2020-07-30 Final Poster Abstracts_EK

Abstract Number: 406 Abstract Title: Academic Detailing For Judges: Concepts Of Evidence-Based Medicine And Health Policies Adopted In Brazil Abstract Status: Accepted Poster Presentation Author Name: Marcus Carvalho Borin ([email protected]), Carla Barbosa Morais Alves, Nelio Gomes Ribeiro Junior, Melissa de Fátima Guimarães, Francisco de Assis Acurcio, Juliana Álvares- Teodoro, Augusto Afonso Guerra Junior Introduction The Brazilian health system has not been able to enforce the constitutional Right to Health for the entire population, leaving litigation as the last alternative for the fulfillment of the right. In order to harmonize and underpin the decision making of federal judges, an Academic Detailing (AD) program with concepts of evidence-based medicine and health policies for federal judges will be conducted nationwide in Brazil. AD is a strategy to provide information, combining an interactive outreach approach with the best evidence. This study reports the method used to define key messages to be used during visits. Methods Government, federal judges and academy representatives were invited to a workshop on health litigation in Brazil. They were divided into six groups to discuss five hypothetical scenarios. In each scenario, groups listed two possible key messages to disseminate during AD, addressing the legal, scientific, economic or ethical dimensions. After the definition, a vote was taken, according to the importance that each participant attributed to them (1 to 10), and then a score was generated. Results Of the thirty-one participants, five were from the judiciary, three prosecutors, one health insurance representative, nine managers and thirteen from the academy. From the case study presented, fifty-five key messages were suggested. After removing duplicates, twenty-five were selected to assign importance values by each participant. The key messages were sorted from highest to lowest score, the most important being “Explain what evidence-based medicine means and what scientific evidence means”. Conclusions Knowledge of theoretical concepts of evidence-based medicine, health policies and the technology incorporation process seem to be of fundamental importance to federal judges. This information will serve as a basis for decision making regarding litigious proceedings involving the constitutional Right to Health in Brazil. From the selected key messages, bulletins to the academic detailing program to federal judges shall be produced.

Abstract Number: 422 Abstract Title: Including Empowering Community Into Primary Healthcare Team Scope Of Practice – A Technology For Ensuring Universal Healthcare Coverage Abstract Status: Accepted Poster Presentation Author Name: Rabiga Khozhamkul ([email protected]), Lyazzat Kosherbaeva, Ainur Bakdaulet, Talapkali Izmukhambetov, Arnoldas Jurgutis, Saule Tolegenova Introduction Considering the World Health Organization (WHO) Astana Declaration, in order to provide universal healthcare coverage, Kazakhstan through ongoing healthcare reform committed to the promotion of a people-centered Primary Healthcare (PHC) system. Since the implemented top-down policies showed low buy-in from community members and put more constraints on PHC facilities and teams, the Kazakh National Medical University, the Medeo district mayor’s office and the WHO European Centre for PHC supported the initiative of a local non-governmental organization “Community health committee” and Outpatient clinic of Almaty State hospital #5, for creation of an integrated plan to develop people- centered PHC through better coverage and engagement of patients with non-communicable disease and enhancing the health literacy of the population above 65 years. Methods We used a community-based participatory approach. The process consisted of: forming a steering committee with at least one member from each stakeholder group; two interactive workshops where the community worked jointly with PHC professionals in defining priority health needs and proposing actions to address selected priorities; and, after, joint development by all stakeholders of an action plan for empowerment of the community, and for assessment and review of the scope of practice of PHC teams. Results The interactive workshops identified priority health needs such as low health literacy, low responsibility for health, low engagement of the elderly in prevention and self-management of non-communicable diseases. The main findings of semi-structured interviews were that there are no planning approaches (neither shared planning by a PHC team, or individually by PHC professionals) for addressing defined priority needs of the community, families and/or patients, and that the role of a PHC team in community empowerment is very limited. Conclusions Using results of the review on PHC teams’ scope of practice, we will develop, discuss and agree with the national and local stakeholders’ proposal of a conceptual model of PHC service delivery. Further, we will implement and evaluate the results of implementation.

Abstract Number: 434 Abstract Title: Quality Of Studies Submitted To Support Requests For Medical Devices And Equipment Incorporation In A Teaching Hospital Abstract Status: Accepted Poster Presentation Author Name: Katiuce Tomazi Kny ([email protected]), Maria Angélica Pires Ferreira Introduction In hospitals with standardized processes of health technology assessment (HTA), clinical studies are usually required to support incorporation requests. Factors such as conflicts of interest and confirmation bias affect the quality of the evidence. The objective of this study was to assess the quality of studies submitted to the university hospital to support the incorporation of non-drugs technologies. Methods All submissions received from 2017 to 2019 were reviewed. Data about methodological quality of studies sent by requesters and their utilization of final recommendation were collected, as well as about studies retrieved during additional search performed by HTA. The Jadad and Assessing the Methodological Quality of Systematic Reviews (AMSTAR) scales were used for assessing the quality of randomized clinical trials (RCTs) and systematic reviews (SRs), respectively. Results Twenty-one requests for incorporation of equipment were analyzed. The average number of studies attached was 4.5. In eight requests (53.3%), both SRs and RCTs were attached. In seven (46.7%) only low- quality studies were included were included; additional search identified RCTs and SRs in four of these cases. According to the Jadad and AMSTAR scales, 60 percent of the submitted RCTs showed a high risk of bias, while 57 percent of the SRs showed moderate quality, respectively. Conclusions The best evidence is not always submitted during the incorporation request process. Requirements for studies of moderate-to-high quality to accept the incorporation demand could be helpful to avoid inefficiencies in hospital-based HTA.

Abstract Number: 443 Abstract Title: Effectiveness And Safety Of Transcatheter Therapy Interventions Devices For Treatment Of Tricuspid Regurgitation Abstract Status: Accepted Poster Presentation Author Name: Esther Elena Garcia_Carpintero ([email protected]), Jordi Gol Freixa; Luis María Sánchez Gómez Introduction Tricuspid regurgitation (TR) is defined as incompetence of the tricuspid valve (TV), which produces the movement of blood flow from the right ventricle (RV) to the right atrium during systole. Pathological TR is functional in nearly 80-90 percent of cases, secondary to volume and/or pressure overload in the RV. Surgical intervention of TR is associated with mortality rates of 10 percent. Transcatheter therapy interventions (TTI) can be an alternative for severe TR. The aim of this study is to assess effectiveness and safety of TTI. Methods A systematic review was carried out. The scientific literature search was performed in major medical databases. Studies analyzing the efficacy and safety of the devices were included. Outcomes related with mortality rates, TR volume reduction, echocardiographic findings and adverse events were analyzed. The methodological quality of the studies was analyzed with the Canadian Institute of Health Economics Quality Appraisal Checklist. Results Nine studies comprising 557 patients were included (two first-in-human studies, one retrospective, five single arm prospective studies and one international registry). The studies were small with short follow up. The outcome of procedural success ranged from 80 to 100 percent. Mortality rates at 30 days were lower than 5 percent. Improvements in reduction of TR, European System for Cardiac Operative Risk Evaluation (EuroSCORE), heart failure symptoms or quality of life scores were observed in all studies. Conclusions TTI for moderate-severe TR show significant reduction of annulus dimension, improvements in heart failure symptoms and quality of life, which are maintained in mid-term follow up. TTI present lower rates of major serious adverse events. No differences were observed between different TTI devices in terms of procedural success, mortality or safety. Randomized studies comparing TTI with optimal medical therapy are needed to confirm the preliminary clinical impact in patients with severe TR, and define aspects such as patient selection, risk factors associated with procedural success or mortality rates.

Abstract Number: 445 Abstract Title: Mapping The Trend Of The Da Vinci Surgical System Use In China Abstract Status: Accepted Poster Presentation Author Name: Jin Zhao ([email protected]), Min Zhong, Min Hu Introduction A robotically assisted surgical system, the da Vinci surgical system (DVSS), is a sophisticated surgical platform equipped with immersive 3D visualization and dexterous articulating endoscopic instruments. Surgeons can intuitively control the surgical system to perform delicate surgical tasks. Robotic surgery has gained popularity globally ever since its birth and was approved to market by the China Food and Drug Administration in 2006. This study aims to map the current use of DVSSs in mainland China and the trends from 2009 to 2019. Methods A full-sample survey of all hospitals equipped with DVSSs was conducted in mainland China, collecting data on hospitals and surgical departments using DVSSs, operation volume, and time of installation. Disease classification was standardized to obtain DVSS use in each department. EXCEL software was used for logging and cleaning the data. The analysis focused on descriptive analysis to map trends of DVSS use in China and present geographical and department distribution. Results The DVSSs installed have grown from seven in 2009 to 135 in 2019. By the end of 2019, twenty-eight provinces in China have been equipped with the DVSSs, among which eighty-seven in the eastern regions, twenty-seven in the central regions, and twenty-one in the western regions. The annual volume of operations grew from 339 in 2009 to 38,991 in 2019, at an annual rate of 60.7 percent. The average workload conducted by a single robot is much higher than that of their counterparts in other countries. The largest share of the volume is in department of urology (48%), followed by general surgery (25%) and thoracic surgery (13%). Conclusions The use of DVSSs in China has been growing rapidly and extensively, with certain differences between geographical regions and surgical departments. We need to further explore the factors affecting its use and operation efficiency and to evaluate the effectiveness as well as cost-effectiveness in real-world clinical practice to inform public policies on application of DVSS, for example, license and insurance.

Abstract Number: 455 Abstract Title: Cost-effectiveness Of Ixazomib-Based Regimen Compared With Bortezomib-Based Regimen In Chinese Patients With Relapsed/Refractory Multiple Myeloma: A Retrospective Study Abstract Status: Accepted Poster Presentation Author Name: Pei Wang, Jing Li, Yang Yang, Peng Liu ([email protected]) Introduction The treatment of relapsed/refractory multiple myeloma (RRMM), a common hematological malignancy, remains a great challenge in China, partially due to the limited accessibility to novel agents and inadequate public health insurance coverage. Ixazomib, a novel oral proteasome inhibitor (PI), was approved by the China Food and Drug Administration (CFDA) for RRMM in 2018. While bortezomib, a traditional PI, is the recommended agent in the clinical guideline for MM. Here, we compared their costs and effectiveness. Methods RRMM patients who has received an ixazomib-based regimen (at least 2 cycles) were analyzed. Using a propensity score matching method, we generated a control group of RRMM patients who received the bortezomib-based regimen. The criteria included the number of treatment lines, age, and the revised international staging system stage (R-ISS) which representing the disease stage for myeloma, and paired at a ratio of 1:2 (allowing one control to match multiples). The difference in hospitalization stay, grade 3/4 adverse events rates, overall response rate (ORR), mortality during treatment, and treatment costs was then compared. Results Nineteen patients received ixazomib and twenty-seven that received bortezomib were included. The ixazomib-group demonstrated a shorter hospital stay (9 days versus27 days, p<0.001), lower grade 3-4 adverse events rates (42.1% versus55.6%, p<0.001), higher ORR (63.2% versus48.1%, p=0.228), and lower mortality rate during treatment (0%versus7.4%, p=0.169) than that of bortezomib-group. The ixazomib group had lower total costs (127,620CNY versus156,424CNY [18,033USD versus 22,103 USD] , p>0.05), lower drug costs (98,376CNY versus 103,307CNY [13,901USD versus 14,598 USD] , p>0.05), and the lower costs of supportive treatment (5,507CNY versus14,701 CNY [778USD versus 2,077 USD] , p<0.001). Only in terms of self-funded costs, the bortezomib-based regimen was significantly lower (37,127CNY versus11,521CNY [5,246USD versus 1,628 USD], p<0.001). Conclusions Compared with the bortezomib-based regimen, the ixazomib-based regimen has better therapeutic effects on MM patients while saving costs. Hence, it may be preferable for use in the treatment of RRMM in China.

Abstract Number: 459 Abstract Title: Healthcare Resource Utilisation Of Anti-Neutrophil Cytoplasmic Antibody Associated Vasculitis Patients: Real-World Data From English Clinical Practice Research Datalink Abstract Status: Accepted Poster Presentation Authors: Antonio Ramirez de Arellano Serna ([email protected]), Matt Glover, Cormac Sammon, Tzu-Chun Kuo, Philip Spearpoint, Peter Rutherford Introduction Anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) is a rare, serious and often life- threatening disease. The use of available treatments options (immunosuppressants and glucocorticoids (GCs)) improves the prognosis of AAV greatly; however, GC use is associated with significant toxicity related morbidities and the management of AAV is costly. However, information of the costs associated with AAV in the United Kingdom is limited. This study aimed to quantify the burden of AAV using a large England and Wales source of real-world data, the Clinical Practice Research Datalink (CPRD) Hospital Episode Statistics (HES) linked database, to identify healthcare resource utilization and generate estimates of costs. Methods Incident patients (n=220) were included if ≥ eighteen years, with diagnosis read codes G754.00/G75A.00; ICD codes M31.3/M31.7 from January 1997 to December 2017. Costs were taken from Unit Costs of Social and Health Care, National Health Service reference costs and electronic drug tariff. Distinction was made between type of consultations, outpatient visits and inpatient admission based on Healthcare Resource Grouping. Costs were summarised as mean per member per year (PMPY) in 2016 prices and presented before and after diagnosis. Results In the year preceding AAV diagnosis, mean costs PMPY were GBP12,012 [USD15,400], (GBP5,339 [USD6,845] inpatient, GBP766 [USD982] outpatient, GBP314 [USD403] GP, GBP5,594 [USD7,172] GP prescribing). In the year of AAV diagnosis (Y0) costs PMPY were GBP28,252 [USD36,220], GBP15,436 [USD19,790] inpatient, GBP1,863 [USD2,388] outpatient, GBP2,407 [USD3,086] GBP8,545 [USD10,956] GP prescribing). Costs in the years post-diagnosis remained higher than pre-diagnosis with a low of GBP22,839 [USD29,281] in Y4. The prescribing costs (GC, methotrexate and azathioprine) were the largest contributor in Y0-Y4 (GBP15,047 [USD19,291] Y1; GBP12,325 [USD15,801] Y4). Conclusions Diagnosis of AAV is associated with increased healthcare costs, including higher inpatients costs in the year of diagnosis and subsequently higher prescribing costs in the community. Given the incidence (17.2 cases per million) and considering only costs in the year of diagnosis, an additional GBP15.6 million [USD24.6 million] of healthcare resource utilization occurs every year from new diagnoses of AAV. However, this will likely be underestimated due to the lack of secondary care prescribing data in CPRD- HES and prescribing of immunosuppressant treatments in this setting.

Abstract Number: 469 Abstract Title: From Theory To Practice: Which Value Framework Is Applied For Onco-Hematology Therapies In Italy? A 5-year Retrospective Analysis Abstract Status: Accepted Poster Presentation Author Name: Americo Cicchetti, Rossella Di Bidino ([email protected]), Entela Xoxi, Irene Luccarini, Alessia Brigido Introduction Different value frameworks (VFs) have been proposed in order to translate available evidence on risk- benefit profiles of new treatments into Pricing & Reimbursement (P&R) decisions. However limited evidence is available on the impact of their implementation. It’s relevant to distinguish among VFs proposed by scientific societies and providers, which usually are applicable to all treatments, and VFs elaborated by regulatory agencies and health technology assessment (HTA), which focused on specific therapeutic areas. Such heterogeneity in VFs has significant implications in terms of value dimension considered and criteria adopted to define or support a price decision. Methods A literature research was conducted to identify already proposed or adopted VF for onco-hematology treatments. Both scientific and grey literature were investigated. Then, an ad hoc data collection was conducted for multiple myeloma; breast, prostate and urothelial cancer; and Non Small Cell Lung Cancer (NSCLC) therapies. Pharmaceutical products authorized by European Medicines Agency from January 2014 till December 2019 were identified. Primary sources of data were European Public Assessment Reports and P&R decision taken by the Italian Medicines Agency (AIFA) till September 2019. Results The analysis allowed to define a taxonomy to distinguish categories of VF relevant to onco- hematological treatments. We identified the “real-world” VF that emerged given past P&R decisions taken at the Italian level. Data was collected both for clinical and economical outcomes/indicators, as well as decisions taken on innovativeness of therapies. Relevant differences emerge between the real world value framework and the one that should be applied given the normative framework of the Italian Health System. Conclusions The value framework that emerged from the analysis addressed issues of specific aspects of onco- hematological treatments which emerged during an ad hoc analysis conducted on treatment authorized in the last 5 years. The perspective adopted to elaborate the VF was the one of an HTA agency responsible for P&R decisions at a national level. Furthermore, comparing a real-world value framework with the one based on the general criteria defined by the national legislation, our analysis allowed identification of the most critical point of the current national P&R process in terms of sustainability of current and future therapies as advance therapies and agnostic-tumor therapies.

Abstract Number: 482 Abstract Title: Invasive Electrorncephalography In The Pre-Surgical Diagnosis Of Pharmacoresistant Epilepsy Abstract Status: Accepted Poster Presentation Author Name: Andrey Avdeyev ([email protected]), Azat Shpekov, Valeriy Benberin, Nasrulla Shanazarov, Leilya Ismailova, Ruslan Akhmedullin, Arailym Nurmasheva Introduction Worldwide, more than 50 million people suffer from epilepsy, and there are 16-51 new cases per 100,000 population each year. Up to 30 percent of patients with epilepsy are pharmacoresistant, who are candidates for surgical treatment. Invasive electroencephalography (iEEG) is a mandatory method in the arsenal of epileptic centers, and is gradually becoming the gold standard for invasive determination of boundaries between the affected and functional zones of the cortex and subcortical brain. Treatment costs correlate with the severity of the disease, with patients having uncontrolled seizures incurring eight times the costs compared to those with controlled epilepsy. Methods To assess the clinical and cost-effectiveness of the iEEG in the pre-surgical diagnosis of pharmacoresistant epilepsy, a systematic search of literature by keywords in the MEDLINE database was conducted. The search resulted in sixty-six articles. The analysis included twenty studies that met the search criteria. Results Most studies including meta-analysis show very low rates of complications of iEEG. Literature data demonstrate cost-effectiveness of the method in patients with pharmacoresistant epilepsy in comparison with continued antiepileptic drug therapy. As an integrated method, rather than a simple method, it takes maximum account of clinical, neurophysiological and anatomical-functional data to achieve accurate localization of the epileptogenic zone. Currently, iEEG is a clinically effective method to improve the safety and specificity of resective surgery. Conclusions With the use of iEEG, mortality and disability of patients with pharmacoresistant epilepsy will be significantly reduced. It has also been proven that epilepsy surgery leads to significant financial savings in the treatment of pharmacoresistant epilepsy. The results of the clinical and economic evaluation (mini-HTA report) have been submitted to the Ministry of Healthcare for decision-making on including iEEG in government reimbursement system.

Abstract Number: 495 Abstract Title: Addressing The Interactions Between Health Regulation And Health Technology Assessment In Brazil Abstract Status: Accepted Poster Presentation Author Name Maíra Catharina Ramos ([email protected]), Margarete Martins de Oliveira, Erica Tatiane da Silva, Daniella Cristina Rodrigues Pereira, Flávia Tavares da Silva Elias Introduction The interaction of health technology assessment (HTA) and health regulatory agencies has been widespread, especially for decision-making in health system coverage. The objective of this paper is to report the HTA-regulatory interaction in Brazil. Methods This is a case study on the interaction between HTA ATS and regulation in Brazil. Technical documents and Brazilian legislation on health regulation and HTA were analyzed. The study was conducted in July 2019. Results HTA-Regulatory Interaction in Brazil is still incipient. There is no responsible agency for interaction between agencies, as there is in Europe and Canada, for example. In the last 4 years, cooperation has started between the Brazilian Health Surveillance Agency (Anvisa) and the Oswaldo Cruz Foundation (Fiocruz) for post-registration monitoring of medicines. During this partnership, 170 post-marketing drug opinions were prepared, assisting the regulatory agency in decision-making. Conclusions Brazil legislation guarantees essential medicines at low cost or free. The interaction between HTA and regulation has the potential to reduce the time taken to incorporate technology to the patient, in addition to ensuring greater safety for users of the Unified Health System. In this sense, it was observed that the interaction between health regulation and science and technology institutions has innovative potential in this approach.

Abstract Number: 498 Abstract Title: Decision Support Tool For Investments In Health Technology Replacement: Experience Of A Public Teaching Hospital In Brazil Abstract Status: Accepted Poster Presentation Author Name: Rosane Paixão Schlatter ([email protected]), Ana Paula Coutinho, João Antonio Paim Rodrigues, Paulo Roberto Stefani Sanches, Luciano Ribeiro, Mauricio Leser Casella, Simone Dalla Pozza Mahmud, André Frota Muller, Bruno Rodriguez Tondin Introduction Based on the needs assessment of the medical and non-medical departments, the Investment Committee of the Hospital de Clínicas de Porto Alegre (HCPA), a teaching hospital in Brazil, recommends on which technologies the limited financial resources should be invested. Technology inclusion requests are evaluated by the hospital's technology assessment unit. For technology replacement, we have found models to assess the criticality of medical equipment, but they were insufficient to support the decision, which involves all departments of our hospital. This study aimed to develop an automated tool to support decision making regarding investments in equipment replacement in the hospital. Methods A working group was set up with professionals from healthcare administration, clinical engineering and research departments. From the hospital’s inventory database, we developed the tool using Google SheetsR. We have defined three departments for pilot testing of the tool: hemodynamics, laundry, and basic research. These departments represent the areas of healthcare, support services, and teaching and research in the hospital. Results The criticality of medical equipment is assessed based on the criteria of function, physical risk, impact, remaining equipment life cycle, intensity of use and number of corrective maintenance actions performed. For the equipment in the administrative, support and research areas, the function and physical risk criteria were replaced by the safety and by the risks to the quality of service criteria. The evaluation is carried out by a multidisciplinary team. The tool categorizes the equipment into low, medium and high criticality. Conclusions The tool prioritized the equipment based on objective criteria evaluated by the departments’ multidisciplinary team comprising experts who use the equipment in their activities, the department administrator and clinical engineers, and provided transparency regarding the decision-making of the hospital's Investment Committee. In 2019, the limited financial resources were invested only in the replacement of highly critical equipment. We believe the tool can be reproduced in hospitals in low and middle-income countries.

Abstract Number: 499 Abstract Title: Disinvestment Supported By A Hospital-Based Health Technology Assessment Unit: A Case Of A Teaching Hospital In Brazil Abstract Status: Accepted Poster Presentation Authors Name: Rosane Paixão Schlatter ([email protected]), Katiuce Tomazi Kny, Maria Angelica Pires Ferreira Introduction The Hospital de Clínicas de Porto Alegre (HCPA), a public teaching hospital, has a Hospital-based Health Technology Assessment (HB-HTA) unit to support the decision-making process on technology incorporation, rationalization or disinvestment. In 2017, the plastic adhesive drape was standardized at HCPA for use in cardiovascular, digestive, orthopedic, and neurological surgery for the purpose of preventing surgical site infection (SSI). This study evaluated whether the plastic adhesive drape technology is more effective than the no adhesive drapes in the surgical procedures in which it is used in the HCPA, so as to support the medical board's decision regarding the rationalization of use. Methods The primary outcome was the surgical site infection rate (SSI). Searches were performed in PubMed, Cochrane and national and international health agencies: World Health Organization (WHO), National Institute for Health and Care Excellence (NICE), Centers for Disease Control and Prevention (CDC), Society for Healthcare Epidemiology of America (SHEA), Brazilian National Commission for the Incorporation of Technologies (CONITEC) and Brazilian National Health Surveillance Agency (ANVISA) databases. The search strategy combined terms related to the technology and types of surgery in which it is used in the HCPA. The quality of the included studies was assessed. Additionally, data on technology utilization and costs in the hospital were analyzed. Results Technology assessment followed AdHopHTA project recommendations. Data from the hospital showed that the technology has been used in fifteen surgical specialties, different from the proposed incorporation, with a progressive increase in consumption from 2017 to 2018. The literature review included a systematic review with seven clinical trials, which concluded that the plastic adhesive drape lacks benefits, with potential for increased risk of SSI. The evidence was of moderate quality. Conclusions The expenses associated with the use of the technology were considered unjustified as it is not reimbursed by the Brazilian Ministry of Health and its disinvestment was recommended. The Medical Board approved the disinvestment of the technology based on the evidence found by the HB-HTA unit, and the medical staff complied with the decision.

Abstract Number: 501 Abstract Title: Inclusion Of Key Stakeholders’ Views When Developing A mHealthAssessment Tool: Focus Groups And Health Consensus Results Abstract Status: Accepted Poster Presentation Author Name: Elisa Puigdomènech, Elisa Poses, César Velasco, Xavier Garcia ([email protected]), Mireia Espallargues Introduction The Agency for Health Quality and Assessment of Catalonia (AQuAS) is developing an evaluation tool for mobile health (mHealth) solutions to be used by health technology assessment (HTA) agencies and evaluation experts. In order to have a practical and comprehensive tool taking into account the particularities and challenges of mobile interventions, we considered the views and opinions of key stakeholders. The objective was to present the final selection of general aspects (dimensions) to be assessed in the evaluation, as well as the specific items (criteria) to be included in each of these topics, as a result of different co-design approaches with health professionals, developers, hospital managers, HTA agencies and patients. Methods A list of criteria used for health apps evaluation were drawn from a literature review. The initial list included eighty-nine criteria items grouped in nine domains. Those criteria and domains were discussed during four focus groups (FG). The importance of the criteria that were not considered as mandatory were later rated through a Delphi online sub-study, in a scale from one to six points, taking as consensus value when median value (median 6, Interquartile range, 0-1) was reached. Results FG reduced domains and criteria from nine to seven and from eighty-nine to thirty-three, respectively. Most mandatory criteria were related with security, user experience, and clinical effectiveness. Fifty- seven individuals (53.7% of 106 invited to participate) were registered in the online platform (50.1% women, 68.4% 35-64 years old and 42.1% from HTA agencies). From fifty non-mandatory criteria under consensus, ten criteria reached consensus (most from solution’s content and health problem covered domains) concluding with a 43/7 criteria/domain tool. Conclusions Insights from main stakeholders on the content of the tool for mHealth assessment were considered through the FG and Delphi technique. The dimensions of security and privacy, clinical effectiveness, solutions’ content, technological aspects, users’ experience and costs were considered mandatory. The dimension related to the impact on the organization was appraised as a secondary domain for evaluation. A workshop with AQuAS research team and HTA external researchers will help to define: the assessment methods (type of instrument, dichotomous responses and/or Likert scales) for the evaluation and the format and dimension’s weights of the final design of the tool.

Abstract Number: 502 Abstract Title: AQuAS Learnings From Implementing GRADE Approach, Especially The Evidence To Decision Framework And Multidisciplinary Final Consensus Abstract Status: Accepted Poster Presentation Author Name: Emmanuel Giménez, Maria-Dolors Estrada, Xavier Garcia ([email protected]), Maria Auxiliadora Castillo, Leonor Varela, Mireia Espallargues INTRODUCTION In recent years, the GRADE system has been adopted internationally to make judgments about evidence and/or recommendations. Recently, the Spanish Network of HTA (RedETS) has promoted among its members the use of the GRADE system, especially the “Evidence to Decision” tool (EtD) where a multidisciplinary panel is involved. The objective is to describe the methodological approach with the first AQuAS experience using this methodology in HTA, focused on inclusion/exclusion of these technologies in the Spanish National Health Service (NHS) portfolio. METHODS The standard EtD tool was used for clinical and financial decisions. Four multidisciplinary panels were constituted by relevant professionals in clinical practice and, where possible, patients. The four panels discussed on the following four interventions: in two cases, AQuAS prepared preliminary recommendations and participants provided feedback, while in the other two cases, experts received the evidence review and were asked to formulate recommendations. These recommendations were voted on and, in the case of disagreement, adapted and then voted on for a second time. Finally, any discrepancies were noted in the report. Evaluated interventions were: maxillofacial 3D-reconstruction, cataract surgery equipment, non-invasive surgery in obesity and pharmacological treatment in secondary fracture prevention. RESULTS Especially when more than one evaluative question was addressed in the HTA report the EtD and the consensus results required discussion. Consensus was fast but not immediate. Meeting length depended on the number of HTA questions and the amount of original disagreement in the recommendations. The nuances on how to write recommendations also affected the panel duration. All panels were successful in formulating the final recommendations. CONCLUSIONS Standardizing methodologies increased the homogeneity across HTA reports. The GRADE system is a feasible and useful tool because it favors transparency and rigor in drawing up recommendations on the inclusion/exclusion of technologies in the NHS portfolio. The EtD framework complements GRADE tables, which display the relevant evidence in a way that can be used by multidisciplinary groups to reach a consensual recommendation. Although all participants received a short training video, more experience in the use of these methodologies might shorten the duration of the process and facilitate reaching consensus. Some considerations on how to overcome the difficulties and complexity of this methodological approach are discussed.

Abstract Number: 506 Abstract Title: Health Technology Reassessment (HTR) Of A Non-Drug Technology: Methods Used By A Regional HTA Unit Abstract Status: Accepted Poster Presentation Author Name: Marie-Belle Poirier ([email protected]), Maria Benkhalti, Jérémy Joncas, Ouifak El Warrari, Aghiles Addad, Sonia Cheng, Frédéric Mior, Anne Méziat- Burdin, Pierre Dagenais Introduction An environmental scan conducted by the Canadian Agency for Drugs and Technologies (CADTH-March- 2019) revealed that several health technology assessment (HTA) organisations are currently developing standard health technology reassessment (HTR) processes. Here we present methods used to conduct an HTR of a prioritization programme for non-immediate life-threatening urgent surgeries implemented in 2017 at a tertiary referral hospital in (Quebec-Canada). This HTR initiative was conducted by a regional HTA unit to optimize the programme efficiency and resources utilization as well as to motivate change in the clinical community of other hospitals within its healthcare network. Patient and healthcare personnel satisfaction levels towards the programme were also considered. Methods In this case study, HTR methods and outputs were elaborated using elements presented in the CADTH environmental scan and relevant publications identified through PubMed and in the grey literature. Documents in English and French, published between January 2002 and March 2019 were considered. Key stakeholders were consulted to identify barriers of the programme implementation to other hospitals in regards to aspects related either to the local medical practice or organizational factors. Results The prioritization process was conducted using the same tool applied for HTA appraisal with the additional criterion that the HTR could facilitate the programme implementation. The research processes used in this HTR included: i) systematic review of the literature, ii) hospital database search (efficacy and resource utilization), iii) perceptions of healthcare teams and patients. HTR outputs consist of specific recommendations on implementation barriers and methods to monitor the impacts of the programme. Conclusions In this evolving field, sharing lessons from HTR methods provides information to develop standard adaptable processes to different contexts. Hence, this work applies HTR to a healthcare programme while most of the literature focuses on the HTR processes on drug and interventional medicine disinvestment. These elements represented HTR methods used from prioritization appraisal, research processes for evaluation and outputs used to plan the implementation and finally monitoring from a regional HTA unit. It also showcases that HTR being conducted as a structured evidence-based assessment adds value to a healthcare programme and could also facilitate its implementation.

Abstract Number: 531 Abstract Title: Quality Of Life Of Patients With Long-Chain Fatty Acid Oxidation Disorders Before And During Treatment With Triheptanoin Abstract Status: Accepted Poster Presentation Author Name: Eliza Kruger ([email protected]), Deborah Marsden, Arielle Bensimon, Erin Cook, Eli Orvis, Hongbo Yang, Jill Mayhew, Nina Thomas Introduction Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of serious diseases in which patients are at risk of metabolic decompensation, resulting in cardiomyopathy, hypoglycemia and rhabdomyolysis and premature mortality. In addition, LC-FAOD are a burdensome disease that adversely effects quality of life (QoL) via symptoms of muscle pain, fatigue, and a difficult diet. Previous studies have reported improvements in QoL during treatment with triheptanoin as measured by short form (SF) instruments. This study sought to convert the QoL measure into a utility value for a sample of patients with LC-FAOD at baseline and 78 weeks during treatment with triheptanoin. Methods In an open-label Phase 2 study of triheptanoin (UX007-CL201, NCT01886378), caregivers of patients (n=9/23 enrolled) or patients aged 18+ years (n=6/6 enrolled) completed the SF-10 or the SF-12v2, respectively. Component summary scores at baseline and 78-week during treatment period were converted to EuroQol-Five Dimension (EQ-5D) utility (with zero representing death and 1.0 perfect health) using a published conversion algorithm (Lawrence and Fleishman 2004). Generalized linear mixed-effects models with individual-level random effects were used to estimate the utility values. Results At baseline, patients’ utility was estimated to be 0.365 (standard error [SE]=0.090) compared with 0.629 (SE=0.072) 78-weeks during treatment, a significant improvement (p=0.0073). In a sensitivity analysis using SF-12v2 data only (that is, only adult patients), utility estimates were 0.498 (SE=0.084) at baseline versus 0.690 (SE=0.068) during treatment (p=0.0499). No patients had a major clinical event during the SF instrument recall period, indicating the benefit was driven by day-to-day improvement in QoL. Conclusions Treatment with triheptanoin resulted in a substantial improvement in daily QoL for patients with LC- FAOD. Limitations of this study include that the estimation of utilities is from a single-arm study with small sample sizes and that the assessment of utility was based on a conversion algorithm rather than direct measurement. Nevertheless, results indicate significant improvement in QoL for patients treated with triheptanoin.

Abstract Number: 538 Abstract Title: Burden Of Disease For Patients With Acute Myocardial Infarction Combining Dyslipidemia In China Abstract Status: Accepted Poster Presentation Author Name: Mengran Zhang, Hongchao Li, Aixia Ma, Pingyu Chen ([email protected]) Introduction Prevalence of dyslipidemia in Chinese adults is increasing rapidly. Dyslipidemia is one of the most important risk factors for acute myocardial infarction (AMI), which represents a serious disease burden to the country. However, there is no published research on the costs of Chinese patients diagnosed with AMI combining dyslipidemia. This study aimed to report key findings of the disease burden in China, including direct medical costs and direct non-medical costs. Methods Six hospitals from different geographic areas were selected in China for data collection. Patients who were hospitalized due to AMI combining dyslipidemia from January 1 2016 to December 31 2016 in the six sites were enrolled. Direct medical costs including inpatient and outpatient costs were extracted through electronic medical records; medical costs occurred in other healthcare institutions and direct non-medical costs were collected by a face-to-face questionnaire survey. Results were analyzed with descriptive statistics. Results Data of 900 patients were analyzed. There were more males (78.40%) than females. The mean age was 62.1 (SD: 11.5). The times of inpatient and outpatient per year were 0.57 and 8.67, respectively. Medium direct medical costs and medium direct non-medical costs were 31,440 RMB (Interquartile range (IQR): 21,533-48,202) (4,443 USD: 3043-6812) and 665 RMB (IQR: 351-1328) (94 USD: 50-188), respectively; while corresponding medium indirect costs per year were 659 RMB (IQR: 226-1579) (93 USD: 32-223). Conclusions This is the first study comprehensively analyzing the disease burden of patients diagnosed with AMI combining dyslipidemia in China. The results suggested that the medical cost of this population is still high. Hospitalization cost accounted for 81 percent of the total cost, which was around 1.3 times of the annual per capita disposable income over the same period. Therefore, the importance of providing effective clinical management as well as dyslipidemia prevention and control intervention should be highlighted, especially for middle-aged and elderly males with dyslipidemia.

Abstract Number: 548 Abstract Title: The Design Of Long-term Care Insurance Payment: Base On Pilot Practice In Jingmen Abstract Status: Accepted Poster Presentation Author Name: Tiantian Du, Junting Yang, Ying Li, Meng Zhang, Yuehua Liu ([email protected]) Introduction With the aging of population, miniaturization of family size and changes of diseases spectrum, the demand for long-term care of Chinese elderly is increasing, which is challenging the existing long-term care system. China is currently carrying out pilot work for a long-term care insurance system, and Jingmen is one of the pilot cities, however more detailed research on payment is needed. Therefore, this paper draws on case-mixed-adjusted prospective payment system to provide designs for long-term care insurance in pilot cities. Methods Adopting a case analysis method, this paper focuses on system for payment of Skilled Nursing Facility under Part A of the Medicare program—Patient Driven Payment Model, and discusses the implementation plan of a long-term care insurance in Jingmen City from the perspectives of payment methods, payment grouping and payment standards. Results Currently Jingmen adopts per-diem payment for long-term care insurance, so it is necessary to establish a payment based on population characteristics and demands. So, the patients should be classified into a group for each of the five case-mix adjusted components: physical therapy, occupational therapy, speech therapy, nursing and non-therapy ancillary. In addition, this payment model also includes a “variable per diem adjustment” to account for the changes in patient costs more accurately. Conclusions The theoretical system of a long-term care insurance payment method is developed, and a localization plan for case-mixed-adjusted prospective payment system for long-term care insurance is provided. Therefore, Jingmen long-term care insurance payment should adopt \"case-mixed adjustment\", strengthening the relationship between individual clinical characteristics and payment.

Abstract Number: 553 Abstract Title: Study On The Utilization Of Medical Resources In Patients With Malignant Tumors in China Abstract Status: Accepted Poster Presentation Author Name: Yuehua Liu, Tiantian Du, Fan Zhang, Kun Zhao ([email protected]) Introduction Malignant tumors have become a major public health problem and their treatment cost is increasing rapidly in China, but treatment aimed at healing diseases or extending patients’ life. There is little empirical research on utilization of healthcare resources of terminally ill cancer patients. In order to explore the optimal treatment decision for patients and provide information for relevant decision makers, this study analyzed the consumption status of medical resources in patients with cancer during the whole treatment period, and the current medical resource utilization efficiency in different levels of hospital for end-stage cancer patients. Methods This study was based on the clinical treatment and payment data of 2,536 cases of patients with lung cancer from the medical insurance database during the period of 2007 to 2014 in Hubei province. We retrospectively analyzed patients’ medical expenditure and utilization of different medical resources during their whole treatment period as well as at the end stage. Results The per capita inpatient expenditures of patients under 50 years old was 193,000 CNY (27,451 USD), while that of the patients over 70 years were 80,000-90,000 CNY (11,379-12,802 USD). Secondly, the medical expenditures spent during the last 6 months of life accounted for 66.1 percent of the total expenditures. Lastly, the medical expenditure spent in tertiary hospitals accounted for 95.3 percent of the total expenditure, and the expenditure was 14,200±17,030 CNY (2,019.82± 2,422.36 USD) per visit. Conclusions Population aging is not the only factor causing the rise of medical expenditure. The unclear objectives of treatment and the reverse of medical resource allocation are also important factors to boost the growth of medical expenditure. It is necessary to improve the healthcare insurance payment system, strengthen the capacity of primary medical institutions, and develop the palliative care system in China.

Abstract Number: 554 Abstract Title: Quality Assessment Of Clinical Practice Guidelines On Chronic Obstructive Pulmonary Disease Based On The Appraisal Of Guidelines For Research And Evaluation II (AGREE II) Instrument Abstract Status: Accepted Poster Presentation Author Name: Qian Xu, Kun Zhao, Cheng A Xin Duan, DanDan Ai, BinYan Sui ([email protected]) Introduction The scientific application of clinical evidence-based guidelines can reduce the variability of clinical practice, and standardize clinical diagnosis and treatment pathways. At present, many evidence-based guidelines on Chronic Obstructive Pulmonary Disease (COPD) prevention have been issued in countries around the world, but the procedures and evaluation strategies developed by different guidelines are not the same. This study aimed to evaluate the quality of published clinical practice guidelines (CPGs) relating to COPD using the Appraisal of Guidelines for Research and Evaluation II (AGREE II) instrument. Methods Databases were systematically searched PubMed, EMBASE, Wan Fang, and CNKI as well as guidelines websites on COPD prevention and treatment. The search period was from inception of the database up to May 2019. The inclusion criteria for this study are as follows: (i) published and in accordance with the definition of the practice guidelines; (ii)the main target population is COPD patients with the diagnostic criteria of the 2019 edition of the global initiative for COPD (GOLD), and the content of the guideline is related to the prevention and treatment practice of COPD; (ii) the same guide is included in the latest updated version; (iv) the published language is English or Chinese. Guidelines that met these inclusion criteria were evaluated for the quality of the AGREE II guidelines. Then, a descriptive analysis was made of the consensus that exists in the guidelines. Results A total of fifteen guidelines/Consensuses Statements were included in the study. Two guidelines were assessed as recommended, eleven guidelines were assessed as recommended with modifications and two guidelines were not recommended. The mean scores of the included guidelines in the six domains (scope and purpose, personnel involved in guideline development, rigor of development, clarity, applicability, independence) were 90 percent, 72 percent, 49 percent, 96 percent, 60 percent, 69 percent, respectively. Thus, the study identified a consensus that disease risk factors and recommended interventions were mentioned in the guidelines, and that they comprehensively evaluated the quality of guideline reporting to provide reference for standardizing the development of practice guidelines for COPD in China. Conclusions The overall methodological quality of COPD CPGs should be improved. The key recommended areas for improvement include standardization of guideline report writing and synthesis of the latest and best evidence, to develop CPGs for COPD to improve the quality of clinical diagnosis and treatment for COPD.

Abstract Number: 574 Abstract Title: Cost-Effectiveness Of Newborn Screening Of Primary Immunodeficiency Diseases: A Systematic Review And Economic Evaluation Abstract Status: Accepted Poster Presentation Author Name: Junting Yang, Fan Zhang, Zhao Liu, Ning Yue, Yuehua Liu ([email protected]) Introduction Primary immunodeficiency diseases (PIDs) are a heterogeneous group of over 200 disorders with defects in the function and/or development of the immune system. Although early screening is imperative for improving therapeutic efficiency and preventing disease-associated morbidity, its widespread use has been limited, owing to the low incidence of PIDs. It is particularly important to evaluate the cost- effectiveness of PIDs screening for newborns. The aim of this study was to provide an overview of the existing cost-effectiveness evidence on newborn screening of PIDs and to provide reference for decision- makers in China and other developing countries. Methods We conducted a systematic review using three electronic databases (PubMed, CNKI, and CSPD) of cost and cost-effectiveness studies of PIDs screening published during 2000-2019. Two reviewers independently searched databases and screened titles, abstracts and full texts; a third reviewer resolved disputes when necessary. The initial search returned 124 references, of which 10 full articles were included in the review. Five of the studies conducted analyses using model-based techniques. Results Severe combined immunodeficiency (SCID) was the predominantly studied condition (80%). Most studies (70%) examined the T-cell receptor excision circle (TREC) assay. A healthcare system’s perspective was commonly used (50%) for cost calculations, and most studies (50%) were US-based. The majority (67%) of the studies found the TREC assay an effective screening tool for SCID, but the incremental cost-effectiveness ratio (ICER) varied across screening test specificity and disease incidence. Conclusions Evidence from the published literature demonstrated that newborn screening for PIDs generally appeared to be cost-effective, and most importantly, it is lifesaving and allows children with PIDs an opportunity to live a healthier life. However, the type of PIDs included in this study were limited and most studies were done in developed countries whose health systems are different from low-/middle- income countries (LMIC). Further research is required to identify the cost-effectiveness of PIDs screening both in developed and developing countries.

Abstract Number: 581 Abstract Title: Catastrophic Costs Of Multidrug-Resistant Tuberculosis: Estimation Based On The Cost Of Treatment In China Abstract Status: Accepted Poster Presentation Author Name: Lijun Shen, Shangshang Gu, Fan Zhang ([email protected]), Zhao Liu, Yuehua Liu Introduction China bears a considerably high burden of multidrug-resistant tuberculosis (MDR-TB). Second-line anti- TB drugs are urgently needed yet domestic MDR-TB drugs are expensive and lack policy support. Patients’ living conditions are closely related to the drug affordability. The national TB prevention programs should play a critical role. The purpose of this study is to measure the cost of treating MDR-TB patients under different treatment schemes and price sources. The results of this study are expected to inform the relevant drug protection policies and provide inputs for further cost-effectiveness analyses. Methods Based on the treatment plan of China's Multidrug-Resistant Pulmonary Tuberculosis Clinical Path (2012 edition) and the World Health Organization (WHO) Drug-Resistant Tuberculosis Treatment Guide (2018 edition), the treatment costs of MDR-TB were measured under different scenarios. Catastrophic health expenditure was then calculated if the treatment cost exceeds 40 percent of the household's non- subsistence income. National, rural and disposable income per capita in 2018, were used to represent Chinese patients’ affordability. Results Under varied treatment schemes and market price sources in China, the total costs for MDR-TB patients range from 19,401 to 126,703 CNY [2,853 to 18,633 USD] per person. Under current prices, all treatment schemes recommended by the WHO will incur catastrophic costs for Chinese MDR-TB patients. Significant differences were found between rural and urban areas as 52.8 percent of the treatment listed in the 2012 China Guideline would lead to catastrophic cost for rural patients but not urban ones. Conclusions Our study concludes that the domestic drugs are more expensive than the international purchase price and the treatment of MDR-TB imposes substantial economic burden on patients, especially in the rural areas. The results of the study also indicate that it is urgent for the state to emphasize government responsibility and initiate centralized procurement for price negotiations to reduce the market price of MDR-TB drugs. The urban-rural gap should also be addressed in the design of future policies to ensure the drug affordability for all patients in need.

Abstract Number: 585 Abstract Title: A New Hope For Breast Cancer Survivors: Early Assessment Of A Breast Cancer Vaccine Abstract Status: Accepted Poster Presentation Author Name: Norrina Jamaluddin ([email protected]), Izzuna Mudla Mohamed Ghazali, Junainah Sabirin Introduction Breast cancer is the most frequent cancer among women globally, impacting 2.1 million women each year, causing the greatest number of cancer-related deaths among women. In Malaysia, the new cases of breast cancer comprised of 32.7 percent of all new cancer cases in women as reported by The International Agency for Research on Cancer (IARC). The recurrence rate was about 16.4 percent post-mastectomy. This early assessment is to evaluate the effectiveness and safety of a breast cancer vaccine. Methods A systematic review was conducted. Searches were done through PubMed, Medline and ClinicalTrial.gov. The articles were selected based on inclusion and exclusion criteria and appraised using Critical Appraisal Skills Programme (CASP) checklist. More than twenty cancer vaccines under development were identified. Results The most advanced breast cancer vaccine is Nelipepimut-S (NPS). In a Phase 2b clinical trial, improvement disease-free survival (DFS) in the NPS group was 89.8 percent. The DFS rate in the NPS group was 92.6 percent for triple negative patients. Median DFS in the NPS group [hazard ratio (HR): 0.26 (95% confidence interval, Cl: 0.08-0.81)] showed a significant difference. A projection study showed the cost-effectiveness will be 90 percent success if the cost less than USD1,000 per patient. No safety issues were reported. Conclusions Early assessment showed potential benefit in patient with triple-negative breast cancer (TNBC). However, further research is required to ensure its efficacy, safety, and cost-effectiveness.

Abstract Number: 597 Abstract Title: The Model Approach For Evaluation Of The Economic Burden For Hemophilia A In China Abstract Status: Accepted Poster Presentation Author Name: Renchi Yang, Yongqiang Zhao, Feng Xue, Jing Sun, Chao Liu, Xuerong Ran ([email protected]), Na Yuan, Chang Xing, Ranran Wang, Ziyi Lin, Tengbin Xiong Introduction Hemophilia was included in the first National Rare Diseases List in China and hemophilia A (HA) is the most common type (about 80%) which could impair patients' quality of life. Most patients do not have regular prophylaxis as per the clinical guidelines recommendation, and bleeding is not well controlled, which causes a heavy economic burden. There are few studies to illustrate the evaluation model and economic burden of HA in China. Therefore, the aim of this study was to map out the economic burden of HA including direct and indirect costs to attract the attention of stakeholders. Methods To fully estimate the costs for HA, the societal perspective was adapted. We identified diagnostic pathways and treatment patterns of HA based on clinical guidelines and practices in China. A prevalence-based disease burden computing framework was developed following the principle of China guidelines for pharmacoeconomic evaluations. The data for each parameter was retrieved through literature review, key clinical expert interviews and patient interviews, which reflected the real-world clinical practice. Results A “SMART” HA Model was developed as a specific, measurable, adjustable, rational and timely model to estimate direct and indirect costs for patient with Hemophilia A, including costs of drug, test, surgery, transportation, accommodation and productivity loss. In addition, it enabled estimating cost of patients with different demographic and clinical characteristics, such as age, severity of disease, inhibitor status, and treatment patterns. The inputs of the SMART HA Model could be adjusted in a timely way to reflect the new situation when the treatment pattern changes. Conclusions The SMART HA Model comprehensively evaluated the economic burden of patients with Hemophilia A. It reflected real-world clinical diagnosis and treatment in China. The corresponding economic cost can be obtained by putting the latest data into the model, which could help policymakers to do reimbursement decision-making more scientifically and provide evidence for allocating healthcare resource more reasonably. Additionally, this model provides the opportunity for HA patients and healthcare providers to understand the costs associated with the disease and to promote standardized prevention and treatment regimens.

Abstract Number: 612 Abstract Title: Use of Rapid Review Methods In Health Technology Assessment For Central Procurement Decisions: Experience In Ukraine Abstract Status: Accepted Poster Presentation Author Name: Oresta Piniazhko, Kachveci Rabia, Dumenko Tetyana, Mariya Leleka ([email protected]), Alona Masheiko, Valeriia Serediuk Introduction The Health Technology Assessment (HTA) Department of the “State Expert Center of the Ministry of Health of Ukraine” was established in 2019 for conducting assessments and development of recommendations for informing decisions of the Ukrainian Ministry of Health regarding financing of the health technologies. Since 2015, procurement of medicines by the state budget in Ukraine was carried out through international specialized organizations. The central procurement program covers forty-one programs for different disease areas. The list of medicines and medical devices purchased were based of procurement agreements with specialized organizations approved by Cabinet of Ministers Decree of Ukraine № 255 of 13 March 2019. Methods Descriptive analysis of methodological processes around rapid review methods in HTA used for the procedure of central procurements of medicines. In the process of conducting rapid assessment we use a modern tool based on the HTA Core Model® for Rapid Relative Effectiveness Assessments version (V3.0). HTA Guidance in Ukraine was developed in 2017. Results The list of government programs, for which rapid review methods in HTA have been conducted, included following diseases: adult and pediatric oncology, juvenile rheumatoid arthritis, hemophilia, and orphan diseases. Reports include an overview of comparative efficacy, effectiveness and safety, as well as analysis of reports from HTA agencies and thresholds, budget impact for informing decisions for development of the central procurement programs in 2019. The HTA Department conducted more than forty reviews in less than 6 months. Conclusions Use of rapid review methods in HTA in Ukraine for informing decisions for the central procurement programs of medicines is the perspective for rational resource allocation and spending. This increases and improves patients’ access to effective, safe and cost-effective medicines.

Abstract Number: 613 Abstract Title: The Сost Analysis Of Subcutaneous And Intravenous Dosage Forms For Systemic Juvenile Idiopathic Arthritis Treatment In Ukraine Abstract Status: Accepted Poster Presentation Author Name: Alona Masheiko ([email protected]), Oresta Piniazhko, Iryna Romanenko, Mariya Leleka, Valeriia Serediuk Introduction The Health Technology Assessment (HTA) Department of the “State Expert Center of the Ministry of Health (MoH) of Ukraine” was established in 2019 for conducting assessments and development of recommendations for informing decisions for the Ministry of Health of Ukraine (MoH) regarding the financing of health technologies based on HTA. The study aimed to conduct cost analysis and compare the treatment costs of two available dosage forms of tocilizumab for subcutaneous and intravenous administration for systemic juvenile idiopathic arthritis in Ukraine. Currently there is a central procurement program financed by the state budget with pharmaceuticals provided to patients with juvenile idiopathic arthritis approved by the Cabinet of Ministers Decree of Ukraine №255 dated 13.03.2019. Methods The cost analysis was carried out for tocilizumab over a 1-year horizon per one patient. The analysis included drug manufacturers' prices from the registry of the MoH dated 19.09.2019. The annual number of tocilizumab vials for intravenous infusion for each weight category of patients is approved by the order MoH №334 from 14 February 2019. The direct medical costs were included in the analysis omitting cost of administration. Results The direct medical costs of treatment with intravenous tocilizumab per one patient for one budgetary year ranged from between USD7,563.83 and USD30,255.30 depending on patient’s weight that was in the range of 10-80 kg. The direct medical costs of treatment per one patient for one year with subcutaneous tocilizumab was USD7,782.40 for patients < 30 kg and USD15,564.80 for patients ≥ 30 kg. Conclusions The introduction of subcutaneous tocilizumab can potentially lead to cost savings on average USD 4,041.97 (34.2%) for patients < 30 kg and USD 5,245.82 (25.2%) for patients ≥ 30 kg per one patient for 1-year treatment compared to the intravenous route. Intravenous tocilizumab has an economic advantage over the subcutaneous route solely for the pediatric population of certain weight categories. The cost of intravenous tocilizumab was USD 218.58 lower (2.9%) for patients ≤ 13 kg and USD 2,320.25 lower (17.5%) for patients with weight 31-35 kg, compared to the cost of subcutaneous tocilizumab.