CORDIS - Résultats de la recherche de l’UE

Improved Methods and Actionable Tools for Enhancing Health Technology Assessment

Periodic Reporting for period 2 - IMPACT HTA (Improved Methods and Actionable Tools for Enhancing Health Technology Assessment)

Période du rapport: 2019-07-01 au 2021-06-30

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. Recently, there have been significant changes such that costs and health outcomes data are now available from a 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. The IMPACT HTA objectives 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 randomised controlled trials 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.
IMPACT HTA achievements and impacts are outlined below.
1. A tool to combine and use randomised clinical trials and observational/registry data in economic evaluation: HTA staff can now undertake quick, robust cost-effectiveness evaluations using local data through the freely available Discretely Integrated Condition-Event (DICE) simulation model which is easily modified for local adaptation, enabling models sharing across agencies and across appraisals in a given therapeutic area.
2. A costing methodology and a database of unit costs in health and social care: The first European common database of health & social care unit costs in nine countries is easily accessible and can be used to feed into health-economic evaluations by transferring economic evaluation analyses and models across countries. It saves researchers’ time in searching costs, allows cross-country comparisons and provides an understanding of costs variations across countries.
3. Health preferences of different sub-population groups using EQ-5D: HTA agencies should consider the differences between patient and general population health state preferences as the choice of value set can lead to different decisions. Where patient preferences are favoured, it is feasible to combine patient preferences and general population-based value sets. Patient preferences can be used for treatment decisions in clinical settings and in organising health care processes. Value sets derived from the generic youth-specific EQ-5D-Y can be used in economic evaluations of paediatric treatments in the countries in which they were developed.
4. Methodological guidance on the analysis and interpretation of non-randomised studies to inform health economic evaluations: IMPACT HTA has developed a set of evidence-based recommendations for the use of non-randomised studies to estimate treatment effects in HTA, including the need to justify their use to prospectively plan such studies. They should consider possible biases and how to address them and should transparently report the methods used. HTA systems need to be strengthened to allow effective appraisal of non-randomised evidence.
5. Methodological tools using multi-criteria value methods for HTA decision-making, including the creation of the IMPACT-HTA Value Framework: Recognising that the relative value of a health care technology spans across a multi-dimensional domain going beyond the classical concept of “economic efficiency” as measured by incremental cost effectiveness ratios, two distinct contributions are made in developing and testing the IMPACT-HTA Value Framework: 1) a model of the parameters associated with HTA coverage recommendations has been developed and tested empirically, encompassing product- and country-specific variables, regulatory variables, critical clinical and economic evidence parameters, the role of evidence uncertainties and the importance of social value judgements. 2) the IMPACT-HTA Value Framework captures the value concerns of decision-makers and other stakeholder groups within a common framework to evaluate new medicines within the context of HTA, reflecting on what is important for HTA agency decision-makers and the broader stakeholder community.
6. Analysis of economic evaluation methods for hospital-based assessment: Hospital performance is multi-faceted and multiple hospital contextual factors can affect the adoption of health technologies. 2 toolkits were developed: the 1st assesses the overall hospital performance; and the 2nd provides a methodology to assess how contextual factors affect the implementation of a technology. They are supported by a decision model that identifies technologies of equal efficacy and safety but which are subject to clinical variation and assesses different interventions with the goal of improving the performance and efficiency of healthcare delivery.
7. Methods for measuring the fiscal impact of new healthcare interventions. The fiscal impact of productivity losses provides an additional dimension characterizing the value for money of a medical intervention. There is a strong recommendation for fiscal impact not to be used to inform rationing policies or perform cross-group comparisons among different socio-economic categories as it can lead to discrimination against the elderly and low-income subgroups.
8. Appraisal of Orphan Medicinal Products (OMPs): An appraisal framework for OMPs has been created that ensures a fair, lenient, flexible and consistent appraisal, beyond clinical and cost effectiveness in order to overcome the small patients number and the technology’s high cost. The framework is supported by recommendations and guidance for the appropriate use of patient-reported outcomes and outcomes-based managed entry agreements for OMPs. The framework can be used by individual health systems or for collaborative HTA initiatives.
9. Behavioural considerations in the adoption of decrementally cost-effective technologies (d-CEIs): Decremental cost-effective interventions imply cost-savings at the price of a reduction in efficacy compared to usual care. Some of these interventions can be highly effective but are much less studied compared to interventions implying both higher costs and efficacy gains. A discrete choice experiment was designed to investigate under which conditions policymakers may be willing to adopt d-CEIs. A ‘decision-tree’ and a Political Economy Report are delivered to address the broader economic, ethical and political aspects associated with the assessment and adoption of d-CEIs. These outputs provide policymakers with guidance on whether and under what circumstances to d-CEIs.
IMPACT HTA created impactful methodological frameworks and tools; it (a) delivered validated new approaches for collection and analysis of data for health economic evaluation, (b) produced validated new approaches for data integration from all relevant sources and (c) made available validated new indicators, measures and tools for resource allocation purposes, that are patient-specific, efficient and sustainable. Project outputs are useful to HTA bodies, guideline developers, patient organisations, clinical experts, academic groups, and industry.
The 10 research WPs of IMPACT HTA