone metastases were 0.98 (95% confidence interval [CI]: 0.95, 0.99), 0.90 (95% CI: 0.86, 0.93), 123.2, and 0.97, respectively. 18F-NaF-based PET/CT was highly effective in detecting bone metastases during staging and restaging of patients with high-risk prostate cancer. The effectiveness of 18F-NaF-based PET/CT was superior to bone scintigraphy with technetium-99m and single-photon emission computed tomography (SPECT) and was comparable to diffusion-weighted imaging. Conclusions. PET/CT with 18F-NaF is a more accurate method of localizing and characterizing malignant bone lesions than SPECT. This method has improved clinical accuracy and provides greater convenience for patients and referring physicians. PET/CT with 18F-NaF in malignant neoplasms is a more specific, sensitive study than 18F- fluorodeoxyglucose PET/CT. These results were submitted to the Ministry of Health for a decision on the inclusion of 18F-NaF-based PET/CT in the state reimbursement system.
Oral Presentation Number: OP83 Abstract Title: Diagnostic Accuracy And Cost Effectiveness Of Automated Ankle Brachial Pressure Index Measurement For Peripheral Arterial Disease In People With Leg Ulceration Author Names: Dwayne Boyers ([email protected]), Moira Cruickshank, Lorna Aucott, Charlotte Kennedy, Paul Manson, Paul Bachoo, Miriam Brazzelli Introduction. Leg ulcers are usually treated with compression therapy unless there is evidence of peripheral arterial disease (PAD). Compression may lead to vascular complications in people with PAD. Timely diagnosis and appropriate treatment are important to ensure the best patient outcomes. PAD is usually assessed using a manual ankle brachial pressure index (ABPI). Automated devices may reduce time to diagnosis and treatment of leg ulcers. This study investigated the diagnostic accuracy, clinical effectiveness, and cost effectiveness of automated ABPI measurement, compared with manual doppler testing, to detect PAD in people with leg ulcers. Methods. We conducted a systematic review of studies of any design assessing automated devices in any population. Meta-analyses were conducted where possible. A decision tree and Markov model was used to capture lifetime costs, quality-adjusted life-years (QALYs), and cost effectiveness from a UK National Health Service perspective. Sensitivity analyses captured the uncertainty surrounding model assumptions and probabilistic sensitivity analyses described parameter uncertainty. Value of information analysis was conducted to identify future research priorities. Results. A total of 22 studies provided diagnostic accuracy data for five automated devices, but there were no studies in people with leg ulcers. Meta-analysis of 12 studies demonstrated a pooled sensitivity of 64 percent (95% confidence interval [CI]: 57, 71) and a specificity of 96 percent (95% CI: 92, 98) for detecting PAD. Automated devices were cheaper to complete due to shorter test times, but the increased risk of inappropriate or delayed treatment due to inaccurate test results offset the cost savings and reduced the QALYs for automated devices. Conclusions. Given the current limited evidence base, automated devices would only be cost effective if they can demonstrate substantial reductions in time to diagnosis in clinical practice. Value of information analysis identified the following research priorities: (i) to determine diagnostic accuracy in a population with leg ulcers; (ii) to determine the impact of test results on ulcer healing times, and (iii) to determine the risk of providing inappropriate treatment based on inaccurate test results.
Oral Presentation Number: OP84 Abstract Title: Cost Effectiveness Of Testosterone Treatment For Men With Hypogonadism Author Names: Rodolfo Hernández ([email protected]), Jemma Hudson, Moira Cruickshank, Charlotte Kennedy, Richard Quinton, Magaly Aceves-Martins, Katie Gillies, Paul Manson, Waljit S. Dhillo, Siladitya Bhattacharya, Miriam Brazzelli, Channa N Jayasena Introduction. Testosterone treatment (TT) is the standard therapy for male hypogonadism (MH). Testosterone prescribing rates have risen in the UK without an observable increase in the prevalence of hypogonadism. There is uncertainty about the safety and efficacy of TT for MH, especially regarding its impact on cardiovascular risk, which has led to mandatory safety labelling in some countries. We conducted a cost-utility analysis of TT versus no TT in men with hypogonadism using individual patient data (IPD) on efficacy and safety outcomes from over 3,000 participants as part of an international collaboration (TestES Consortium comprising 17 randomized controlled trials). Methods. We developed a Markov model with five states: no complications, death, and post-cardiac, post-peripheral vascular system, and post-cerebrovascular system pathologies. Care pathways were informed by existing clinical guidelines, the TestES Consortium IPD meta-analysis, and discussions with clinical experts. All-cause mortality, cardiovascular and cerebrovascular complication risks, and utility weights were obtained from the IPD analyses. Ten-year and lifetime time horizons were considered from the UK National Health Service perspective. The model was run probabilistically for three starting ages: 40, 60, and 75 years. Expected costs, quality-adjusted life-years, and incremental cost-effectiveness ratios were calculated. Results. TT cost effectiveness appeared dependent on the relative risk (RR) of all-cause mortality, favoring TT, and the methods used to derive health state utility scores. When the mortality RR and Beck Depression Inventory utilities were used, ICERs remained below the GBP20,000 (EUR24,000) conventional UK threshold but increased above this threshold when the all-cause mortality difference was dropped and SF-6D utility scores were used. Conclusions. We report the most in-depth economic analysis of MH treatment to date. Data for generating utility weights for economic evaluation remain limited. Further clarity on the long-term cardiovascular safety of TT for MH and mapping of clinical outcomes to generic preference-based measures of health-related quality of life will be crucial to inform robust estimates of the cost effectiveness of TT for MH.
Oral Presentation Number: OP85 Abstract Title: Cost Effectiveness Of Prednisolone To Treat Bell’s Palsy In Children: An Economic Evaluation Alongside A Randomized Controlled Trial Author Names: Xiuqin Xiong ([email protected]), Li Huang, David Herd, Meredith Borland, Andrew Davidson, Stephen Hearps, Mark Mackay, Katherine Lee, Stuart Dalziel, Kim Dalziel, John Cheek, Franz Babl Introduction. The cost effectiveness of treating Bell’s palsy with prednisolone in children is unknown. This study aimed to assess the cost effectiveness of prednisolone, compared with placebo, in treating Bell’s Palsy in children from a healthcare sector perspective. Methods. This economic evaluation was a prospectively planned secondary analysis of a triple-blind randomized superiority trial conducted from 2015 to 2020 that compared prednisolone with placebo. The time horizon was six months after randomization. The 180 participants were aged from six months to 17 years and presented within 72 hours of onset of clinician diagnosed Bell’s palsy. Interventions were oral prednisolone (1 mg per kg daily) or taste-matched placebo administered for ten days. Incremental cost-effectiveness ratios comparing prednisolone with placebo were estimated. Costs included medication costs, doctor visits, and medical tests over the six-month study period. Effectiveness was measured using quality-adjusted life-years (QALYs) derived from the Child Health Utility 9D instrument. Nonparametric bootstrapping was performed to capture uncertainties. Prespecified subgroup analyses by age (12 to 17 years versus <12 years) were performed. Results. The mean cost per patient was USD188 in the prednisolone group and USD121 in the placebo group over the six-month period (difference USD66, 95% confidence interval [CI]: 47, 179). The mean QALYs gained over six months were 0.45 in the prednisolone group and 0.44 in the placebo group (difference 0.01, 95%CI: -0.01, 0.03). Prednisolone was very likely cost effective given a conventional willingness-to-pay threshold of USD50,000 per QALY gained (the cost per additional QALY gained was USD6,625 using prednisolone compared with placebo). Subgroup analysis suggested that this was primarily driven by the high probability of prednisolone being cost effective in children aged 12 to 17 years (98%), compared with those younger than 12 years (51%). Conclusions. This study provides new evidence to stakeholders and policy makers who are considering whether to make prednisolone available for treating Bell’s palsy in children aged 12 to 17 years.
Oral Presentation Number: OP86 Abstract Title: Cost-effectiveness Of High-Sensitivity Troponin Testing In The Investigation And Management Of Chest Pain In The Emergency Department Author Names: Aubyn Pincombe ([email protected]), Jodi Gray, Frank Sanfilippo, Tom Briffa, Jonathan Karnon Introduction. Troponin tests (Tn) have been used to detect heart muscle damage since the late 1990s. High-sensitivity Troponin (hsTn) tests have recently become available. These enable a more timely and accurate diagnosis of MI. It is hypothesized that switching to hsTn testing will lead to better resource utilization, clinical management, shorter times to discharge and improve patient outcomes, therefore justifying the additional cost per test. However, a recent Australian randomized controlled trial reported hsTn testing did not reduce 12-month ischemic events, and the implementation method may have increased mortality in patients with newly identified troponin elevations. A retrospective multisite pre-post stratified study of hsTn implementation in Australia is being undertaken. The objective is to evaluate the real-world costs and benefits of hsTn testing to inform decisions about its cost-effective use in Australia. Methods. Routinely collected administrative emergency department (ED) data from 10 Australian major metropolitan hospitals in five states were linked to patient admissions, pathology, and mortality data for 167,100 patients, aged more than 20 years, who presented to the ED between 2011 and 2015. Patient level costs were estimated using the National Efficient Price with price weights for each admission based on Australian-Refined Diagnostic Group codes (AR-DRGs) adjusted for length of stay. Costs were estimated for the full range of resources during the index hospital admission and for subsequent admissions within 12 months regardless of final diagnosis. The outcome of interest was a composite endpoint of death or myocardial infarction (MI) at 30-days and 1-year. Non-parametric bootstrapping will be used to derive paired estimates of mean costs and events avoided to capture uncertainty in the estimates. Results. The initial outcome analyses have commenced, and we expect to be able to report on the final results of the cost-effectiveness study at the 2023 HTAi meeting. Conclusions. Real-world assessments of implemented health technologies have a potentially important role in demonstrating real-world value and assessing the effects of variation in implementation to inform improved use of new health technologies.
Oral Presentation Number: OP87 Abstract Title: A Roadmap for Increasing The Usefulness And Impact Of Patient Preference Studies In Health Technology Assessment (HTA) Author Names: Deborah A Marshall ([email protected]), Esther W de Bekker-Grob, Brett Hauber, Sebastian Heidenreich, Ellen Janssen, Alice Bast, Janel Hanmer, Laura Lee Johnson, Andriy Danyliv, Eric Low, Jacoline C Bouvy, John F. P. Bridges Introduction. The number of patient preference studies in health has increased dramatically. There is growing use of patient preferences in a wide variety of contexts, including health technology assessment. Patient preference studies can help inform decision makers on the needs and priorities of patients and the tradeoffs they are willing to make about health technologies. Methods. This International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force included international experts, health preference researchers and others from diverse backgrounds, including regulatory, health technology assessment, medicine, patient advocacy, and the pharmaceutical industry. The report underwent two rounds of written reviews by ISPOR Preferences Special Interest Group members until a final consensus was reached. The Task Force focused on developing a roadmap that would: (i) apply to the wide variety of preference methods, (ii) identify key domains to guide researchers and other stakeholders in making patient preference studies more useful to decision makers, and (iii) detail important questions to guide researchers conducting preference studies and those critically appraising them. Results. This Task Force report provides a novel roadmap that invites patient-preference researchers to work with decision makers, patients and other stakeholders to do even more to ensure that studies are useful and impactful. The ISPOR Roadmap consists of five key elements: (i) Context; (ii) Purpose; (iii) Population; (iv) Method; and (v) Impact. In this report, we define these five elements and provide good practices on how patient-preference researchers can actively contribute to increasing the usefulness and impact of patient preference studies in decision-making. We also present a set of key questions that can support researchers and other stakeholders in assessing efforts that promote preference studies' intended and unintended impact. Conclusions. This roadmap can help increase the usefulness and impact of patient preference studies in decision-making by challenging researchers to engage and partner with decision makers, patients and others, and together consider the intended and unintended impacts of patient preference studies on decision-making while actively fostering positive impact.
Oral Presentation Number: OP88 Abstract Title: Translating Patient Reported Measure Score Into Specific Outcomes From The Patient Perspective–Example Using Health Assessment Questionnaire-Disability Index Author Names: Chenglong Han ([email protected]), Marcie Strauss, Fareen Hassan, Sarah Alulis, Oluwakayode Adejoro, Adrian Griffin Introduction. To facilitate communication of clinical study results among patients, clinicians, and payers, this post-hoc analysis examined the association between achieving the Health Assessment Questionnaire-Disability Index (HAQ-DI) clinical meaningful improvement (CMI) and normalization of individual daily activities using pooled clinical trial data of patients with moderate-severe psoriatic arthritis (PsA). Methods. Patients with active PsA (n=1,120) who participated in two Phase III trials were administered the HAQ-DI at baseline and weeks (W) 24 and 52. The HAQ-DI is a patient-reported instrument measuring functional status that is validated for rheumatological diseases including PsA. It contains 20-items assessing activities of daily living using a 0-3 Likert scale [0=able to perform activity without difficulty (normalization) through to 3=unable to perform activity]; 0.35 or more improvement in total HAQ-DI represents the CMI in PsA patients. Study participants with baseline item-specific scores more than zero (indicating some level of impairment) who experienced both .35or more improvement in total HAQ-DI and item-specific score equal to zero, were compared over time. Results. The proportion of patients with compromised ability to perform individual activities at baseline ranged from 42.3 percent (Difficulty Turning Faucet) to 84.1 percent (Difficulty Doing Chores). Fourteen of 20 activities with 60 percent or more of patients reporting impairment at baseline decreased to one of 20 activities in patients reaching the CMI at W24. Fourteen of 20 activities with 50 percent or more of patients reporting no impairment at W24 increased to 18 of 20 activities at W52. Notably, the proportion of patients reporting impairment for the two outlier activities at W52 (Reach-Get Down 5lb Item/Doing Chores) decreased during the follow-up period by 28 percent and 30 percent, respectively, despite presenting as very challenging at baseline. Conclusions. PsA patients achieving a clinically meaningful change in total HAQ-DI over time were more likely to achieve normalization of individual daily activities. Translating changes in patient-reported outcome scores to specific symptom or functional improvements are meaningful to help patients understand clinical trial results, as well as to communicate with payers the value of accessing treatment for specific health conditions.
Oral Presentation Number: OP90 Abstract Title: Multiple Myeloma: Developing A Benchmark Patient Experience Index In Australia And New Zealand Author Names: Simon Fifer ([email protected]), Bronwyn West, Jenni Godsell, Nella Combe, Todd Stephenson Introduction. Within Australia and New Zealand (ANZ) there is limited evidence regarding the experience and satisfaction across the healthcare system of people living with multiple myeloma (MM). We aimed to quantify the patient experience across the healthcare system to help identify potential areas of the healthcare pathway that could be targeted for improvement to maximize patient satisfaction. Methods. A 30- to 40-minute online survey was completed by adults in ANZ diagnosed with MM. Anchored best-worst scaling (ABWS) is a technique that takes advantage of an individual’s ability to reliably identify extremes (‘best’ and ‘worst’) in sets of items, eliciting discriminating rankings free of scale bias. This study implemented a novel anchoring process to rescale importance and satisfaction best-worst scores for factors across the MM healthcare pathway, which could be compared and combined to form a patient experience index (PEI). There were 15 factors or ‘moments that matter’ (MTM), each describing a different aspect of the patient journey, such as time to diagnosis, treatment logistics, and side effects. The MTM were derived from qualitative research with patients as well as a workshop with key opinion leaders. Additional survey questions were included to help identify potential ways to improve patient satisfaction. Results. The results were based on 62 patients with MM. The overall median PEI score was 63.1. The top three MTM that were most important to patients, but they were least satisfied with (calculated by combining the top four of each most important/least satisfied factor for each participant), were side effects of medication, effectiveness of medication, and medication access. Conclusions. The findings from this research contribute to the understanding of patient experiences of treatment and care for MM. The results can inform healthcare decisions for prioritizing interventions that align with patient experiences. In the future, the study could be executed longitudinally to assess shifts in satisfaction within the MM healthcare journey, which would be especially worthwhile if new programs are implemented to improve patient satisfaction.
Oral Presentation Number: OP92 Abstract Title: The Hidden Burden Of Patients And Families In Rare Diseases: A Scoping Review Of Economic Evaluations Author Names: Gillian Currie ([email protected]), Brittany Gerber, Diane Lorenzetti, Karen MacDonald, Riley Jewel Bohach, Deborah Marshall Introduction. There are more than 7000 rare diseases (RDs), which are individually rare but have a large collective impact on patients and families, the health system, and society. There are few treatments for RD; where treatments do exist, they are often exceptionally expensive. Understanding the socioeconomic burden (SEB) of RD is crucial to properly valuing these treatments and informing health technology assessment. Our team has developed a framework of cost elements for inclusion in studies of the costs of RDs using an evidence-informed consensus- based approach. Methods. We conducted a scoping review to identify published economic evaluations studies in RD, searching five electronic databases to identify English language RD studies published 2010-2021. We applied our framework of cost elements to assess studies regarding what cost elements were included. Results. Of 4,890 records identified, 48 studies were screened for inclusion. Most were from the US (n=27), UK (n=6), and Canada (n=6), and focused on hemophilia (n=14) or cystic fibrosis (n=11). Healthcare system and payer perspectives were most often reported (n=41), with only seven studies reporting a societal perspective. Cost elements most often included were medications (n=41), hospitalizations (n=35), surgery (n=20) medical tests (n=16), and outpatient (unspecified) visits (n=16). Costs to patients, families, and society were less commonly included: productivity (n=5), travel/accommodation (n=3), government benefits (n=2), family impacts (n=0), or other costs relevant to RD (n=1). While unsurprising, given that most analyses focused on healthcare or payer perspectives, this finding illustrates the extent to which the burden of RD is largely unstudied. Conclusions. Our scoping review demonstrated that most studies are conducted from a healthcare system/payer perspective, and largely consider only medical costs. These studies undercount the hidden burden of rare disease borne by patients and families leading to a gap in our global understanding of the full impact of rare diseases on families. To properly account for the these and value emerging treatments for RD, patient- and family-borne costs must be considered in economic evaluations for health technology assessment.
Oral Presentation Number: OP93 Abstract Title: The Cost Effectiveness Of Antiretroviral Therapy Adherence Interventions In HIV/AIDS Patients: A Systematic Review Of Decision Analytic Models Author Names: Ali Ahmed ([email protected]), Juman Dujaili, Long Le, Saval Khanal, Lay Hong Chuah Introduction. People living with HIV/AIDS (PLWHA) frequently struggle to maintain optimal adherence to antiretrovirals (ARVs). Different adherence-improving interventions have been developed and examined through decision analytic model-based health technology assessments. Therefore, we aimed to conduct a systematic review of all decision analytic models developed to improve adherence in PLWHA. Methods. The review protocol was registered on Prospero (CRD42022270039) and relevant studies published from inception to 23 October 2022 were identified through searches of the following databases: PubMed, Embase, the National Health Service Economic Evaluation Database, PsycINFO, the Health Economic Evaluations Database, and EconLit. Studies were included if they were modeling works of full economic evaluations, including cost-effectiveness analyses (CEA), cost-utility analyses, cost-benefit analyses, and cost-consequence analyses. The primary outcome was the cost effectiveness of adherence interventions reported as the incremental cost per additional quality-adjusted life-year (QALY). Study quality was assessed with the Quality of Health Economics Studies instrument. Due to the heterogeneity of the data, a permutation matrix was used for quantitative data synthesis rather than a meta-analysis. Results. The 15 studies identified were conducted in North America (8/15), Africa (4/15), and Europe (3/15). The time horizon was one year in one study, ten years in one study, 20 years in three studies, and a lifetime horizon in ten studies. The types of interventions were smartphone-based (5/15), nurse involved (2/15), directly observed therapy (2/15), case manager involved (1/15), simplification of regimens (1/15), Link4Health (1/15), and others (3/15) that involved multicomponent intervention. The interventions gained higher QALYs with cost savings in all 15 studies and gained QALYs at a higher cost at an acceptable incremental cost-effectiveness ratio in 80 percent (12/15) of studies. The studies were of fair (13%) to high quality (87%). Conclusions. This study is the first systematic review of decision analytic model-based CEAs of adherence interventions in the management of PLWHA. Most of the identified studies recently published good quality cost-effectiveness analyses with an adequate timeframe.
Oral Presentation Number: OP94 Abstract Title: Economic Evidence On Hemodialysis Access Creation Procedures In Patients With End-Stage Kidney Disease: A Systematic Literature Review Author Names: Ritu Gupta, Upasna Gaba, Christopher Delaney, George Papadopoulos ([email protected]) Introduction. It is important to create and maintain durable hemodialysis (HD) access in health systems to reduce morbidity and maintain overall cost control in patients with end-stage kidney disease (ESKD). To evaluate the choice of HD vascular access creation procedures and their related economic costs, we aimed to identify economic evaluations on vascular access (VA) creation procedures in patients with ESKD. Methods. A systematic literature review was conducted using the Cochrane methodology to identify cost-effectiveness analyses (CEAs), budget impact analyses, and cost analyses of various HD access creation procedures. Eligible publications published from 2012 onwards were retrieved by searching PubMed, Embase, and the Cochrane Library. The Consolidated Health Economic Evaluation Reporting Standards 2022 checklist and ISPOR Task Force guidelines were used to appraise the quality of the economic evaluations and budget impact analyses, respectively. Costs were adjusted for inflation and purchasing power parity and standardized to US dollars. Results. A total of 40 economic evaluations met the inclusion criteria, including 28 cost analyses, three budget impact analyses, and nine CEAs. Widely evaluated procedures in the published literature were endovascular and surgical arteriovenous fistula (AVF), arteriovenous graft (AVG), and central venous catheterization (CVC). The results indicated that AVF was the most cost-effective strategy, followed by AVG, and CVC. Three studies showed that endovascular AVF was cost effective, compared with surgical AVF, and resulted in overall cost savings of about USD53 million dollars over a five-year period. Results of the quality assessment showed that budget impact analyses scored 63 percent, while the average score for economic evaluations was 58 percent. Conclusions. It was challenging to identify a single effective method of managing vascular access due to the substantial heterogeneity among VA creation techniques. However, most of the included economic evaluations showed that AVF was a cost-effective method of VA creation relative to other identified techniques for patients with ESKD on HD.
Oral Presentation Number: OP95 Abstract Title: A Systematic Review Of The Cost And Cost Effectiveness Of Immunoglobulin Treatment In Patients With Hematological Malignancies Author Names: Sara Carrillo De Albornoz ([email protected]), Khai Li Chai, Alisa M. Higgins, Dennis Petrie, Erica M. Wood, Zoe K. McQuilten Introduction. Patients with hematological malignancies are likely to develop hypogammaglobulinemia (HGG) and subsequent infections. Immunoglobulin (Ig) replacement is commonly given to prevent infections, but the total costs and cost effectiveness of its use are unknown. Methods. A systematic review was conducted following PRISMA guidelines to assess evidence on the costs and cost effectiveness of Ig replacement, administered intravenously (IVIg) or subcutaneously (SCIg), in adult patients with hematological malignancies. This review was registered with PROSPERO (CRD42022321908). Results. Six studies were included out of a total of 3,612 citations. A narrative synthesis was conducted because of the high level of heterogeneity across the included studies. Two economic evaluations were identified: one cost- utility analysis (CUA) of IVIg versus no Ig and one comparing IVIg with SCIg. The quality of the evidence was low, with most studies having small patient numbers and a high risk of bias. Compared with no treatment, Ig replacement reduced the hospitalization rate in patients with hematological malignancies. One study reported no change in hospitalization rates following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg found more hospitalizations with SCIg but lower total costs per patient. The CUA comparing IVIg with no IVIg suggested that IVIg treatment was not cost effective, but this study was published in 1991 and had significant limitations. The other CUA found that home-based SCIg was more cost effective than IVIg, but model inputs were derived from unpublished data in a very small patient cohort with HGG and different malignancies. Conclusions. Our review highlights key gaps in the literature. The cost effectiveness of Ig replacement in patients with hematological malignancies is still very uncertain. Despite the increasing use of Ig replacement there are limited data regarding its direct and indirect costs, and its optimal use and implications for healthcare resources remain unclear. Given the paucity of data on the cost and cost effectiveness of Ig treatment in this population, further health economic research is warranted.
Oral Presentation Number: OP96 Abstract Title: Adapting Patient Involvement For Fast Track Appraisals Author Names: Mark Rasburn ([email protected]), Helen Crosbie, Laura Marsden Introduction. The National Institute for Health and Care Excellence (NICE) is piloting a new innovative approach to the way digital products, devices, and diagnostics that most reflect system need and demand are assessed. This early value assessment (EVA) approach will allow a more rapid assessment to enable patients to benefit from promising technologies sooner. Involving patients in the health technology assessment (HTA) lifecycle is a core principle at NICE, but established methods are not suitable for a rapid timeframe. NICE needs to adapt the approach to ensure that patients are supported to participate in EVAs and that their involvement is meaningful. Methods. Due to the rapid timeframe, it was important to ensure patient contributors were not overloaded with information and that contact points were aligned. NICE reviewed the standard induction, support documents, and contact points to adapt the support provided. This included: • updating recruitment documents to communicate the role of the committee and the EVA process; • combining induction meetings between various NICE teams and providing recorded presentations; • organizing earlier peer support with experienced lay members; and • advising which of NICE’s nine online modules were most relevant. Results. Support for patient contributors has been an important part of the HTA process, so enabling people to prepare and confidently deliver content at a committee meeting is vital. There has been some variation in the processes for different topics, but the feedback received from patient contributors indicated that their involvement was meaningful and valued. This was attributed to their close working relationship with the project team. NICE is collecting feedback from all patient contributors using an online survey. The findings of this survey and the evaluation of the support mechanisms will be presented. Conclusions. Despite shorter timeframes, patient involvement has not been compromised. NICE will use the feedback from patient contributors to review and adapt the induction process and support offered. This will support patient contributors and enable NICE to allocate appropriate resources in the shortened timeframe.
Oral Presentation Number: OP98 Abstract Title: Improving Patient Involvement In Health Technology Assessments: Is It Enough To Train Just The Patients? Author Names: Heidi Livingstone ([email protected]), Ella Fitzpatrick, Marsden Laura, Mandy Tonkinson, Sally Taylor Introduction. Patient involvement is a core principle of the National Institute for Health and Care Excellence (NICE) and we continually strive to improve patient involvement in health technology assessments (HTAs) of medicines. We iteratively surveyed and reviewed how patient involvement can be improved with patient organizations, patient experts, NICE HTA decision-making committees, and staff. We re-examined feedback that we collect on an ongoing basis, as well as one-off evaluations, to check how we can improve patient involvement. Improvements ranged from support for and how we work with patient stakeholders to training the various stakeholders who take part in the HTA process to build up a comprehensive and evolving training package and stimulate a cycle of continually improving patient involvement. Methods. We reviewed the outcomes and recommendations from the following larger projects: • Review of public involvement across NICE 2015; • Improving meaningful patient involvement in HTAs 2019; • Improving patient expert involvement in committee meetings 2019; and • The value of patient expert input 2022. Feedback from monthly surveys of patient experts and organizations was also reviewed. Results. The results included recommendations about: • Changing the culture so that patient involvement at NICE is everybody’s business; • The key role of the committee chair in including patient experts; • The importance of committee culture and behavior in including and valuing patient input; • The need for a greater understanding of how NICE technical teams can best support and obtain the most meaningful evidence from patients; and • What additional support and training patient organizations and experts want from NICE’s public involvement team. Conclusions. We concluded that not only patients need training, but also everybody included in the NICE medicines HTA process. Over time we have gradually added to our training portfolio for patient organizations and experts as well as NICE staff and independent committees. We now run patient involvement as part of the induction
program for all staff, technical staff, medicines committee chairs, and NICE committees and lay members. We also provide monthly training for patient organizations and patient experts.
Oral Presentation Number: OP100 Abstract Title: Patient Perspectives In Value Assessment Frameworks: The Asia Pacific Perspective Author Names: Alex Best ([email protected]), I-Ching Tsai, Jin Yu Tan, David bin-chia Wu, DaeYoung Yu, Alison Keetley, Durhane Wong-Rieger, Ritu Jain Introduction. The importance of patient centricity in healthcare decision making has been recognized and advocated for decades. However, approaches for including the patient perspective are diverse, and progress varies among countries. Some reimbursement bodies acknowledge the importance of patient preferences in health technology assessment (HTA) and funding decision processes. However, patients’ perspectives are not yet systematically and transparently included in value assessment frameworks globally, and even less so in the Asia-Pacific (APAC) region. This systematic review aimed to investigate how patients' perspectives are used to inform pricing and reimbursement decisions in the APAC region. Methods. A systematic review is ongoing that utilized a search of 12 databases, including MEDLINE and Embase, to identify publications on the consideration of patient perspectives in health policy decision-making published to November 2022. Conference abstracts published in the last five years from ISPOR and Health Technology Assessment International (HTAi) were screened, along with gray literature and government websites from Australia, China, Japan, Malaysia, New Zealand, the Philippines, Singapore, South Korea, Taiwan, and Thailand. Publications were included if the impact of either one or more of the following on HTA decision-making was assessed: active participation of patients or patient advocacy groups; type, extent, and evolution of patient- reported outcomes; health-related quality of life or quality of life tools; and themes where the impact of patients' perspectives on value assessment was the primary outcome. Countries were characterized into archetypes based on similarities or differences in the weight and value assigned to patient perspectives in decision-making. Results. A total of 6,438 retrieved citations will undergo the systematic review process. Additionally, 758 conference abstracts from ISPOR, 1,312 from HTAi conferences and 73 records from gray literature will be screened. The results of the systematic review will be consolidated into country archetypes, examples, and learnings. Gaps and opportunities will also be identified. Conclusions. The research will provide recommendations to increase shared decision-making and support the development of decision-making frameworks that systematically incorporate patients' perspectives in value assessment across APAC countries.
Oral Presentation Number: OP102 Abstract Title: Towards Universal Health Coverage: Health Technology Assessment Roadmap Development In The Emirate Of Abu Dhabi Involving The Whole Ecosystem Author Names: Amna Alsaeedi ([email protected]), Dirk Richter, Hamda Alazeezi, Farah Nassri, Mahmoud Wael, Wija Oortwijn, Leon Bijlmakers Introduction. The mission of the Department of Health (DoH) of Abu Dhabi in the United Arab Emirates is to provide its population with a healthy life and world leading preventive and curative services. While the DoH has regulations in place to grant market approval to new health technologies, there is a need to develop a clear overall framework for reimbursement and disinvestment decisions. Establishing a structured health technology assessment (HTA) framework is critical for informing decisions on health technologies that offer value for money, with the aim of improving equitable access to health care, financial risk protection, and, ultimately, better health outcomes. Methods. During 2022, the DoH collaborated with the Radboud University Medical Center to explore the feasibility of applying an evidence-informed deliberative process (EDP) HTA approach through workshops and interviews involving all stakeholders in the ecosystem, such as policy makers, principal investigators, providers, patients and public groups, product manufacturers, payers, and purchasers. A situational analysis was conducted to collect stakeholders’ views and build EDPs. Based on this analysis, a structured roadmap was developed. Results. The comprehensive five-year roadmap to implement a holistic HTA framework in Abu Dhabi consisted of five major elements, starting with the establishment of an appropriate HTA policy framework as a foundation. Abu Dhabi should firmly establish its HTA structure and program (in one to two years), and at the same time invest in developing and retaining HTA training capacity so that over time (within three to five years) the country can build up its own expertise to sustain the program. This needs to be accompanied by continuous awareness raising among all relevant stakeholders. Conclusions. This roadmap is the first and most important step toward implementing a holistic HTA framework in Abu Dhabi. Technical work needs to be complemented by continuously raising awareness and involving all relevant stakeholders. Looking at the initial results and international benchmarks, HTA will significantly contribute to achieving a sustainable, high quality healthcare system.
Oral Presentation Number: OP103 Abstract Title: What To Include In A Health Technology Assessment Of Artificial Intelligence-Based Technologies: Results Of A Delphi Expert Survey Author Names: Signe Daugbjerg, Rossella Di Bidino ([email protected]), Americo Cicchetti Introduction. Clinicians are increasingly relying on artificial intelligence (AI) generated technologies for support in diagnosis, therapeutic decision-making, and prediction. Despite the increased focus on AI in health, an agreed HTA model for AI technologies, including consensus on new domains and topics to be assessed, is lacking. Methods. A Delphi survey was sent to a multidisciplinary expert panel asking about the importance of including the nine domains and associated topics presented in the EUnetHTA Core Model, as well as 20 additional topics identified through literature reviews, when assessing AI-supported health technologies. The Delphi survey was repeated twice among the same panelists and a nine-point Likert scale was used to identify the perceived relevance of each domain and topic. Results. The survey was sent to 87 various experts, with a total 47 of experts completing both Delphi rounds. The majority of panelists was knowledgeable of HTA (80%), familiar with the EUnetHTA Core Model (61%), and had adequate or high-level knowledge of AI (65%). The EUnetHTA domains most often indicated as “critical to include” were clinical effectiveness (82%), ethical aspects (81%), and cost effectiveness (77%), whereas organizational (59%) and social aspects (63%) were less often perceived as critical to assess. For the additional 20 topics identified through literature reviews, bias in data, accuracy in the AI model, appropriateness, and trustworthiness emerged as some of the new topics deemed critical to include in HTAs (all above 85%), whereas there was a lack of agreement on the relevance of including environmental (51%) and social sustainability (55%). Conclusions. The study investigated in detail which issues should be included in an AI HTA core model. Current models need some adjustment and revision. At the same time, it is essential to open the discussion on including new domains and topics.
Oral Presentation Number: OP104 Abstract Title: Pilot Implementation Of Health Technology Assessment Topic Prioritization In The Philippines: Lessons And Plans For Moving Forward Author Names: Sheena Jasley Samonte ([email protected]), Lara Alyssa Liban, Princess Allyza Mondala, Jason Oliver Angeles, Russell Cabus, Patrick Wincy Reyes, Anne Julienne Marfori, Anna Melissa Guerrero, Health Technology Assessment Council Introduction. The Health Technology Assessment (HTA) Council in the Philippines carried out its process tracks while the implementing guidelines were being finalized in 2020, due to the urgent need to respond to COVID-19. Two years later, as mandated by the Universal Healthcare (UHC) law, we opened the nominations for the HTA Council’s topic priority list, which will be assessed to inform government financing decisions. Methods. We adopted the former Philippine National Formulary System (PNFS)but set the prioritization criteria according to the decision framework stipulated by the UHC law and allowed industry submissions. We streamlined dossier completion for topics with numerous proponents, supplemented dossier deficiencies, and adjusted the timelines of crucial steps for better reach, while accounting for possible setbacks during the time periods. We satisfied the prioritization criteria using a Delphi technique at the HTA Council subcommittee and Core Committee levels in conjunction with consultations with the Department of Health and the national payer. We also shared evidence base and topic prioritization criteria scores with stakeholders during the public consultation. Results. In the pilot implementation, we processed a total of 140 nominations (88 complete submissions) and released the priority list in five months. After processing 31 appeals from all key stakeholder groups, the 2022 priority list covered 31 assessments based on topics from the Department of Health, the national payer, industry, hospitals, and medical societies. Although we found gaps in the set timelines, inadequacy in the prioritization criteria parameters, and the need to increase exposure of the public to the process, we were able to accommodate all stakeholder concerns and maintain flexibility in the process. Conclusions. We need to update our HTA process guidelines, accept joint dossier submissions, and review our topic prioritization process. The changing health system landscape and transitioning of national health priorities require coordination with the Philippine Food and Drug Administration for horizon scanning, early HTA, and managed entry agreements. Finally, there is a need to create special pathways for rare disease and innovative technologies.
Oral Presentation Number: OP106 Abstract Title: Standing On The Shoulders Of Giants: Adapting Published Health Technology Assessments On Medical Technologies To Singapore’s Context Author Names: Lydia Ooi ([email protected]), Rachel See-Toh, Swee Sung Soon, Hong Ju, Kwong Ng Introduction. Given time and resource constraints, health technology assessments (HTAs) should avoid duplicating prior HTAs. This abstract describes the experience of the Agency for Care Effectiveness (ACE) with adapting and building on published HTAs of medical technologies to support efficient decision-making. Methods. Upon defining the scope of an evaluation topic, searches were performed on reference agency websites and HTA networks, such as the International Network of Agencies for Health Technology Assessment, to identify relevant published HTAs. Identified reports were included for evidence synthesis if the inclusion criteria were met. If multiple relevant HTAs were identified, the most recent or comprehensive HTA identified was used as the foundational evidence base and supplemented by additional primary or secondary literature as needed. When deemed necessary, de novo economic models were developed. Where available, reimbursement decisions or recommendations from published HTAs and their applicability to Singapore’s context were also considered. Following ACE’s evaluations, the Ministry of Health Medical Technology Advisory Committee (MTAC) made subsidy recommendations. Results. To date, ACE has completed 54 HTA evaluations of medical technologies, 47 (87%) of which incorporated published HTA evidence. Although some MTAC recommendations were aligned with published HTAs, discrepancies were observed that were mainly attributed to evolution of the technology or evidence since publication of HTAs; different local clinical needs or management algorithms; and different local costs of the intervention and comparator. Key challenges in adapting published HTAs included differences in the target population and intervention or comparator in published HTAs, mixed recommendations from published HTAs, and non-English language reports, which may underscore the need to adapt published HTAs to Singapore’s context. To overcome some of these challenges, close consultation with stakeholders is critical to understand local clinical need and care pathways and to gather key information such as costs. Conclusions. Published HTAs, when used as an evidence base and supplemented by evidence updates and local contextualization, allow for efficient and robust HTA to support decision-making.
Oral Presentation Number: OP107 Abstract Title: Streamlining Health Technology Assessment Of Medical Devices Through Development Of The Philippine Essential Medical Device List Author Names: Patrick Wincy Reyes ([email protected]), Lara Alyssa Liban, Ma. Cecilia Victoria Arellano, Joshua Santillan, Sheena Jasley Samonte, Anne Julienne Marfori, Anna Melissa Guerrero, Marc James delos Santos, Catherine Manuela Lee-Ramos, Agnette Peralta, Department of Health - Medical Device Unit, Health Technology Assessment Council Introduction. Pursuant to the Universal Health Care Act of 2019, the Health Technology Assessment Council (HTAC) in the Philippines was mandated to make recommendations for government financing of health technologies, including medical devices. The development of a Philippine Essential Medical Device List (PEMDL) will serve as a guide for the procurement of medical devices and supplies in government health facilities and as the basis for creating a price reference index. Methods. The HTAC and a team of medical device experts (the Expert Advisory Committee) developed a streamlined pathway and guidelines using a Delphi technique with the Philippine Food and Drug Administration (FDA) and the Department of Health (DOH) to determine the necessary criteria for fast tracking the inclusion of medical devices. The pathway was also revised throughout a pilot processing of commonly procured medical devices (e.g., consumables) that were either already covered by existing national health insurance packages or funded by DOH programs, among others. Results. The final pathway consisted of validating whether the device was already approved by the Philippine FDA and whether it was already part of standard of care. Similar to the usual HTA nomination requirement, the local FDA approval attested to the safety and quality of the device. Meanwhile, the inclusion of the device in the standard of care guaranteed that it was essential in the healthcare setting. This can be written in the World Health Organization technical documents and databases such as the Medical Devices Information System, health facility listing requirements, or locally adopted clinical practice guidelines. The initial PEMDL for release contained a total of 307 medical devices across 16 categories. Conclusions. Unlike the mandate for the Philippine National Formulary, government health facilities are not yet required to base their procurement on the PEMDL because the list is still in its infancy. Moving forward, the list will include big ticket items and will be updated through consultations with specialty centers and hospitals
Oral Presentation Number: OP109 Abstract Title: The Conceptualization And Value Of A Disease Management Approach To HTA In Canada: Findings From A Qualitative Study Author Names: Marina Richardson ([email protected]), Fiona Miller, Nick Daneman, Chloe Mighton, Beate Sander Introduction. Our objective was to conceptualize and assess the potential value of a disease management approach to health technology assessment (HTA) in Canada. Methods. We conducted 18 semi-structured interviews between April 2022 and October 2022 to elicit informant views on potential opportunities for re-conceptualizing the decision-problem in HTA as a disease management problem versus a technology management problem. Participants were purposefully sampled from national and provincial HTA agencies and related organizations in Canada to achieve representation across the disease pathway including prevention, screening, and treatment, and the decision-making pathway including HTA organizations, expert committee members, and decision-makers. Data were analyzed using thematic organizations (based on the interview guide) and manual line by line coding of the data. Ethics approval was received from the Health Sciences Research Ethics Board at the University of Toronto. Results. Three key features of a disease management approach to HTA (i.e., disease-based, multi-interventional, and dynamic) emerged from informants that differed from traditional HTA processes in Canada. The concept was generally not perceived to be a new idea – some informants indicating that it was implicit in the HTA analysis framework. There was general support for an explicit disease management approach to HTA if the impact of the approach could be demonstrated, if the assessment could be completed within an appropriate time frame, and if the assessment could include the equity, ethical and implementation domains of HTA. Informants indicated that the reconceptualization of HTA could lead to effective and efficient decision-making throughout a technology’s lifecycle, help breakdown system silos, and offer a platform for greater consideration of non- drug alternatives and upstream interventions. The impact of this approach was anticipated to contribute to a proactive health system that could improve population health, enhance the patient experience, and ensure appropriate stewardship of health care resources. Conclusions. A disease management approach to HTA has international relevance as an approach that could promote integrated, proactive, sustainable, and resilient health systems.
Oral Presentation Number: OP112 Abstract Title: Evaluation Of A Self-Administered Smart Phone-Based Application As A Wellness Measure In A Clinical Trial Of Zanubrutinib Author Names: Rocco Crescenzo, Adam Idoine, Kunthel By, Keri Yang ([email protected]) Introduction. Assessments self-administered via smart phone-based apps can gather patient wellness data; however, data regarding app utilization in clinical trials are limited. This study evaluated engagement with a self-administered wellness app as a supplemental tool to assess quality-of-life (QoL) in study BGB-3111-215 (NCT04116437), which included patients with B-cell malignancies treated daily with oral zanubrutinib. Methods. This exploratory analysis evaluated the feasibility of using a voluntary device-based, self-administered activity and QoL questionnaire app (Medable, California, USA) in clinical trial patients. Feasibility was measured by rates of consent, utilization, and compliance. Consent was assessed by the percentage of patients who agreed to use the app. Utilization was assessed by the percentage of patients who engaged with the app. Compliance was assessed by actual versus scheduled engagements. The app also included passive activity tracking and a six-minute walk test. Results. As of 1 September 2022, 78 patients had enrolled in the study (median age 71 years) and 20 had consented to participate (26% consent rate). Eleven patients (median age 65 years) engaged with the app at least once (14% utilization rate; n=11 for the questionnaire; n=7 for the walk test). Engagement occurred a median two times (range 1 to 17) for the questionnaire and a median seven times (range 1 to 15) for the walk test. Four patients engaged with the questionnaire and six patients engaged in the walk test beyond week 12. Passive activity was collected for eight patients beyond week 12. Among patients who engaged with the QoL questionnaire and walk test, compliance rates were 18 and 21 percent, respectively. Conclusions. This initial study showed that a subset of patients was willing to participate in the self-administered QoL questionnaire and activity tracker. Further comparison of app results and clinical trial findings are ongoing to explore factors affecting utilization and compliance rates.
Oral Presentation Number: OP114 Abstract Title: Health Technology Assessment Of Digital Technologies: Results From Two Past Examples And Thoughts For The Future Author Name: Jamie Erskine ([email protected]) Introduction. Digital health care is a rapidly growing and increasingly diverse area of medicine and social care. Health technology assessment (HTA) agencies around the world are engaging with digital health technologies and developing methods for appropriately assessing their value. Given our experience in HTA for pharmaceuticals and how methodologies must differ when assessing medical devices and diagnostics, we must be proactive in rapidly developing useful methods for assessing a variety of digital health technologies. Methods. We will first present the results of two completed HTAs from the UK and contrast their methods and results, while looking at the UK approach to assessing digital health care in general. Next, we will present preliminary results from an ongoing consensus conference study of HTA’s for integrated digital technologies. This study will use a modified Delphi methodology to develop statements for discussion at a consensus conference. The primary research question is “how digital technologies can help build resilient healthcare systems and how HTAs can assess their value”. Results. An HTA conducted in the UK of an artificial intelligence-enabled ambulatory electrocardiogram device resulted in a recommendation for further real-world evidence generation. Another HTA of a digital therapeutic app for insomnia, which was also conducted in the UK, resulted in a positive recommendation but the process was lengthy and needed to be flexible. The preliminary results of an upcoming consensus conference study will also be presented. The consensus panel will consist of a range of stakeholders from digital experts, HTA practitioners, clinicians, health economists, industry representatives, and policy makers. Conclusions. Digital technology is now a broad field with a wide range of very different technologies included in its definition. Questions remain as to whether digital technologies should be assessed using a traditional pharma- based framework for HTA, a MedTech model, or something else entirely. Given our past experience of developing flexible methods for HTA, we have an opportunity to develop useful recommendations and best practices from the ground up.
Oral Presentation Number: OP115 Abstract Title: Scoping Meta-Review On Methods Used To Assess Artificial Intelligence-Based Medical Devices For Heart Failure Author Names: Rossella Di Bidino ([email protected]), Davide Piaggio, Ernesto Iadanza, Leandro Pecchia, Martina Andellini, Raza Zeeshan, Beatriz Merino-Barbancho, Laura López Pérez, Giuseppe Fico, Melody Ni, Simone Borsci, Tianhui Zhu Introduction. The International Federation of Medical and Biological Engineering created a multidisciplinary working group to discuss assessments of artificial intelligence and machine learning (AI/ML) applications in health care. Engineers, clinicians, and economists identified evidence generation as a critical topic. Heart failure (HF) was selected to investigate the available evidence on the clinical effectiveness and safety of AI/ML applications. Attention was paid to transparency of AI/ML methods and their data sources. Methods. A scoping review was conducted on AI/ML algorithms developed for the management of HF. A search for systematic reviews, scoping reviews, and meta-analyses published from 1976 to October 2022 was conducted in Embase, MEDLINE, and Scopus. Results. Of 456 relevant publications, 21 papers were included in the final analysis. Most papers (10 systematic reviews, five meta-analyses, and six non-systematic or scoping reviews) included studies conducted in North America. No study was conducted in Africa. The healthcare setting was not clearly stated in approximately half of the studies. A lack of agreement was noticed regarding the quality assessment tools used among the reviews. The most common data source for AI/ML algorithms was electronic health records, but in some cases data sources were not reported. While deep learning emerged as the most common adopted methodology, covariates were not always included in the algorithm development. The review demonstrated that comparative assessment of algorithms requires further investigation, given the high variability in the comparator used (e.g., clinical gold standard, other AI/ML algorithms, or other statistical methods). The main investigated endpoints were the incidence of HF and the number of hospital admissions. Conclusions. When assessing innovative health technologies such as AI/ML applications in health care, evidence is among the main challenges. Our scoping review, focusing on algorithms developed to manage HF, showed that the biggest challenges relate to the quality of the studies, the adoption of a comparative approach, and transparency of methods.
Oral Presentation Number: OP116 Abstract Title: Building Alignment Between Industry, Academia, And Patients On Health Technology Assessment Methods Author Names: Colman Taylor ([email protected]) Introduction. With the announcement of the health technology assessment (HTA) review in Australia, a HTA Summit was organized by ISPOR Australia in November 2022. The aim of the Summit was to provide a forum for industry, academia, and patients to share ideas and find common ground with respect to HTA policy, processes, and methods. Topics were determined by a Steering Committee and included: managing uncertainty; patient engagement; second order effects; genetics, genomics and precision medicines; conditional listing; and real- world evidence. Presentations on each topic were conducted by industry and non-industry experts. Breakout sessions led by facilitators were also held for each topic with members of the audience. Methods. Discussions were recorded during the event and a thematic analysis was performed. Results. The following themes were identified from the event. • There was a strong sentiment that participants enjoyed the opportunity to discuss ideas and work toward solutions. • There was a consistent theme that many of the issues arising in HTA were due to a lack of communication between sponsors, evaluators, patients, and decision makers. • It was noted that HTA encompasses several technical terms that have different meanings among various stakeholders. • There was a clear consensus that patients should be involved in HTA earlier and throughout the process. • HTA reform can help drive better access to real-world evidence. • To improve the efficiency of the process, uncertainty could be reframed as risk management, which incorporates the effect of uncertainty in the funding decision. • HTA includes policies, processes, and methods and is used as a tool by decision makers to make informed funding decisions. It was noted on several occasions during the Summit that funding decisions have a political element that should be separated from the HTA process. Conclusions. It is possible to achieve better collaboration between industry, academic, and patient groups with respect to HTA reform. To promote more collaborative work a consistent conflict of interest definition would be helpful.
Oral Presentation Number: OP119 Abstract Title: Collectively Improving The Quality Use Of Highly Specialized Medicines: Starting With Biologics Author Names: Jonathan Dartnell ([email protected]), Sue Brown, Louise Hardy, Claire Barrett, Rachelle Buchbinder, Sam Whittle, Lisa Pulver, Nena Nikolic, Debra Rowett, Kylee Hayward, Catherine Hill Introduction. In contrast to high-volume medicines prescribed by general practitioners, low-volume highly specialized medicines have not been supported by national quality use of medicine (QUM) programs in Australia. The first area addressed has focused on optimizing use of biological disease-modifying antirheumatic drugs (bDMARDs). Methods. The program was designed, developed and implemented in partnership with nine consortium member organizations and four affiliate organizations representing consumer and clinical audiences, program development expertize and implementation capability. The common agenda for the collective impact approach was to achieve better health outcomes for people with inflammatory arthritis, inflammatory bowel disease and plaque psoriasis. Multidisciplinary expert working groups reviewed formative QUM research and agreed on objectives, audiences, messages and interventions. Interventions were selected based on identified barriers, enablers and behavioral drivers, informed by the Theoretical Domains Framework. Interventions were co-designed and tested with end-users. Marketing and promotion activity supported implementation of all interventions through consortium channels and networks. Evaluation includes process, impact and outcome measures, and a realist evaluation of the academic detailing. Results. Program objectives were to optimize: (i) first-line therapy before bDMARD use; (ii) first-choice bDMARDs; (iii) biosimilar prescribing and dispensing; (iv) bDMARD dosage; (v) glucocorticoid and analgesic use. Over 60 interventions supporting key messages for each objective were developed for audiences: consumers; rheumatologists, gastroenterologists, dermatologists; pharmacists; drug and therapeutic committees. Interventions implemented between September 2020 and September 2022 included: consumer decision aids, action plans, fact sheets, lived experience videos; living guidelines and evidence summaries; guidance/position statements for hospitals, podcasts, webinars, online learning; prescribing feedback reports; and academic detailing. Uptake of interventions has largely met targets and surveys have demonstrated shifts in specialist and consumer knowledge and behavior in line with key messages and objectives. Realist and outcome evaluation is ongoing.
Conclusions Our experience demonstrates the value of a consortium of stakeholder organizations, with different expertise and interests but agreed goals and roles, working together to progress the quality use of highly specialized drugs.
Oral Presentation Number: OP120 Abstract Title: Developing A Call To Action For Patient Involvement In Health Technology Assessment (HTA) In Southern Africa. Author Names: Lauren Pretorius ([email protected]), Debjani Muller, Anke Peggy Holtorf Introduction. In building health technology assessment (HTA) and related decision processes in Southern Africa, institutions and stakeholders face region-specific challenges such as disease prevalence and population makeup. These can be addressed by collaboratively discussing patient engagement solutions that fit the local culture and systems and serve to ensure equitable and sustainable access to patient-relevant health technologies. Our aim is to initiate a collaboration for driving patient involvement (PI) suitable for the Southern African context and Sub-Saharan patient communities. In addition, we explore current experiences in PI, including the stakeholder expectations, gaps, limitations, and new opportunities. Methods. A one-day hybrid multi-stakeholder PI in HTA workshop was held in Johannesburg, South Africa. Co-created by the participants, the outputs are a call to action and a concept draft for the vital success criteria for PI in the region. The content of the call to action is gathered from pre-workshop surveys, interviews, and outcomes from historic meetings held in conjunction with the Health Technology Assessment International (HTAi) PI workstream as well as facilitated discussion from the actual workshop. Results. The workshop was attended by 42 participants from nine countries, representing diverse stakeholder groups. The attendees represented multiple PCI stakeholder groups. The workshop survey was completed by 44 respondents, while 12 participants completed the post-event survey. A workshop outcomes document highlighting a high level of alignment and identifying seven key success factors was developed. A workshop proceeding detailing the outcomes is now being drafted. Conclusions. Over 95 percent of respondents to pre-and post-surveys indicated an interest in contributing to a more in- depth description of PCI in their country. While the majority of participants were from South Africa, participants from Tanzania, Ethiopia, Zambia, and Zimbabwe emphasized that trans-African-engagement for HTA will provide an additional opportunity for HTA in Africa and patient- and community participation in HTA and healthcare decision-making. Hence, a collaborative platform could help all African countries to advance and benefit from improved healthcare decision processes.
Oral Presentation Number: OP121 Abstract Title: Cost-Utility Analysis Of A Supervised Exercise Intervention For Women With Early-Stage Endometrial Cancer Author Names: Yufan Wang ([email protected]), Alexandra L. McCarthy, Haitham Tuffaha Introduction. Cardiovascular disease (CVD) is the leading cause of death in women diagnosed with endometrial cancer. There is clinical evidence that exercise significantly reduces the risks of CVD and cancer recurrence; however, it is unclear whether there is value for money in integrating exercise into clinical cancer care for this population. This paper aimed to assess the long-term cost effectiveness of a 12-week supervised exercise intervention, compared with standard care, for women diagnosed with early-stage endometrial cancer. Methods. A cost-utility analysis was conducted from the Australian health system perspective for a time horizon of five years using a five percent discount rate. A Markov cohort model was designed with six mutually exclusive health states: no CVD; post-stroke; post-coronary heart diseases; post-heart failure; post-cancer recurrence; and death. The model was populated from the best evidence available in the literature. The incremental cost- effectiveness ratio (ICER) and net monetary benefit were reported. Uncertainty in the results was explored using deterministic and probabilistic sensitivity analyses. Results. Over the time horizon of five years, the incremental cost of supervised exercise versus standard care was AUD358 (USD236.74) and the incremental quality-adjusted life-year (QALY) was 0.079, resulting in an ICER of AUD5,148 (USD3,404) per QALY gained. The incremental net monetary benefit was AUD3,589 (USD2373.24) and the likelihood that the supervised exercise intervention was cost effective at a willingness-to-pay threshold of AUD50,000 (USD33,062.75) per QALY was 99.5 percent. Conclusions. This is the first economic evaluation of exercise for endometrial cancer survivors. The results suggest that exercise is cost effective in this population. Given the low uncertainty in the outcomes, efforts should focus on implementation of exercise as part of clinical cancer care.
Oral Presentation Number: OP122 Abstract Title: Economic Evidence To Support Expanding Use Of Existing Positron Emission Tomography Technology As A Diagnostic Tool For High-Risk Cancer Patients Author Names: Michelle Tew ([email protected]), Abby Douglas, Jeff Szer, Ashish Bajel, Simon Harrison, Shio Yen Tio, Leon Worth, Rodney Hicks, David Ritchie, Monica Slavin, Karin Thursky, Kim Dalziel Introduction. In Australia, 18F-fluorodeoxyglucose positron emission tomography with low-dose computed tomography (FDG-PET/CT) is currently only funded for cancer staging-related indications. A recent multicenter randomized trial demonstrated that FDG-PET/CT, compared with standard of care computed tomography (CT) imaging, improved antimicrobial management and the outcomes of patients with persistent and recurrent neutropenic fever. There is potential value in expanding the use of FDG-PET/CT as a diagnostic tool for this high-risk population. We conducted an economic evaluation from a healthcare perspective alongside the randomized trial and compared FDG-PET/CT with standard CT up to 6 months after the scans. Methods. Case report forms were used to collect resource utilization data and length of hospitalization. Effectiveness was measured as the number of patients with antimicrobial rationalization and quality-adjusted life-years (QALYs) derived from patient-reported trial-based health-related quality of life. Generalized linear models (GLM) were used to analyze costs and outcomes. Incremental cost-effectiveness ratios (ICERs) for each of the outcomes were calculated and interpreted as the cost per patient with antimicrobial rationalization and cost per QALY gained. To account for sampling, we performed bootstrapping with 1,000 replications using the recycled predictions method. Results. The adjusted healthcare costs were lower in the FDG-PET/CT group (mean AUD49,563, 95% confidence interval [CI]: 36,867, 65,133; equivalent to USD34,268, 95% CI: 25,490, 45,033) compared with the standard CT group (mean AUD57,574, 95% CI: 44,837, 73,347; equivalent to USD39,807, 95% CI: 31,000, 50,712). The magnitude of differences in QALYs between the two groups was small (0.001; 95% CI: -0.001, -0.001). When simulated 1,000 times, our analysis showed that across both outcomes FDG-PET/CT was the dominant strategy as it was cheaper and had better outcomes than standard CT in 74 percent of simulations. Conclusions. FDG-PET/CT is cost effective when compared with standard CT for investigating persistent or recurrent neutropenic fever in high-risk patients. Aligning economic evaluations with clinical studies is key to an integrated evidence generation approach for supporting funding for FDG-PET/CT in this patient group.
Oral Presentation Number: OP123 Abstract Title: Lifetime Cost Effectiveness Analysis In Patients With Early Hepatocellular Carcinoma Receiving Two Competitive Treatments: Liver Transplantation Versus Hepatic Resection Author Names: Yih-Jyh Lin ([email protected]), Chia-Ni Lin, Li-Jung Elizabeth Ku, Jung-Der Wang Introduction. Both liver transplantation (LT) and hepatic resection (HR) are considered potentially curative treatments for patients with early-stage hepatocellular carcinoma (HCC). Our previous study found that patients with stage I HCC receiving LT did not survive longer than those receiving HR. We therefore studied the incremental cost- effectiveness ratio (ICER) for LT and HR. Methods. From Taiwan’s national databases we identified patients with stage I and II HCC who received LT or HR as the initial therapy and had 17 years’ follow up. After excluding severe liver cirrhosis and major comorbidities, we estimated life expectancy (LE) by extrapolating survival with an algorithm rollover, which was compared with controls simulated from national lifetables and matched for age, sex, and calendar year to obtain loss of LE. We multiplied the monthly healthcare costs with corresponding survival rates and summed them to estimate the lifetime expenditures. The difference in lifetime costs between LT and HR was divided by the difference in potential saving of loss of LEs to obtain an ICER. The results were corroborated by comparison with one-to-one and one-to-two matched cohorts. Results. The differences in loss of LE in stage I (tumor size ≤2 cm), stage I (size 2 to 5 cm), stage II (size ≤2 cm), and stage II (size 2 to 5 cm) were 0.2, 3.3, 6.2, and 4.9, respectively. The ICER for loss of LE in stage I (size ≤2 cm) was USD9,222,905, which was much higher than for other stages: stage I (size 2 to 5 cm) was USD77,476; stage II (size ≤2 cm) was USD45,085; and stage II (size 2 to 5 cm) was USD48,437. Similar trends were observed in one-to-one and one-to-two matched analyses, indicating the robustness of the analysis. Conclusions. Our results indicated that HR is more cost effective than LT in treating patients with stage I HCC, especially among those with tumors no bigger than 2 cm.
Oral Presentation Number: OP124 Abstract Title: Cost Effectiveness Of End-Stage Renal Disease Treatment Methods In Türkiye Author Names: Mustafa Kılıç ([email protected]), Vahit Yigit Introduction. Chronic kidney disease is an important public health problem and is a leading cause of morbidity and mortality worldwide. Hemodialysis (HD), peritoneal dialysis (PD), and kidney transplantation (Tx) are the main treatments for this disease. The aim of this research was to determine the cost effectiveness of treatments for end-stage renal disease from the perspective of a reimbursement institution in Türkiye. Methods. A Markov model was developed to measure costs and health outcomes in terms of quality-adjusted life-years (QALYs). The model parameters were based on a six percent discount rate, lifetime time horizon, and a reimbursement agency perspective. The main outcome measures were the incremental cost-effectiveness ratio (ICER) and the cost per QALY. One-way and probabilistic sensitivity analyses were performed to determine parameter uncertainty. Results. The lifetime costs of HD, PD, and Tx were USD26,883, USD37,672, and USD31,227, respectively. The lifetime QALYs gained with HD, PD, and Tx were 5.21, 6.77, and 9.73, respectively. The cost per QALY of HD, PD, and Tx were USD5,161, USD5,567, and USD3,211, respectively. Compared with Tx, the ICERs for HD and PD were USD961 and USD2,178, respectively. Conclusions. Cost differences have occurred between the treatment options for end-stage renal disease due to the increase in drug costs in Türkiye in recent years. As seen in the Markov model in this research, HD, PD, and Tx are complementary rather than rival treatments. This study found that the cost effectiveness of Tx is higher than HD or PD. However, the rate of Tx, which has a higher quality of life compared with HD, is around 22 percent in Türkiye; the rate for PD is four percent. It is therefore recommended that a health policy be developed to encourage kidney donation and promote PD as a superior alternative to HD for eligible patients.
Oral Presentation Number: OP125 Abstract Title: How Can Health Technology Assessment Evolve To Better Consider Benefits For Patients, Their Families, And Carers? Author Names: John Gillespie ([email protected]), Paul Davies Introduction. In Australia, technical guidelines for the health technology assessment (HTA) of medical technologies do not formally include broader societal benefits in the base case economic evaluation; they are considered supplementary analyses. If what matters to patients is relevant and valuable, then why shouldn't these broader benefits play a more important role? This presentation will consider the challenges and opportunities for HTA guidelines to change to allow this, and the broader implications for decision makers. Methods. A targeted literature review was undertaken to assess whether economic evaluation methods and their application in HTA are well positioned to assess what matters to patients. Practical challenges for this will be considered, particularly from the perspective of decision makers having a full understanding of broader societal benefits. Results. Preliminary findings from the literature review suggested that taking a broader societal perspective in economic evaluations used in HTA has the potential to enable more informed decisions for policy makers. However, there are practical considerations regarding consistent approaches to assessing broader societal and patient benefits. Conclusions. For decision makers to be fully informed on the impact of their decisions beyond healthcare budgets alone, explicit consideration of a societal perspective is necessary. However, for decisions to be equitable across different patient groups, there must be consistency in methodological approaches. Fixing this current limitation should not prevent HTA from giving what matters to patients a central role now, and refining methods on an ongoing basis.
Oral Presentation Number: OP126 Abstract Title: Clinical And Economic Evaluation Of The Effectiveness Of Cerebrolysin® In Neurological Patients With Post-Stroke Complications In Kazakhstan Author Names: Andrey Avdeyev ([email protected]), Alexander Gulyaev Introduction. Medical rehabilitation, one of the main components in the care of patients after stroke, is currently not specified in Kazakhstan, even though neurological disorders are a frequent and potentially disabling consequence of a stroke. The study aimed to evaluate the clinical and economic effectiveness of using Cerebrolysin in patients with post-stroke complications in the Republic of Kazakhstan. Methods. An annual cost per patient Markov model was developed to compare the use of Cerebrolysin with placebo in the medical rehabilitation of adult patients after acute ischemic stroke. Outcomes and costs were assessed at day 90. Secondary analysis was performed at the end of one year. The primary criterion for effectiveness was change in Action Research Arm Test (ARAT) scale - Hand Function Assessment Test scores. The modified Rankin Scale (mRS) was used as a secondary measure of effectiveness. Results. The results of the cost-effectiveness analysis showed a pharmacoeconomic advantage in using Cerebrolysin, in comparison with placebo, in the early rehabilitation of patients after stroke. Cerebrolysin resulted in a better ratio of the main cost-effectiveness ratio (CER) parameters and a negative incremental cost-effectiveness ratio (ICER), regardless of which effectiveness criterion was used. For the ARAT scale, the CER was USD63.33 versus USD148.07 and the ICER was -USD27.71; for the mRS, the CER was USD45.95 versus USD158.54 and the ICER was -USD14.93. The annual budget impact per patient of funding Cerebrolysin is expected to be an increase in the cost of purchasing the drug (an additional USD343.85) and an overall cost saving in the Cerebrolysin group due to accelerated patient rehabilitation (USD1,944.30 versus USD2,354.37). Conclusions. New evidence has emerged on the effectiveness and safety of Cerebrolysin in patients after stroke, which has served as the basis for including this drug in many international clinical recommendations. The pharmacoeconomic advantages of Cerebrolysin make it possible to recommend its use in the medical rehabilitation of patients after stroke in Kazakhstan.
Oral Presentation Number: OP127 Abstract Title: The Cost Effectiveness Of Anti-Vascular Endothelial Growth Factor Treatments For Age-Related Macular Degeneration In The Italian Healthcare Setting Author Names: Michele Basile ([email protected]), Eugenio Di Brino, Filippo Rumi, Americo Cicchetti Introduction. Age-related macular degeneration (AMD) is a common condition that affects the middle part of a patient’s vision. Typically, it first appears in people in their 50s and 60s. While it does not cause total blindness, it can make everyday activities, such as reading and recognizing faces, more difficult. This analysis aimed to define the resource absorption and cost-effectiveness profiles of the anti-vascular endothelial growth factor therapies currently available in the Italian healthcare context. Methods. A questionnaire was prepared to gather information on specific drivers involved in the provision pathway. The economic analysis was conducted according to activity-based costing methods. A cost-effectiveness analysis was carried out to provide information on the sustainability profile of the treatments available in the Italian setting. Results were reported in terms of the incremental cost-effectiveness ratio (ICER). Deterministic and probabilistic sensitivity analyses were carried out to test the robustness of the results. Results. The average absorption of resources per patient along the whole clinical pathway for aflibercept, bevacizumab, ranibizumab, and brolucizumab was EUR6,858, EUR 1,420, EUR7,930, and EUR5,667, respectively. Brolucizumab was characterized by an unacceptable cost-effectiveness profile (ICER EUR43,454) versus bevacizumab, considering a willingness-to-pay threshold of EUR40,000 per quality-adjusted life-year (QALY). Compared with ranibizumab, brolucizumab was associated with lower costs (EUR22,368 versus EUR29,333) and higher QALYs (12.8 versus 12.6). Brolucizumab had a higher level of QALYs (12.8 vs 12.7) and lower resources absorbed than aflibercept, with a saving of EUR4,222. Therefore, brolucizumab was a dominant alternative to ranibizumab and aflibercept. Conclusions. The analysis underlined how brolucizumab is a cost-saving strategy, compared with aflibercept and ranibizumab, and is likely to be cost-effective relative to bevacizumab in the Italian healthcare context.
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