AM23_OralAbstractBook_01

["Oral Presentation Number: OP128 Abstract Title: Uncertainties In The Cost-Effectiveness Analysis Of Onasemnogene Abeparvovec For Spinal Muscular Atrophy Type 1 Author Names: Stefani Borges ([email protected]), Br\u00edgida Fernandes, Fernanda Rodrigues, H\u00e9rica N\u00fabia Cirilo, B\u00e1rbara Krug, Livia Probst, Ivan Zimmermann Introduction. Nusinersen and risdiplam are available in the Brazilian Unified Health System (SUS) for the treatment of spinal muscular atrophy (SMA) type 1. Onasemnogene abeparvovec, a promising gene therapy, was approved in 2020 in Brazil. Given the high cost of this therapy and its promise of a lifetime effect, the objective of this study was to evaluate the cost effectiveness of onasemnogene abeparvovec, compared with nusinersen and risdiplam, in the treatment of SMA type 1 from the perspective of SUS in different scenarios. Methods. A Markov model was adapted from one originally developed for the USA that considers five states of health. Short-term data were obtained from pivotal clinical trials and long-term survival curves were extracted from published reports from the USA. Maintenance of motor function milestones achieved at the end of follow up in clinical trials was considered until death. Costs and quality-adjusted life-years (QALYs) were discounted at five percent per year over a baseline lifetime time horizon. Alternative scenarios were evaluated for horizons of five and ten years, with and without a discount. Results. Onasemnogene abeparvovec resulted in an incremental cost of BRL742,890 (USD297,156) per QALY and an increase of 3.32 QALYs in relation to the alternatives over a lifetime time horizon. In the same time horizon, but without the discount, onasemnogene abeparvovec resulted in an incremental cost-effectiveness ratio (ICER) of BRL166,539 (USD66,615) per QALY. In a five-year time horizon, considering the discount rate, the therapy resulted in an ICER of BRL12,527,667 (USD5,011,066); in ten years the ICER was BRL3,384,793 (USD1,353,917). Conclusions. Since the benefits of onasemnogene abeparvovec mainly occur in the long term, decision makers need to consider the uncertainty of assumptions of sustained effectiveness in view of the high initial cost of the technology.","Oral Presentation Number: OP129 Abstract Title: A Cost-Utility Analysis Of Denosumab (Prolia\u00ae) For Treating Osteoporosis In Postmenopausal Women: A Swiss Healthcare Payer Perspective Author Names: Danielle Stringer, Magdalena Ruth Moshi ([email protected]), Konstance Nicolopoulos, Mathias Jenal, Thomas Vreugdenburg, Ning Ma Introduction. Osteoporosis is characterized by decreased bone mass and density, increasing skeletal fragility, and the risk of fragility fracture. Fragility fractures are associated with a high economic burden. Denosumab (Prolia\u00ae) is a pharmacological therapy used to treat osteoporosis and reduce the risk of fragility fracture. This study aimed to assess the cost effectiveness of denosumab, compared with other pharmacological therapies (oral bisphosphonates, intravenous [IV] ibandronate, zoledronate, raloxifene, and bazedoxifene) and no treatment, for treating postmenopausal women with osteoporosis. Methods. A discrete event simulation model was developed using a lifetime time horizon. A Swiss healthcare payer perspective was adopted. Time-to-fracture distributions were derived from Swiss-specific Fracture Risk Assessment Tool (FRAX\u00ae) probabilities. Reductions in the risk of vertebral and nonvertebral fractures due to treatment were informed by a Bayesian network meta-analysis. Cost-effectiveness frontier analysis was utilized. Pairwise incremental cost-effectiveness ratios (ICERs) between denosumab and each comparator were also estimated. Sensitivity analyses were conducted to identify key drivers and explore the overall certainty of findings. Results. At a hypothetical willingness-to-pay (WTP) threshold of CHF100,000 (EUR101,630), IV ibandronate was the most cost-effective therapy in women aged 60 years who had a very high risk of fracture, and in women aged 70 or 80 years of any risk level. In women aged 60 years with a lower risk level, zoledronate was the most cost- effective option. Nevertheless, ICERs from pairwise comparisons between denosumab and some comparators (no treatment, bazedoxifene, raloxifene, and\/or zoledronate depending on the cohort\u2019s age and risk profile) were below the hypothetical WTP threshold. Higher intervention costs, smaller reductions in the risk of hip fracture, and shorter duration of residual benefit associated with denosumab contributed to the high ICER values seen in pairwise comparisons with oral bisphosphonates (as a class) and IV ibandronate. Conclusions. The present evaluation supported the cost effectiveness of denosumab against some, but not all, comparators. Nevertheless, these results should be interpreted cautiously in light of uncertainty in the true effect of treatments on fracture risk.","Oral Presentation Number: OP130 Abstract Title: Estimating The Economic Burden Of HIV\/AIDS In T\u00fcrkiye Towards The Next Decade Author Names: Yaren Erkut, Mustafa Kurnaz, Ismail Balik, Birol Tibet ([email protected]), G\u00fcvenc Kockaya Introduction. Approximately 34.8 million people globally are affected by HIV, 34,000 of whom are in T\u00fcrkiye, and the impact of HIV continues to grow. Not providing the necessary treatment eventually leads to life threatening AIDS- related consequences. In this study, a descriptive analysis of the published official data on HIV\/AIDS was undertaken to assess existing statistics and raise social awareness. The aim was to provide data that will help decision makers in future planning by forecasting the possible number of cases and treatment costs to 2030 using available statistics in T\u00fcrkiye. Methods. The forecast of HIV-infected patients, deaths, and healthcare costs to 2030 were calculated using linear regression based on data published between 1985 and 2022 in the literature or by official authorities. The proportion of past deaths caused by AIDS was used to forecast deaths, and the number of patients living with HIV was estimated indirectly using this forecast. Possible treatment expenditures related to HIV\/AIDS in T\u00fcrkiye were estimated based on published healthcare cost data and the number of living patients obtained by considering the possible increase in HIV\/AIDS cases, future inflation predictions from the Turkish Central Bank, existing literature, and information from statista.com. Results. Estimates suggested that 3,002 new cases of HIV\/AIDS in 2021 will become 5,709 among a total of 74,227 living patients in 2030. The 598 deaths reported between 1985 and 2022 are predicted to rise to 1,256 by 2030. Estimation of the total treatment cost of HIV\/AIDS was TRY1,051,026,183 (USD118,683,580) in 2021, which was estimated to reach TRY15,432,842,049 (USD296,450,696) by 2030. This consisted of outpatient visits (TRY246,710,838 [USD4,739,088]), laboratory tests (TRY2,072,489,815 [USD39,810,623]), inpatient visits (TRY1,352,192,479 [USD25,974,373]), management of complications (TRY3,371,126,960 [USD64,756,247]), and antiretroviral therapy drugs (TRY8,390,321,957 [USD161,170,365]). Conclusions. The prevalence of HIV\/AIDS is expected to rise by 137 percent by 2030, with the economic burden increasing 14.7 times in TRY (2.5 times in USD) from 2021 to 2030 in T\u00fcrkiye. This proves the severity of the situation and the need for relevant policy measures for society.","Oral Presentation Number: OP133 Abstract Title: COVIDIAGNOSTIX: Health Technology Assessment For COVID-19 Serological Tests As Companion Diagnostics To Vaccination Author Names: Rossella Tomaiuolo, Chiara Di Resta, Pietro Derrico, Matteo Ritrovato, Giuseppe Banfi ([email protected]) Introduction. In scenarios of vaccine scarcity or the context of organizational complexity, it is necessary to define prioritization strategies for allocating vaccine in compliance with the criteria of equity and efficiency of health resources. The COVIDIAGNOSTIX project, based on health technology assessment (HTA), assessed the role of SARS-CoV-2 serological tests as a companion diagnostic in the definition of strategies for vaccine administration. To guarantee evidence support for health policy choices, two different strategies were analyzed: one based on administering the vaccine booster dose to the entire population (VACCINE strategy) and the other based on allocation criteria (TEST&VACCINE strategy). Methods. An Italian multidisciplinary team conducted a decision-oriented HTA using a combination of the EUnetHTA Core Model and the multicriteria decision analysis model based on the Analytic Hierarchy Process. Moreover, the Department of HTA method was integrated with the Susceptible-Exposed-Infectious-Recovered model, appropriate modelling techniques, simulation, and quantification of uncertainty that considered the ability to reduce deaths and to contain the pandemic. After identifying the evaluation elements and the decision- making structure, the weights of the evaluation areas and key performance indicators were calculated. This is a constituent part of the mathematical model of data processing, as the Analytic Hierarchy Process was based on a structured questionnaire that compared the relative importance of the two elements on a qualitative scale (1=equal importance; 9=more important). Results. The processing of the scores attributed to the key performance indicators concerning all the evaluation domains resulted in a performance of 94 percent for the TEST&VACCINE strategy and 84 percent for the VACCINE strategy. The TEST&VACCINE strategy was the most advantageous in various scenarios due to the greater speed of response from an operational and economic point of view. Conclusions. The assessment schemes defined by COVIDIAGNOSTIX (i.e., technologies, intended use, and settings), which highlight the characteristics that differentiate the tests from each other and guarantee a timely and appropriate evaluation, can be adapted to respond to similar health policy management situations.","Oral Presentation Number: OP134 Abstract Title: Mapping The Health Of The Nation Outcomes Scale To The EQ-5D Using The Pharmacotherapy Monitoring And Outcome Survey Author Names: Anne Kleijburg ([email protected]), Ben Wijnen, Wouter Den Hollander, Silvia Evers, PHAMOUS investigators, Hans Kroon, Joran Lokkerbol Introduction. In clinical practice, health outcomes relevant for economic evaluations, such as health-related quality of life (i.e., utilities), are not always available. However, algorithms can be developed to map available outcome measures to utilities. In the Netherlands, the Health of the Nations Outcomes Scores (HoNOS) is a physician- reported, disease-specific outcome measure frequently used for patients with severe mental illness for which no mapping algorithms for utilities currently exist. The aim of this study was to develop an algorithm to map responses on the HoNOS to EQ-5D utilities for use in economic evaluations. Methods. A dataset was obtained from the Pharmacotherapy Monitoring and Outcome Survey cohort study containing data from patients with psychotic disorders. The dataset contains EQ-5D-3L domain and HoNOS scores, the age and sex of patients, and additional demographics. Correlation between the EQ-5D-3L and HoNOS was evaluated. To derive mapping functions, least absolute shrinkage and selection operator (LASSO) regression and random forest algorithms were applied with various predictor variables using a machine learning approach, whereby data were split into separate training and test sets. Cross-validation was then used to compare the performance of different models using R-squared and the root mean square error (RMSE). Results. A total of 2,111 patients were included in the study. Spearman\u2019s correlation coefficients indicated a weak to moderate negative correlation of -0.31. Based on model performance metrics, LASSO models outperformed random forest models on the training set, where the model including all individual HoNOS items and the age and sex of patients showed the best overall performance with an RMSE of 0.237 and an R-squared of 0.218. When applied to the test set, this resulted in an R-squared of 0.233 and a mean absolute error of 0.177. Conclusions. The HoNOS can be mapped onto EQ-5D-3L utilities with moderate predictive accuracy. The reported mapping algorithm may be sufficient to predict overall population utility scores for use in health economic evaluations but lacks accuracy for individual patient predictions.","Oral Presentation Number: OP135 Abstract Title: Machine Learning And Cancer Registry: Evaluation Of The Effectiveness Of Case Coding Author Names: Carmelo Ettore Viscosi, Alessia Anna Di Prima, Antonina Torrisi, Antonietta Alfia Torrisi, Margherita Ferrante, Rosalia Ragusa ([email protected]) Introduction. Machine learning (ML) algorithms are computational procedures that use pattern recognition and inference by learning from previously categorized documents to predict the category to which a new document belongs. The role of machine learning within cancer registries remains unclear given the lack of in-depth testing and guidance from health technology assessment (HTA) agencies. We evaluated the effectiveness of coding new cases through machine learning at the Integrated Cancer Registry. Methods. The Integrated Cancer Registry covers the eastern area of Sicily in Italy, which has an annual average incidence of about 10,000 cases of malignant neoplasm. Potential new cancer cases were retrieved from pathology services and processed by pathologists who confirmed the neoplastic nature of supposed cases and specified the morphological type and location of the tumors. The current method involves identification by reading the free-text report when International Classification Diseases for Oncology information was not provided. We used the new Microsoft ML.Net Library, a framework developed in response to the challenge of facilitating machine learning pipeline utilization in large software applications. A total of 1,050,952 free-text pathology reports published from 2003 to 2018 were selected separately from all Sicilian pathology services and uploaded to machine learning software that explored eight binary classification algorithms. Results. We evaluated each algorithm\u2019s performance by calculating metrics (the number of true positives, true negatives, false positives, and false negatives) from the classification procedure applied to the test dataset. The metrics used were accuracy, F1 score, and area under the receiver operating characteristic curve. With a test set of around 210,000 text diagnoses, each algorithm reached an F1 score of up to 95 percent. Conclusions. Machine learning algorithms capture relevant information about tumors from free-text pathology reports, optimizing the process and reducing waste. With the help of machine learning systems, cancer registries can provide more timely data for research and evaluation of all types of new cancer technologies (drugs, devices, radiology and radiotherapy equipment, diagnostic devices, robotic surgery, and vaccines).","Oral Presentation Number: OP136 Abstract Title: A Comparison Of Health Technology Assessment Recommendations In Australia, Canada, And England: Is There Opportunity For Further Alignment? Author Names: Tina Wang ([email protected]), Belen Solar, Neil McAuslane Introduction. Over the past decade there have been increasing interactions between health technology assessment (HTA) agencies through international networks at the policy level and European joint actions at the product level. A pilot project is underway to explore collaboration beyond Europe between HTA agencies in Australia, Canada, and the UK. This study the compared HTA recommendations of new active substances (NAS) appraised by Australia\u2019s Pharmaceutical Benefits Advisory Committee (PBAC), Canada\u2019s CADTH, and England\u2019s National Institute for Health and Care Excellence (NICE). Methods. Using publicly available data and established benchmarking methodology, we examined 45 NAS appraised by PBAC, CADTH, and NICE between 2017 and 2021. Analysis was performed to assess rollout time from regulatory to HTA recommendation, and to the first HTA recommendation. Results. Most products were submitted to the Europe Medicine Agency first (89%). However, 71 percent of NAS in Australia and 69 percent in Canada were submitted to HTA in parallel with regulatory review, which shortened overall rollout time. The median HTA submission gap among the three agencies was 140 days in Australia, 102 days in Canada, and 8 days in England. PBAC had the highest number of negative recommendations (51%), followed by CADTH (18%), and NICE (5%). The congruence of HTA decisions was highest between CADTH and NICE (56%), compared with PBAC and CADTH (11%), and PBAC and NICE (20%) Conclusions. To achieve a more collaborative HTA process it is necessary to understand the rollout time in these jurisdictions. This study identified submission gaps among the regulatory agencies, but there may be more synergy in the future as regulators in the three jurisdictions work collaboratively through the Access Consortium. Currently, the submission gap for the three HTA agencies was mostly within six months, making collaboration on joint assessment possible. We observed divergences in HTA recommendations due to the methodology and decision criteria applied by each agency. Therefore, collaboration on assessment should build on the clinical aspects, although HTA decisions should be grounded in the local context.","Oral Presentation Number: OP137 Abstract Title: A Qualitative Exploration Of The National Institute For Health And Care Excellence's Impact On International Health Technology Assessment Author Names: Nadine Henderson ([email protected]), Simon Brassel, Edward Oliver, Ilana Gibbons, Rachel Allen, Nathalie Largeron, Martina Garau Introduction. The National Institute For Health And Care Excellence (NICE) is widely acknowledged as a seminal health technology assessment (HTA) body, known for its transparent and accountable approach to decision-making. This research aimed to investigate the impact of NICE methodology and decisions on international HTA bodies. We sought to identify direct and indirect factors that may influence an international HTA body's methods or outcomes. To the best of our knowledge, this is the first research to use a qualitative approach to understand the influence of NICE on other HTA bodies. Methods. We conducted 13 semi-structured qualitative interviews with HTA and market access experts from industry and academia from nine countries (Brazil, Israel, Italy, Japan, Poland, Saudi Arabia, South Korea, Sweden, and the United Arab Emirates). The interview script was organized into three main sections: comparing NICE methods and processes with other HTA bodies; the impact of specific NICE decisions; and Likert scale questions (to allow for comparability of opinions). Results. Most interviewees believed their local HTA body would consider NICE's decision when evaluating a medicine. However, the way and extent to which NICE influences HTA varied across countries. The most common means of considering a NICE decision was as background information or context for an HTA evaluation. Generally, interviewees suggested that negative NICE decisions had more impact on local decision-making than positive decisions. Nine of the 13 interviewees agreed or strongly agreed that their country\u2019s HTA body considers the decisions of other HTA bodies in their decision-making process. Eleven of the 13 interviewees agreed or strongly agreed that the development of their country\u2019s HTA body methods and processes was influenced by NICE. Conclusions. NICE is perceived to be a seminal HTA body, with continued influence on HTA agencies in other countries. However, the mechanisms and extent of this influence varies considerably between countries. We suggest that implicit factors are likely to contribute more to NICE's influence than individual decisions. Nevertheless, further research is needed to reveal these factors and increase efficiency in international HTA decision-making processes.","Oral Presentation Number: OP138 Abstract Title: Navigating High-Cost Medicines: Promoting Consistent, Evidence-based Use Of High-Cost Medicines In A Fiscally And Equitable Responsible Manner Author Names: Lisa Pulver ([email protected]), Peter Barclay, Dr Sasha Bennett, Naomi Burgess, Jonathan Dartnell, Catherine Drake, Tracey-Lea Laba, David Liew, Kylie Mason, Terry Melocco, Mary O\u2019Reilly, Linda Sheahan, Kavitha Subramaniam, Catherine Hill Introduction. Hospitals play a significant and important role in funding high-cost medicines so patients can access treatments they need. High-cost medicines are often specialty medicines, which contribute to a significant and increasing portion of the hospital budget. It is imperative that these expensive medicines are governed and managed with a fair, standardized evidence-based process. We aim to provide a framework for Drugs and Therapeutics Committees (DTCs). Methods. During 2021, Guiding Principles were developed following a literature review and survey of current practices by DTCs in Australia. An Expert Advisory Group (EAG) was convened, comprising individuals with expertise in quality use of medicines, evidence-based medicine and medicines governance. The guiding principles were drafted by the EAG, in consultation with a range of stakeholders and relevant external organizations. All feedback was collated, reviewed and discussed to refine the content of the final Guiding Principles released in January 2022. Results. Seven overarching principles provide key recommendations for the governance of high-cost medicines: (i) A definition of high\u2011cost medicines should be determined and clearly articulated for use by each medicines governance committee. (ii) Review of high-cost medicines requires members with relevant expertize to facilitate good and effective decision-making. (iii) The committee should engage directly with the applicant prior to review to ensure a full understanding of the rationale for the request. (iv) A consistent, robust and transparent procedure for the assessment of high-cost medicine applications should be defined and implemented for use by each medicines governance committee to ensure fair process. (v Ethical considerations fundamentally underpin deliberations around high-cost medicines. (vi) The decisions and outcomes of the decision making should be transparent and appropriately communicated to the various audiences. (vii) The high-quality assessment of high-cost medicines requires appropriate training and resourcing. Conclusions.","These national Guiding Principles promote consistent, evidence-based use of high-cost medicines and provide a framework for DTCs to assess and achieve effective governance for the quality use of high\u2011cost medicines.","Oral Presentation Number: OP141 Abstract Title: Expert Knowledge Elicitation in Health Technology Assessment: Our Experience Using the Sheffield Elicitation Framework Author Names: Danielle Stringer ([email protected]), Ning Ma, David Tivey Introduction. Expert judgement has an important role in health technology assessment (HTA), including as a source of evidence to inform economic modeling when published data are lacking. Quantitative information may be elicited from experts to inform model inputs and associated uncertainty using one of many expert elicitation methodologies. Here, the feasibility and potential benefits of one expert elicitation method, the Sheffield Elicitation Framework (SHELF), to the HTA process is examined. Methods. The SHELF method seeks to express the knowledge of multiple experts in the form of a subjective probability distribution. Eliciting a subjective probability distribution allows the uncertainty of experts to be included in probabilistic sensitivity analysis, which is becoming an increasingly prominent feature of HTAs. The individual knowledge of participating experts is combined through behavioral aggregation, where experts participate in a discussion before being asked to provide judgments from the perspective of a rational impartial observer. The whole process is led by a facilitator who ensures all participants contribute and confirm that the final distribution is a product of consensus, not compromise. Results. We recently conducted two SHELF elicitations as part of an ongoing project aiming to streamline the assessment of positron emission tomography (PET) in Australia. These elicitations provided insight into the usefulness of SHELF within the HTA setting. Given the constraints imposed by the COVID-19 pandemic, the elicitation sessions were conducted online rather than in the ideal face-to-face manner. In collaboration with one of the developers, we successfully adapted the method by making use of video conferencing technology to provide an online environment that mimicked the face-to-face setup as much as possible. Conclusions. SHELF provides a rigorous and scientific method by which to elicit the knowledge of multiple experts in the form of a probability distribution. However, the method is resource intensive and may be best reserved for when data on key drivers are lacking.","Oral Presentation Number: OP142 Abstract Title: Progression Analysis Versus Traditional Methods To Quantify Slowing Of Disease Progression In Alzheimer\u2019s Disease Author Names: Linus J\u00f6nsson, Milana Ivkovic ([email protected]), Ron Handels, Anders Gustavsson, Teresa Le\u00f3n, Julie Hviid Hahn-Pedersen, Lars Lau Raket Introduction. New statistical methodology, known as progression models for repeated measures (PMRM), can estimate the slowing of progression (percentage slowing or time delay) of Alzheimer\u2019s disease from trial data on disease- modifying therapies. We compared the PMRM methodology with mixed models for repeated measures (MMRM) and Cox time-to-event analysis on simulated trial data with respect to their power and interpretability of estimates. Methods. Two novel models were included: PMRM (estimating slowing of progression and allowing different rates across visits) and proportional-slowing PMRM. Clinical Dementia Rating (CDR) Sum of Boxes score and progression to dementia as assessed by CDR global score were the primary outcomes for MMRM\/PMRM and the Cox model, respectively. Subject-level placebo arm trajectories were jointly simulated based on estimated CDR mean trajectories and joint temporal correlation structure of 538 amyloid-positive patients with mild cognitive impairment who met typical disease-modifying trial inclusion criteria from the Alzheimer\u2019s Disease Neuroimaging Initiative study. Active arm trajectories were simulated to show an average 20 percent slowing of disease progression, compared with placebo, at each visit. We conducted 1,000 simulations across multiple scenarios, varying the number of patients per arm (200 to 700) and clinical trial duration (18 to 36 months). Results. The power of PMRM models was greater than that of MMRM, and much greater than that of the Cox model whose power never exceeded 45 percent. PMRM models accurately estimated the underlying treatment effect (median 20% slowed progression, which translated to a delay in progression of 5 and 7 months at trial durations of 24 and 36 months, respectively), unlike quantifications of the MMRM (median estimated 25% reduction in decline), and the Cox model (median estimated hazard ratio of 0.9). Conclusions. For disease-modifying therapies, PMRM estimates may have a more intuitive clinical interpretation in terms of delayed progression than MMRM or Cox models and enable a description of the amount of time spent in less severe disease stages. Among all the methods studied, PMRM offered the best combination of interpretability and power.","Oral Presentation Number: OP143 Abstract Title: Association Of Different Venous Access Device With Health\u2011Related Quality Of Life Among Patients With Breast Cancer In China Author Names: Liu Liu ([email protected]), Zhiyuan Xia, Yingyao Chen, Shimeng Liu Introduction. Few studies have explored the relationship between different venous access devices and health-related quality of life (HRQoL) among patients with breast cancer in China. This study aimed to evaluate the HRQoL of patients with breast cancer in China who underwent different venous access devices and to estimate the association between type of venous access device and HRQoL. Methods. A multicenter cross-sectional study was conducted in three tertiary hospitals from three major geographical regions in China. The final sample consisted of 472 patients aged from 18 to 78 years. The HRQoL was measured with the EQ-5D-5L scale and the EuroQoL Group visual analog scale. The venous access devices were divided into totally implantable venous access devices (TIVAD), peripherally inserted central catheters (PICC), and other devices. The multivariate regression analyses were used to explore the association between type of venous access device and HRQoL. Results. Of 472 participants, 352 (75%) used the TIVAD device, 89 (19%) used the PICC device, and 31 (7%) used other devices. The TIVAD group had the highest EQ-5D-5L values (mean 0.89, standard deviation [SD] 0.178), while the PICC group had the lowest values (mean 0.85, SD 0.239). The EQ-5D-5L values for the other venous access device group was in between (mean 0.88, SD 0.127). However, the multivariate analysis indicated that the VAS and EQ-5D-5L scale dimension scores among patients were not significantly different (P>0.05) for the various central venous access devices. Conclusions. This study demonstrates a non-significant association between the type of venous access device used and the HRQoL of patients with breast cancer in China. Although patients with a TIVAD experienced more pain during device insertion and access for chemotherapy, the negative effect on HRQoL scores was smaller than for PICCs.","Oral Presentation Number: OP144 Abstract Title: Impact Of A Training Program For The General Population On Knowledge Of Aortic Valve Stenosis Author Names: Carla Fern\u00e1ndez-Barcel\u00f3 ([email protected]), B\u00e0rbara Vidal, Ismail Abbas, Paloma Gonzalez, Marc Trilla, Marta Sitges Carre\u00f1o, Laura Sampietro-Colom Introduction. Limited knowledge of the symptomatology of aortic stenosis (AS) among the general population may delay diagnosis and have a major impact on morbidity and resource use. Training programs have often been advocated by the scientific community. The present study reported the results of an assessment of a training program for the general population. Methods. Patients who attended healthcare centers were asked to answer a questionnaire on their level of knowledge around AS. A cohort of patients without training (n=681) answered the questionnaire and a second cohort answered the questionnaire via phone 24 hours after training (n=197). Propensity score matching by sex and age was used to obtain a balanced sample between the two cohorts, giving a total study sample of 394 individuals (197 without training and 197 with training). A descriptive analysis was performed to compare differences in the level of knowledge between the two cohorts. Predictors of AS symptomatology were identified using multivariate logistic regression. Results. The trained cohort was more aware of AS disease than the untrained cohort (79% versus 31%, 95% confidence interval [CI]: 0.39, 0.56; p<0.001). They were also better at distinguishing the symptoms associated with AS (80% versus 43%, 95% CI: 0.28, 0.48, p<0.001) and were more aware of its severity (36% versus 12%; 95% CI: 0.16, 0.32, p<0.001). Moreover, the trained cohort were better at identifying symptoms that should make them consider visiting a doctor (76% versus 65%; 95% CI 0.02, 0.20, p<0.02). No differences were observed in level of concern regarding AS (8% versus 4%; 95% CI: -0.0046, 0.09, p=0.08). The trained people who were aware of AS (p=0.04) correctly classified AS as a valvular disease (p=0.025), would seek medical consultation when AS symptoms occurred (p=0.04), and were more likely to correctly detect AS symptoms. Conclusions. The training program significantly improved the knowledge and awareness of AS in the general population. This can improve the timeliness of AS diagnosis, reducing the health and economic burden of AS for the healthcare system.","Oral Presentation Number: OP147 Abstract Title: Measuring Health Technology Assessment Impact On The Introduction Of Transcatheter Aortic Valve Replacement In A Private Healthcare System Author Names: Silvana Kelles ([email protected]), Camila Pereira, Carina Martins, Daniel Reis, Ernesto Azevedo, Geraldo Ribeiro, Karina Zocrato, L\u00e9lia Carvalho, Marcela Freitas, Maria Horta, Mariana Barbosa, Mariza Talim, Marcus Borin Introduction. Aortic stenosis is an insidious disease that has a high mortality rate when it becomes symptomatic. Surgical valve replacement is the treatment of choice and has predictable risks. Transcatheter aortic valve replacement (TAVR) is a less invasive alternative to surgery, which is indicated for high-risk patients. Complications after TAVR include paravalvular leak, cerebrovascular events, and the need for pacemaker implantation. A health technology assessment report carried out by the Health Technology Assessment Unimed-BH group in 2018, two years before it became part of the National Supplementary Health Agency, recommended the introduction of TAVR with the following criteria: indications provided by a group of specialists; forwarding of a report with detailed clinical data; results of imaging exams; and follow-up results for up to one year after the procedure. After the introduction of TAVR with the agreed criteria, it was possible to access TAVR results from the private healthcare system of Unimed-BH. Methods. Administrative data were collected from the Unimed-BH database. All patients who received a TAVR implant from 2013 to 2017 were included by virtue of a court injunction, and after 2018 by operator concession and within agreed criteria. Results. From July 2013 to June 2019, 83 patients underwent TAVR implantation by Unimed BH. The median age of patients was 83.4 years (interquartile range 66.5 to 97.9), most of whom were women (56%). There was a predominance of patients in New York Heart Association classification III (50%) and IV (29%). There were 36 patients who underwent TAVR before 2018 and 47 patients within the agreed criteria. In the period prior to the agreed criteria, 28 percent needed a pacemaker, compared with 23 percent after 2018. During the follow- up period, 39 patients died: 18 (50%) before 2018 and 11 (23%) after 2018. Conclusions. The agreement made with the providers, which included the obligation of having a team of specialists responsible for the indication and access to clinical data through the report, improved patient outcomes. This may be due to having a better indication for the procedure or to the greater experience of the professionals involved in its delivery.","Oral Presentation Number: OP148 Abstract Title: Influence Of The Hospital-Based Health Technology Assessment Unit On New Technologies Transfer At The National Level In Kazakhstan Author Names: Andrey Avdeyev ([email protected]), Aigul Saduakassova, Indira Tleulessova, Ruslan Akhmedullin, Ekaterina Lyugay, Ma\u0445im Fet, Rustam Albayev, Valeriy Benberin, Nasrulla Shanazarov, Makhabbat Okesh, Tansolpan Aimanova, Makpal Akhmetova, Gulzada Bariyeva, Olzhas Turar Introduction. The hospital-based Health Technology Assessment (HB-HTA) Unit in the Hospital of the President\u2019s Affairs Administration has been operating since 2015 and is the first example of the implementation of the HB-HTA system in Kazakhstani hospitals. In addition to assessing the feasibility of implementing new health technologies (HTs) into the hospital\u2019s practice, the Unit also interacts with the National Regulatory Authority (NRA) to transfer new technologies into the National Reimbursement System (NRS). We report on the transformation of the HB-HTA Unit from a stand-alone entity to an integrated, specialized agency. Methods. Data were drawn from reports of the HB-HTA Unit and internal NRA documents. Inclusion of new HTs into the NRS consists of the following sequential stages: (i) implementing the technology in at least one hospital; (ii) filing an application with the Ministry of Health Care (MoH) in the field of HTA to resolve the issue of reimbursement at the national level; (iii) in case of a positive decision, approval at the national level of the clinical protocol for using the medical intervention; (iv) agreement on the reimbursement price of the technology. Results. Based on positive recommendations from the HB-HTA report in 2015, the hospital implemented 19 new nuclear medicine technologies. In 2016, the hospital initiated an application to the MoH to include these technologies in the NRS (previously, these technologies were carried out only for a fee or with private insurance). From 2016 to 2020, a positive decision from the MoH was received, protocols for medical interventions were published at the national level, and cost estimates were formulated. In 2021, 19 new medical services in nuclear imaging and scintigraphy were included in the NRS. Conclusions. Despite the long and bureaucratic process of including new HTs in the NRS, the HB-HTA Unit managed to speed up this process. One of the main priorities of the integrated HB-HTA Unit is to promote the transfer of HTs into the health system at the national level.","Oral Presentation Number: OP149 Abstract Title: How To Improve The Impact Of Health Technology Assessment: Stakeholders\u2019 Perspectives In Spain Author Names: Yolanda Tri\u00f1anes ([email protected]), Patricia G\u00f3mez, Mar\u00eda J Faraldo-Vall\u00e9s, M\u00f3nica P\u00e9rez-R\u00edos, Alberto Ruano-Ravi\u00f1a, Leonor Varela lema Introduction. Health technology assessment (HTA) agencies in Spain have an important role in informing decisions about the introduction and use of health technologies in the Spanish National Health System. However, although different approaches have been taken to measure and improve their impact, no study to date has explored the perceived impact of HTA products at the national level. The aim of this study was to explore the perspectives of macro-, meso-, and micro-level decision makers on how to improve the impact of HTA. Methods. Three online focus groups were conducted with policy makers, healthcare managers, clinicians, and patients. The transcripts were evaluated using a deductive thematic analysis based on a multidimensional framework to explore mechanisms of impact. Results. Four key themes were identified: (i) Timeliness and use of HTA assessments: Although the quality of the reports was recognized, the time taken for the elaboration and extension of reports negatively affected their use. Participants considered that reports should be tailored to the needs of end users (e.g., briefer versions available for meso- and micro-level use); (ii) Effective engagement and external communications: The engagement of multiple stakeholders (policy makers, manufacturers, clinicians, and patients) in the elaboration process was considered crucial to improve HTA impact and ensure adequate communication of results; (iii) Good institutional reputation and fit within the healthcare and policy making system: Stakeholders agreed on the need to strengthen collaboration at the national level and increase public understanding of the value of HTA and its use in healthcare decision-making; and (iv) Effective implementation of policy change regarding health technologies: Stakeholders were very receptive to the results and recommendations of HTA reports when new technologies are demanded, but the identification and selection process should be improved to guarantee that these reports are available on time. Conclusions. This study has identified different proposals and mechanisms that could improve the impact of HTA in Spain.","Oral Presentation Number: OP150 Abstract Title: An Inventory Of Policy Levers For Influencing Appropriate Care Author Names: Lindsey Warkentin ([email protected]), Lisa Tjosvold, Ken Bond Introduction. Healthcare reform through appropriate care is a current focus for many jurisdictions. A variety of policy options, or \u201clevers,\u201d are available to decision makers to influence appropriate care. However, these levers are not always identified in advance of developing policy recommendations, and few direct, empirical analyses are available to support their selection. An appropriate care policy lever inventory was developed for health technology assessment (HTA) users in Alberta, Canada, to support HTA scoping and policy development. Methods. Relevant information was identified by a single reviewer through a scoping search of MEDLINE, forward and backward searching, and targeted gray literature searches. An Excel-based inventory was populated with a list of policy levers and their descriptions, policy effectiveness, and implementation considerations. Filters were developed to identify levers based on key characteristics. The inventory was iteratively refined through presentations to and feedback from key user groups. Results. The inventory contained 53 policy levers aiming to influence service provision, clinician behavior, fiscal policies, populations or organizations, and patient behavior. The levers varied in how they restrict decision- making. Few levers were considered high impact (>5% change to behavior, utilization, or cost) or well- supported (>10 studies reporting effectiveness). Stakeholders found the inventory information useful, particularly for considering potential levers not frequently utilized within their respective programs. A user guide and case examples were also developed to help users learn to navigate the inventory. Conclusions. An inventory of policy levers, which can be tailored to specific clinical areas and topics, can be of assistance to healthcare decision makers developing and utilizing HTAs to improve appropriateness of care. With limited indication-specific evidence, policy makers must utilize the broader evidence base on appropriate care policy levers to select and implement strategies that are applicable and transferable to their context. Challenges remain in systematically identifying all relevant literature given the inventory\u2019s breadth, and in updating the inventory to reflect new evidence.","Oral Presentation Number: OP151 Abstract Title: Health Technology Assessment In Switzerland - Current And Future Challenges Author Names: Goedele van Haasteren ([email protected]), Mark Finlayson Introduction. The Swiss Health Technology Assessment (HTA) program is a unique, innovative program that connects research with policy making. In 2017, a HTA unit was established within the Federal Office of Public Health following a decision by the Federal Council in 2015 to intensify efforts in HTA. Methods. The legal basis of the HTA program is Article 32 of the Federal Health Insurance Act, which specifies that health technologies (i.e., all preventive, diagnostic, and therapeutic interventions in health care) covered by the compulsory health insurance must be effective (E), appropriate (A), and economically efficient (E). Health technologies that do not meet the EAE criteria are not eligible for coverage. For health technologies that are already reimbursed, re-evaluation of the criteria can result in the removal of technologies from the catalog of benefits or limitations being placed on their reimbursement. Results. The initial focus of the HTA program was the re-evaluation of controversial health technologies. This focus was later expanded to evaluating new and upcoming technologies through horizon scanning. Challenges encountered since the start of the program include: \u2022 aligning the classic HTA domains with the EAE criteria; \u2022 identifying suitable re-evaluation topics; \u2022 tailoring HTA processes to regulation options and decision-making processes; and \u2022 involving stakeholders in the HTA process without jeopardizing the quality and objectivity of the research. Conclusions. Despite various initial challenges, the HTA program has become an acknowledged and appreciated actor within the Swiss reimbursement policy landscape. An outlook on the program\u2019s future will also be shared.","Oral Presentation Number: OP152 Abstract Title: Use of Real- world Evidence By The Brazilian Health Technology Assessment Committee (Conitec) For Monitoring Of Health Technologies Author Names: Amanda Oliveira Lyrio ([email protected]), Tacila Pires Mega, Ana Carolina de Freitas Lopes, Felipe Ferr\u00e9, Ant\u00f4nio Marcos Santana Barreira, Clarice Moreira Portugal, Samara Helena de Carvalho, La\u00eds Lessa Neiva Pantuzza, Luciene Fontes Schluckebier Bonan, Vania Cristina Canuto Santos Introduction. In Brazil, the incorporation or disinvestment of health technologies into the Unified Health System (SUS) are advised by the National Committee for Health Technology Incorporation (Conitec). Despite the thorough evaluation carried out by Conitec, the results measured after implementation do not always reflect the economic and clinical impact expected from the incorporation. Thus, real-world evidence (RWE) is essential for monitoring health technologies. The aim of this study was to report how Brazil is using the RWE to obtain additional information about the incorporated technologies. Methods. Actions related to the use of RWE for monitoring of technologies incorporated into the SUS were described. The period evaluated was between 2012 and 2022. Results. The first Conitec recommendation in which the use of real-life data in the decision-making process was evidenced occurred in 2016. Administrative data from a cohort of patients identified that beta-interferons for Multiple Sclerosis were less effective than the other drugs used in the Brazilian public system. A further eight reports have been published assessing the performance of technologies using administrative data. Another strategy for RWE generation was through the funding of primary studies, highlighting a study with 21 rare diseases and another one to evaluate Zolgensma gene therapy, acquired through court for Spinal Muscular Atrophy. Both studies are ongoing and aim to evaluate the effectiveness, safety, adherence, and cost of medications in the evaluated diseases. Conitec is also following studies in RWE financed by pharmaceutical companies to evaluate effectiveness for incorporated technologies subject to reassessment. Additionally, managed access arrangements have been promoted for generating RWE when there is uncertainty about outcomes. Conclusions. Real-world evidence from administrative data and clinical research allows monitoring after the implementation of technologies in the Unified Health System in Brazil. This makes it possible to reallocate resources in health and contribute for the system sustainability, in addition to generating data that reduce the uncertainties assumed at the time of incorporation.","Oral Presentation Number: OP153 Abstract Title: Access To Real-World Data For Use In Health Technology Assessment \u2013 Still Work To Be Done Author Names: Christian Ritz, Hansoo Kim ([email protected]) Introduction Real-world data (RWD) is an important source of evidence for health technology assessment (HTA). It is widely used to fill clinical trial data gaps and to inform risk-sharing agreements. HTA is mandatory in many jurisdictions as it is used for price negotiation between a manufacturer and a payer. HTA practitioners have so far had limited involvement in the debate surrounding access to RWD as regulators have primarily focused on scientific research and market authorization. This study examined the challenges of obtaining RWD for HTA decision-making that is beneficial at the population level when data sources are restricted to maintain the data integrity and rights of the public. Methods Types of RWD and processes for obtaining data were assessed for two jurisdictions (Australia and Denmark). Types of data considered were national registries, ongoing or completed cohorts, surveys at various universities, archived historical data, and medical claims data. The assessment was performed by analyzing a series of cases. Results There were similarities and differences between the two jurisdictions. In both jurisdictions the process for obtaining data included an ethics application as well as data handling fees. Patients and clinicians had little to no say in what their data are used for. It can take up to six months to obtain data. Person identification numbers enable linking of different datasets. Population wide data are accessible in Denmark only through secure servers, whereas full data sets, such as prescription data, can be released for research in Australia. Public hospital data, such as electronic health records, are not easily obtained in Denmark. In Australia, public hospitals are run by individual states and, therefore, additional effort is required to access nationwide data. Conclusions Access to RWD for HTA is challenging in both Australia and Denmark. Improvements in the process of applying for data and linking different data sources for HTA purposes are still needed.","Oral Presentation Number: OP154 Abstract Title: Horses For Courses: Developing A Proportionate Approach To Health Technology Assessment In England Author Names: Ian Watson ([email protected]), Jenna Dilkes, Geoff Ellison-Roberts, Jenniffer Prescott Introduction. The number and range of technologies that the National Institute for Health and Care Excellence (NICE) evaluates has never been greater, and with that comes an increasing requirement for capacity. However, not all technologies need the full intensity of the current standard evaluation process. This presents an opportunity to differentiate evaluation processes, and in doing so release capacity for more evaluations. NICE has embarked on a project to develop proportionate appraisal processes, allowing promising medicines to move through refined processes that better match the specific needs of individual evaluations. Methods. The proportionate approach to technology appraisals (PATT) project was initiated in June 2022. Multiple work strands were established to develop and test potential proportionate approaches, focusing mainly on streamlined approaches for appraisals in which a lighter-touch approach is sufficient. By creating an accountability framework which empowered staff across NICE, new processes were developed using test-and- learn principles: piloting key concepts using retrospective reviews and live appraisals and adjusting the approach based on their findings. The impacts of each approach on NICE, on stakeholders and on the individual appraisals were monitored and assessed. Results. The project has identified a range of procedural, methodological and operational improvements across the NICE technology appraisal process. Substantial efficiencies have been found, including consolidation of activities, reducing duplication of effort and minimizing disproportionate steps. The test-and-learn approach has proved successful, both in rapidly identifying unsuccessful ideas and in refining and adjusting processes in light of new information and challenges. Conclusions. The proportionate approach to technology appraisals project represents an important part of improving and streamlining NICE\u2019s approach to health technology assessment, reflecting the increasing demands on the program. Developing proportionate approaches allows efficient use of limited evaluation resources to continue supporting rapid, evidence-based access to innovative technologies. The project also provides a valuable demonstration of an agile, flexible approach to process improvement, paving the way to rapid, dynamic updates in the future.","Oral Presentation Number: OP156 Abstract Title: Systematic Review And Meta-Analysis Of The Perioperative Association Of Gabapentinoids With Sedation And Respiratory Depression After Abdominal Surgery Author Names: Laure Taher Mansour, Warren Seow, Nathan Procter, Lucy Alexander, Lettie Pule, Joanna Duncan, Lora Papa, Charlene Singh, Guy Maddern ([email protected]) Introduction. With the increasing popularity of enhanced recovery protocols and the growing opioid epidemic, recent pain management pathways have emphasized opioid-sparing measures. As a result, gabapentinoids are being used following surgery and have become one of the most common opioid-sparing analgesics prescribed. However, they are not without risk, with several cases of respiratory depression and oversedation being reported. Methods. This systematic review and meta-analysis aimed to evaluate the impact of gabapentinoids on sedative complications following abdominal surgery in order to guide future clinical decisions. The Pubmed and Embase databases were searched according to PRISMA guidelines to identify randomized controlled trials comparing gabapentinoids with placebo following abdominal surgery with respect to sedation complications. The Cochrane Risk of Bias Tool was used to assess study quality. A comparative meta-analysis was performed on the data. Results. Of the 3,988 studies retrieved, 19 were eligible for meta-analysis. Eleven of the 19 studies assessed pregabalin (100 to 1,200 mg) and eight assessed gabapentin (300 to 1,200 mg). Postoperative sedation scores were higher in the gabapentinoid group (p<0.01) relative to placebo. Subgroup analyses demonstrated higher scores two hours after surgery for gabapentinoids (p=0.03), but no statistical difference at 24 hours (p=0.19). Different doses did not yield any differences on forest plot analyses. Respiratory depression rates were higher in the gabapentinoid group, compared with placebo (p=0.02). Conclusions. The preoperative use of gabapentinoids is associated with sedative complications, including respiratory depression. These results may help guide future perioperative pain protocols.","Oral Presentation Number: OP157 Abstract Title: Evaluating The Clinical And Economic Impact Of Adopting A Closed Peripheral Intravenous Catheter System In A Japanese Hospital Author Names: Kristin Hui Xian Tan ([email protected]), Takeshi Tomaru, Smeet Gala, Yan Ma Introduction. Up to 90 percent of inpatients require an intravenous catheter during their hospitalization. A closed, integrated peripheral intravenous catheter (PIVC) system has been shown to protect veins for longer and reduce the risk of complications and unnecessary restarts when compared with an open system. This study evaluated the annual clinical and economic outcomes of adopting a closed, integrated PIVC system, instead of an open system, for inpatients in a Japanese hospital. Methods. A budget impact analysis was developed to estimate the clinical and economic impact for a 500-bed hospital with an 85 percent occupancy rate and a 96-hour catheter replacement protocol. For the analysis, the average length of stay for patients was 12 days and 90 percent of inpatients required a PIVC. Inputs such as catheter failure rate, complication rate, consumables costs, and complication management costs were informed by global and local data sources. The outcomes evaluated included consumables utilization, complication events, nurse time, and overall cost impact. Results. The analysis estimated that 12,604 patients required PIVCs. Moving from an open to a closed, integrated PIVC system resulted in a 68 percent reduction in consumables (3,786 fewer catheters and 36,315 fewer connecting accessories). Complications (occlusion, extravasation, phlebitis, and bending) were reduced by 62 percent (3,682 fewer episodes). Blood exposure was reduced by 98 percent (3,565 fewer episodes), and nurse time decreased by 17 percent (786 fewer hours). This resulted in a potential overall cost saving of JPY3,955,140 (USD28,756) annually, after offsetting the acquisition cost of JPY888,247 (USD6,458) associated with the closed system. Conclusions. PIVC is the most common vascular access device used in hospitals, and insertion and maintenance are often performed by nurses. Fewer complications can be expected with a closed system, leading to better patient outcomes. In addition, nurses spend less time managing complications and replacing PIVCs, and consumables utilization is reduced. This results in improved operational efficiency and potential cost savings for hospitals.","Oral Presentation Number: OP158 Abstract Title: Point Of Care Testing In Primary Care: How To Add Value To Economic Evidence To Argue For Adoption Author Names: Rachael Hunter ([email protected]), Wessam Abass Introduction. Point of care tests (POCTs) provide rapid diagnostic results, potentially providing patient centric services in more convenient locations. POCTs have had increased attention as part of their role in testing for severe acute respiratory syndrome\u2013related coronavirus (SARS-CoV-2). Beyond SARS-CoV-2 there is an increasing range of POCTs available, with many fitting well into primary care as they allow clinicians to provide a faster diagnosis to patients, prescribe more appropriate treatment, improve referrals and allow for informed monitoring of conditions. Even though there is increasing evidence for the effectiveness and cost-effectiveness of POCTs in primary care their uptake continues to be slow. Methods. The health technology assessment (HTA) framework utilized by most countries evaluates if a new technology should be implemented based on the incremental cost per quality adjusted life year (QALY) gained in relation to a cost-effectiveness threshold from a national perspective. This framework works well for new technologies with clear clinical end-points that can be reliably translated into QALYs. Diagnostics, which POCTs fall under, have traditionally been evaluated based on their sensitivity and specificity, with less evidence for clinical end- points. The European In Vitro Diagnostics Regulation includes a requirement for diagnostics to provide evidence of clinical effectiveness, potentially going some way to addressing this problem. What is lacking though from HTAs of diagnostics is information relevant to primary care clinicians, who are a key stakeholder in the decision to implement POCTs in primary care. A different way of thinking about cost-effectiveness is required to incentivize the local adoption of POCTs as clinicians have not been swayed by the cost per QALY argument. Results. This work proposes a value framework for POCTs designed to facilitate discussion with local decision makers about the adoption of POCTs in primary care. Conclusions. POCTs have the ability to reduce costs, improve patient outcomes and enhance the care-pathway. When communicating this to clinicians though it is important to be clear about where in the system the costs and benefits fall.","Oral Presentation Number: OP159 Abstract Title: Quality Of Evidence For Clinical Benefit Of Cancer Medicines Assessed For Funding In Australia Author Names: Agnes Vitry ([email protected]), Cathy Caird Introduction. This study aimed to describe the type of evidence available for and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC). The evidence was assessed with the European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS). Methods. All data on applications submitted to PBAC between 2010 and 2020 were independently extracted in duplicate from PBAC Public Summary Documents available online. Any disagreements were resolved through discussion. ESMO-MCBS ratings were retrieved from the ESMO -MCBS website. Substantial benefit for the ESMO-MCBS was defined as a grade A or B for (neo)adjuvant intent and four or five for palliative intent. Results. In the study period, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68%) for solid tumors (116 in the palliative setting) and 58 (32%) for hematological cancers. A total of 138 (76%) indications were recommended for public funding, 40 (22%) were rejected, and four (2%) were deferred. Randomized controlled trials (RCTs) were the main source of evidence in 154 indications (85%) and single-arm studies in 27 (15%) indications. RCTs were available in 113 (91%) and 41 (71%) of the solid tumor and hematological cancer indications, respectively. In submissions with RCTs, mature overall survival (OS) was reported in 81 (53%) indications. For indications with a statistically significant improvement in OS, the median gain was 3.0 months (range 0.9 to 17.0) for solid tumors and 8.2 months (range 1 to 9.1) for hematological cancers. The ESMO-MCBS score was available for 99 solid tumor indications. For indications in the palliative setting, 47 (52%) had substantial clinical benefit according to ESMO-MCBS v1.1, including 35 (51%) indications recommended by PBAC and six (35%) indications that were rejected. Conclusions. These results show that only a minority of cancer medicine indications considered by PBAC are supported by a good level of evidence and provide a modest extension of patient survival.","Oral Presentation Number: OP162 Abstract Title: Making Local Economic Evaluation More Relevant: Using Expert Elicitation To Adjust Published Intervention Effects To Reflect Local Context Author Names: Jodi Gray ([email protected]), Jonathan Karnon Introduction. Expert elicitation is often used in economic analyses to estimate uncertain or unobserved parameters for decision models. However, it has rarely been used in the context of local decision-making. A pragmatic elicitation process was used during a local economic evaluation to prompt local experts to assess the relevance of the published evidence to their setting, and to adjust the published effect estimates to better reflect the intervention effect expected in their setting. Methods. Elicitation was undertaken for two interventions that targeted the prevention of hospital-acquired hypoglycemia. Six clinical experts from within the Southern Adelaide Local Health Network (SALHN) were systematically presented with information on the setting of the published evaluation and their local setting. This included information on the hospital and quality of care, patient characteristics, and the research context. After comparing the settings, the experts were asked to estimate the most realistic, most pessimistic, and most optimistic intervention effects for their local context. Results. The local intervention effect was estimated to be smaller than the published estimate for both interventions. For one intervention, this was driven by the lower complexity of the local patient cohort. For the other intervention, it was driven by differences in the scope of implementation, with hospital-wide local implementation expected to reduce staff buy-in relative to the targeted implementation used in the published evaluation. The elicited local intervention effects were used in a cost-consequence analysis to estimate the likely costs and effects of the interventions if they were implemented locally. Conclusions. The pragmatic elicitation process provides a feasible and acceptable way to assess and transparently adjust the published effect estimates to better reflect the expected intervention effect in the local setting. Including this step in local economic evaluations can increase the relevance of these evaluations to local decision makers. Further development and application of these methods may facilitate greater use of economic evaluation in local settings.","Oral Presentation Number: OP163 Abstract Title: Applying A Local Economic Evaluation Framework To Make Evaluations More Relevant For Local Decision Makers Author Names: Jodi Gray ([email protected]), Jonathan Karnon Introduction. Economic evaluation is infrequently used by local health services. To be useful to local decision makers, economic evaluations need to synthesize published evidence on effective interventions with local data and local stakeholder knowledge regarding patient and organizational contexts. A framework for local economic evaluation was applied by health economists working with a local health service to inform their decision- making regarding funding of health service delivery models to reduce hospital-acquired complications. Methods. The framework engaged with local stakeholders to set priorities, assess the relevance of the published evidence, interpret local data, provide insight on the local context, and make recommendations to decision makers. It involved: (i) synthesizing the published evidence in a pragmatic review; (ii) determining local root causes and baseline incidence rates using local clinical and administrative data; and (iii) using expert elicitation to adjust published intervention effects to reflect the local context. This information was synthesized in a cost- consequence analysis that estimated the likely costs and effects of relevant interventions if they were implemented locally. Results. Local stakeholders selected hypoglycemia and urinary tract infections as targets for intervention. Tools and resources developed for each case study included: clinical audit tools and analysis files; pragmatic literature reviews with templates to present interventions to local stakeholders; an expert elicitation framework; and R code for cost-consequence analyses that apply published and elicited intervention effects to local data. Conclusions. The framework provided a feasible and acceptable process for undertaking local economic evaluations. Engagement with local stakeholders ensured the evaluations produced were relevant and tailored to the local setting and were therefore useful to local decision makers. The tools and resources developed can be applied by other local health services. The framework itself can be used for other case studies. However, the time and cost associated with the evaluations was not sustainable and alternative models for applying the framework need to be explored.","Oral Presentation Number: OP165 Abstract Title: Changing Decision Criteria: An Analysis Of Health Technology Assessment Reports To The Brazilian Unified Health System Author Names: Stefani Borges ([email protected]), Fernanda Rodrigues, Wolney Pires, Nathalia da Costa, Priscila Louly, Clementina Prado, Vania Santos Introduction. In Brazil, the National Committee for Health Technology Incorporation of the Brazilian Public Health System (Conitec) defines the criteria for incorporating technologies into the public health system. However, all recommendations are submitted to public consultations to ensure transparency and public participation. Contributions from the public consultation are analyzed and entered into Conitec\u2019s final report and sometimes change the preliminary recommendation. Methods. This descriptive, retrospective exploratory study aimed to gather qualitative and quantitative data on criteria considered by healthcare decision makers from Conitec and to identify which criteria most changed after reports were submitted to public consultation. Data from 2012 to November 2022 were collected from the Conitec website, entered into a specific extraction form, and analyzed using descriptive statistics. Results. Medicines (77%) were the most frequently evaluated type of technology, followed by procedures (15%), and products (8%). A total of 763 recommendations were analyzed. Of these, 70 percent did not change the decision criteria from the preliminary to the final published report, 9 percent did change, and 20 percent were simplified analyses of technologies of relevant public interest that were of low cost and low budget impact (these were not submitted to public consultation or public hearing). As pharmaceutical companies usually propose cost reductions or discounts during the Conitec public consultations, most of the recommendations (45%) were modified in the final report, with incremental budgetary reduction being the main criterion. This study identified that most of the changes occurred in neurology and were related to the high incremental budgetary impact of technologies for the care of rare diseases like spinal muscular atrophy. Conclusions. Although different criteria are considered for Conitec recommendations, some of these are regarded as crucial for decision-making. Unfortunately, as there is no definition for the weight of each of these criteria, it is difficult to understand their influence on the recommendations made. Therefore, it is crucial to establish standardized criteria for proper decision-making.","Oral Presentation Number: OP167 Abstract Title: An Analysis Of Medication Exclusion Reports In The Health Technology Assessment Process For The Brazilian Unified Health System Author Names: Nathalia da Costa, Stefani Borges ([email protected]), Wolney Pires, Fernanda Rodrigues, Priscila Louly, Clementina Prado, Vania Santos Introduction. The National Committee for Health Technology Incorporation of the Brazilian Public Health System (Conitec), which was created in 2011 by federal law, defines the criteria for incorporating and excluding health technologies in the Brazilian health system. For technology evaluation proposals a recommendation report is carried out, which finds the best scientific evidence about the efficacy, accuracy, effectiveness, and safety of the technology being analyzed. The report also provides a comparative economic evaluation of the technologies already in the health system to promote and protect public health, achieve the best allocation of resources, and reduce regional inequalities. Methods. This exploratory, descriptive, and retrospective study aimed to identify the criteria considered for the exclusion of medicines within the Brazilian health system from 2012 to November 2022. Data were extracted from Conitec\u2019s website into a specific extraction form and analyzed using descriptive statistics. Results. During the evaluation period, 763 technology recommendations by Conitec were identified, of which 75 (10%) related to the exclusion of medicines. Several criteria were identified among the exclusion recommendations, including protocol updates, expiry or lack of registration in the National Health Surveillance Agency, and drugs whose clinical indication was not included in the list of drugs registered for treatments in the health system. Conclusions. Although there is no standardization of criteria in Conitec for recommendations on the exclusion of health technologies, this study contributes to a better understanding of these technologies. Disinvestment of technologies positively impacted the population because it resulted in better allocation of resources in the health system, and some of the excluded technologies presented a greater risk of interactions and undesirable adverse reactions.","Oral Presentation Number: OP168 Abstract Title: Costs And Effectiveness Of Whole Exome Sequencing (WES) In Patients With Unsolved Rare Disease Through The Diagnostic Pathway Author Names: Deborah A Marshall ([email protected]), Koen Degeling, Toni Tagimacruz, Trevor A. Seeger, Kym M Boycott, Francois Bernier, Roberto Mendoza-Londona, Karen V. MacDonald, Taila Hartley, Robin Z. Hayeems Introduction. Patients suspected of having a rare genetic disease often experience lengthy and costly diagnostic odysseys. The timing of whole exome sequencing (WES) in the testing sequence, its diagnostic yield and test costs in the sequence all factor into estimates of cost-effectiveness analysis for health technology assessment. Methods. We modeled the diagnostic pathway using a discrete event simulation model, starting with the first test result. We defined and populated the simulation based on data from the electronic medical records of n=307 from the Care-for-Rare SOLVE multi-center Canadian observational cohort. Five alternative diagnostic pathways were modeled based on the observed data: no WES, and WES as the first, second, third or fourth test in the sequence. WES as the second test in the sequence is considered standard of care in medical genetic centers in Canada. We assessed effectiveness of WES in terms of diagnostic yield, time to diagnosis, and costs as patient- level overall test costs (2020 CAD\/USD) across the diagnostic pathway. Results. Compared to molecular and specialized diagnostic tests only (i.e., no WES), WES increased diagnostic yield from 5 percent to 40 percent. The shortest time to diagnosis for those with a diagnosis was 1.82 years in the diagnostic pathway with WES as the second test. Test costs for each pathway were CAD2,800 (USD2,087, no WES), CAD2,700 (USD2,013, WES as first test), CAD3,500 (USD2,609, WES as second test), CAD4,500 (USD3,354, WES as third test), and CAD5,300 (USD3,951, WES as fourth test). Conclusions. Placing WES earlier in the diagnostic pathway for patients suspected of having a rare disease is associated with an increased diagnostic yield, reduced time to diagnosis and lower overall test costs with the benefits being greater the earlier in the pathway that WES is implemented.","Oral Presentation Number: OP171 Abstract Title Canadian Disease Registry Inventory: Environmental Scan Of The Literature Author Names: Amanda Hodgson ([email protected]), Hannah Loshak, James Lachaud, Am\u00e9lie Bernard, Teri Slade, Genevi\u00e8ve M\u00e1k, Heather Smith Fowler Introduction. In consideration of the lessons learned from other jurisdictions and other ongoing work in the disease registry data space, an opportunity existed to investigate the current Canadian landscape and identify opportunities for a Canadian registry list. Previously, no national-level inventory of registries existed in Canada that could provide the necessary information to support awareness and use of available data for decision-making. Methods. A literature search was conducted on key resources, including MEDLINE and a focused internet scan. No methodological filters were applied to limit retrieval by publication type. The search was limited to documents published in English or French. Results. Core characteristics of the identified registries were extracted and contextual information on the current landscape of disease registries in Canada was explored. A literature review and draft inventory list has been produced. Conclusions. A CADTH environmental scan was undertaken to collect and report on existing Canadian disease registries and to identify key features, characteristics, and intersections. This information and analysis increase the potential of Canadian registries to inform decision-making and identifies opportunities for the optimal use of registry data in Canada more broadly.","Oral Presentation Number: OP172 Abstract Title: International Collaboration For Translating The Peer Review Of Electronic Search Strategies (PRESS) Checklist: A Harmonized Approach Author Names: Amanda Hodgson, Daniele Masterson, T. P. Ferreira, Josu\u00e9 Laguardia, Cicera Henrique da Silva, Martha Silvia Martinez Silveira, Jessie McGowan, Danielle Rabb ([email protected]) Introduction. The PRESS guideline and checklist provides a set of recommendations concerning the information that should be used by librarians and other information specialists when they are asked to evaluate electronic search strategies. CADTH and PRESS authors were approached for permission to translate this checklist into various languages. Methods. The team from Funda\u00e7\u00e3o Oswaldo Cruz and the Universidade Federal do Rio de Janeiro asked CADTH for permission to translate the PRESS guideline and checklist for their research work. They translated PRESS following the steps advocated by the scientific literature on the translation of standardized questionnaires. Results. CADTH is now sharing and actively disseminating the PRESS translations in French and Portuguese (and a forthcoming version in Spanish) via the Finding the Evidence website and through related presentation activities. Conclusions. The coordinated translation of key health technology assessment (HTA) tools provides an avenue for international uptake and improvement of best practice in information retrieval, which is a foundational feature of HTA work. With the absence of formal translation guidelines on the translation of protocols such as PRESS, CADTH would benefit from developing guidance for HTA teams requesting to translate our tools. CADTH is currently conducting research on the uptake and use of PRESS, which will inform future knowledge mobilization strategies such as translation standards and communications.","Oral Presentation Number: OP173 Abstract Title: Estimating The Marginal Productivity Of Health Technology Adoption Author Names: Charles Yan ([email protected]), Jemal Mohamed, Manik Saini, Robert Bacigalupo, Selva Bayat, Jeff Round Introduction. Decisions to adopt health technologies rely, in part, on judgements about cost effectiveness. Cost effectiveness is commonly assessed against a willingness-to-pay threshold for health gains. Building an evidence base on the marginal productivity of health spending to inform the value of the threshold is increasingly of interest for resource allocation decision-making and technology implementation. We report on an in-progress analysis to inform a threshold for policy purposes in British Columbia, Canada. Methods. We developed a ten-year panel-data model with instrumental variables, which lessens the degree of time- invariant confounding and addresses biased causal inferences caused by unobserved factors, to provide estimates of the marginal cost per health unit measured using quality-adjusted life-years (QALYs). We use the Johns Hopkins Adjusted Clinical Group (ACG) system and a British Columbia Health System Matrix to classify patients into six resource use bands (RUBs) ranging from \u2018healthy\u2019 to \u2018very high morbidity\u2019. Patients are also classified by chronic conditions and types of services. Place of residence and geographical region of health authorities are considered. Variables included age, gender, mortality and comorbidity rates, costs of hospitalizations, emergency department and physician visits, residential and home care, laboratory services, diagnosis and medications, and quality of life. Instrumental variables included sociodemographic characteristics as reported in the Canadian census. Results. The largest RUB was \u2018moderate\u2019 morbidity (39.3%), while the smallest was \u2018healthy\u2019 (1.5%). The youngest was the \u2018low\u2019 morbidity (mean 31, standard deviation [SD] 21) and the oldest was \u2018very high\u2019 (mean 69, SD 17). The healthy group had the smallest mean costs (CND563, SD CND4,121; equivalent to USD421, SD USD3,083). In contrast, the \u2018very high\u2019 group had the largest (CND20,398, SD CND36,188; equivalent to USD15,258, SD USD27,069). Age and gender standardized comorbidity index scores ranged from 0.05 to 6.41 (median 0.98). Additional analyses (e.g., costs per QALY) are ongoing and the results will be reported at the conference. Conclusions. Our empirical approach is robust and flexible, allowing estimates of marginal productivity according to factors such as disease, geographical region, service type, and care sector. This work has applications at the provincial and national levels and adds to methodological literature in the field.","Oral Presentation Number: OP174 Abstract Title: Health Technology Assessment And Economic Evaluations For A Genomic Strategy In Italy Author Names: Eugenio Di Brino ([email protected]), Debora Antonini, Giulia Falasca, Michele Basile, Filippo Rumi, Americo Cicchetti Introduction. The challenge to health systems is sustainability, not only in the economic and financial sense of compatibility of spending with allocated resources, but also in terms of equity in access to services and care, quality, safety, innovation and research, that is, in terms of the effectiveness of the right to health. Some countries, such as Italy, do not have formal health technology assessment (HTA) or other similar processes that take into account views outside the decision maker. Methods. In the Italian national context, there is currently no single tariff for the reimbursability and pricing of genomic technologies. In fact, although genomic tests have been in clinical practice for many years now, to date they have not yet been included in the LEA (Minimum healthcare provision), especially in view of the fact that a defined and transparent process for updating the Essential Levels of Care has been operational since 2018. With the goal of structuring guidelines for the adaptability of economic evaluations to currently available and developing genomic technologies, a literature review was conducted. Results. The literature review showed that there are some methodological and practical issues that need to be carefully considered when designing and conducting economic evaluations of genomic tests. In more detail, five key concepts were identified in order to implement the most comprehensive economic evaluation of the technologies under study: the PICO model, the survey perspective, the costs included in the analysis, the effectiveness analysis, the time horizon, and the discount rate. Conclusions. Adequate definition of these concepts appears to be of paramount importance in view of the fact that genomic testing may have important consequences for future generations as well. For the purpose of sustainability of access of genomic technologies, the use of Budget Impact Analysis (BIA) is recommended in all analysis settings being essential for the regulator to tie access to its available budget capacity.","Oral Presentation Number: OP176 Abstract Title: Integrating Real-world Compliance In The Assessment Of Left Atrial Appendage Closure Versus Anticoagulation Therapy In Non-valvular Atrial Fibrillation Author Names: Sebastian Pyne, Dominic Tilden ([email protected]), Gordon Calcino, Mia Mudge Introduction. In patients with non-valvular atrial fibrillation (NVAF), left atrial appendage closure (LAAC) has demonstrated non-inferior efficacy and safety relative to life-long oral anticoagulation therapy (OAT) in a four-year randomized controlled trial (RCT) (PRAGUE-17). Sub-optimal compliance to OAT in the real-world setting (Simmons 2016) has been associated with increased risk of stroke (Ozaki 2020) and may alter efficacy estimates derived from RCTs in which compliance is generally higher. The study aims to model disease outcomes in NVAF patients treated with LAAC versus lifelong OAT when applying trial versus real world compliance to OAT. Methods. Real-world compliance to OAT in the Australian setting was investigated in a 10 percent Pharmaceutical Benefits Schedule (PBS) sample scripts analysis which measured treatment adherence and persistence to new oral anticoagulants (NOACs) and warfarin. Design of the 10 percent PBS analysis was informed by the compliance to medicine working group report and included the longest follow-up of any OAT compliance study identified in the literature. A Markov cohort model was developed to estimate the expected numbers of strokes and major bleeding events in NVAF patients. Results. Rates of NOAC discontinuation in PRAGUE-17 was higher at 20 months median follow-up (6.5%) versus compliance in the Australian setting (35.4% and 30.0% according to 3 and 6 month ceasing rules at 20 months follow-up). Applying sub-optimal compliance to lifelong OAT demonstrated in the Australian setting resulted in higher numbers of strokes over a life time modelled time horizon compared with LAAC. Conclusions Real world compliance to medicines should be a consideration in economic analysis comparing lifelong medications to one-off surgical interventions.",""]