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Improved Methods and Actionable Tools for Enhancing Health Technology Assessment

Periodic Reporting for period 1 - IMPACT HTA (Improved Methods and Actionable Tools for Enhancing Health Technology Assessment)

Berichtszeitraum: 2018-01-01 bis 2019-06-30

Improving the effectiveness and efficiency of health technology procurement through cross-country collaboration in areas such as Health Technology Assessment (HTA), constitutes a major policy objective across EU Member States. This applies both to methodological developments, and empirical applications. Often, the results of a specific study can be almost directly applied to other settings (e.g. RCTs and other clinical studies), but in economic evaluations a number of aspects must be customised to country-specific circumstances in order to attain external validity. Recently, there have been significant changes such that data on costs and health outcomes are now available from an increasing range of sources underscoring (a) the need for better data integration and evidence synthesis and (b) the strengthening of data generation processes for economic evaluations and improvements in methodological quality. Against this background, the overall objectives of IMPACT HTA are to:
• Contribute to understanding the variations in costs & health outcomes within and across countries through methodological improvements;
• Develop & disseminate innovative methodologies, toolkits and processes in areas such as extrapolation from RCT data, costing, outcomes measurement, value assessment, real world evidence, aiming to aid decision-making and improve efficiency in resource allocation;
• Develop and disseminate tools facilitating cross-country collaboration across EU Member States, HTA agencies, professionals and other stakeholders.
WP1 & WP12: Prepared & submitted detailed data management plan and collected & continue to collect all relevant paperwork related to project activities (WP1); carried out numerous dissemination and communication activities to increase project visibility and reach and engage potential benefitting stakeholders (WP12).
WP2: (a) Conducted scoping study of literature on extrapolation techniques, including brief internal summary paper; (b) relating to the development of Excel-based platform to allow direct modelling of CEA by HTAs: (i) completed specification of new platform (DICE model); (ii) completed first case-study (conversion of existing HTA model); (iii) developed survey of HTA experts’ preferences for the platform; and (c) made numerous presentations, workshops and trainings using platform’s current version.
WP3: (a) A scoping review of literature and documentation on accounting and costing practices and a related article; (b) built and validated draft of the European Health Care Costs Database (EU HCCD), and health costs from other sources; and (c) conducted one online workshop to discuss methodological and technical aspects of EU HCCD.
WP4: (a) Revised methodological aspects applied to the identification, measurement and valuation of social costs in economic evaluations; (b) Alzheimer’s disease case study (to be published) to quantify ways of incorporating social perspective in economic analysis; (c) ongoing case studies & synergies with WP5 & WP8; (d) developing conceptual framework of database platform and created a draft database on unit costs relating to lost work time and the value of informal & formal care time.
WP5: Focused on preparation of methodology and tools, including questionnaire instrument & online survey: (a) completed translation/adaptation process of necessary language versions of EQ-5D-Y; (b) prepared experimental design of discrete choice experiment (DCE) defining attribute combinations and the levels of described health states was established and the different designs (patient and adult/adolescent survey); (c) nearly completed programming of adult/adolescent survey; (d) sub-design of EQ-5D-5L valuation studies was adopted in patient survey, to allow comparisons with existing data.
WP6: Conducting large-scale meta-epidemiological review: (a) developed (and published on online repository) detailed protocol for empirical investigation of discrepancies in treatment effects between randomised & non-randomised studies; (b) compiled dataset of clinical topics with matching randomised and non-randomised studies using various analytical techniques (to be analysed next).
WP7: (a) Developed model analysing determinants of HTA coverage recommendations across settings; (b) completed study on orphan drugs on determinants of HTA recommendations and the role & relative importance of criteria to inform coverage; (c) published a systematic literature review (SLR) of the methodological quality of multi-criteria decision analysis (MCDA) in HTA applications; (d) created & tested empirically a framework to identify stakeholders engaging in HTA; and (e) conducted research to (i) identify key features for a novel value framework and (ii) how a web-Delphi can be designed to promote participant consensus and interaction participants.
WP8: Conducted three literature reviews, which led to the development of two tools providing material, insights and evidence to fulfil the objectives of the WP: (a) online survey (investigating the influence of contextual factors on the implementation of technology and on clinical evidence-based decision-making; and (b) a semi-structured interview guide (to enable insights to be captured through direct interviews of senior staff).
WP9: (a) Designed basic framework integrating the combined effects on tax revenues and income decreases due to health conditions on patients, enterprises and employers; (b) conducted literature review to identify methods (human capital approach and friction costs methods) and their contribution to support the implementation of an integrated framework; and (c) completed tools to collect data from employers and diabetes patients.
WP10: (a) Completed vignettes of HTA appraisal processes for rare disease (RD) treatments; (b) completed an SLR of mapping approaches to derive health state utility values (HSUVs) from disease specific PROMs in RDs; (c) developed a database of Italian managed entry agreements (MEA) relating to OMPs to compare financial- vs outcome-based MEAs; (d) undertook ethnographic observation of products going through appraisal in Scottish setting (SMC) with follow-up interviews to explore how evidence is judged in deliberative settings compared with stated processes.
WP11: (a) Completed SLR to identify and categorise technologies offering prospects for efficiency gains outside incremental innovations captured in the northeast quadrant of the cost-effectiveness plane, (identified 94 decrementally cost effective (d-CE) interventions); (b) completed research on possible publication bias due to lack of interest in health technologies representing cost and quality reduction profiles.
Through advanced evidence generation & evaluation techniques and multidisciplinary approaches, IMPACT HTA will create impactful methodological frameworks and tools; it will (a) deliver validated new approaches for collection and analysis of data for health economic evaluation; (b) produce validated new approaches for data integration from all relevant sources, facilitating informative and continuous assessment of health interventions and health systems; and (c) make available validated new indicators, measures and tools to be used by decision-makers for resource allocation purposes, that are patient-specific, efficient and sustainable. Project outputs will be useful to HTA bodies, guideline developers, patient organisations, clinical experts, academic groups, and industry.