SUSTAIN-HTA – D1.7 – First Yearly Overview HTA Methods

© Copyright 2025 SUSTAIN-HTA Consortium Lead contributor Saif Elayan (1 – UU), Wim Goettsch (1 – UU) and Karen Facey (1 – UU) Other contributors Shane Collins (15 – NICE), Dalia Dawoud (15 – NICE), Carlo Federici (2 – UB), Zoe Garrett (15 – NICE), Andrea Greco (2 – UB), Wija Oortwijn (5 – RUMC), Peter Rijnbeek (3 – EMC), Fatima Salih (15 – NICE), and Liza van Mun (5 – RUMC). Work Package WP1 – Identification, Synthesis & Prioritisation Due date 31/03/2025 Delivery date 31/03/2025 Deliverable type R Dissemination level PU – Public 101136318 – SUSTAIN-HTA Support Utilisation of Sustainable and TAilored INnovative methods for HTA D1.7 – First yearly overview HTA methods D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 2/16 © Copyright 2025 SUSTAIN-HTA Consortium TABLE OF CONTENTS DOCUMENT HISTORY .................................................................................................................. 3 DEFINITIONS ................................................................................................................................. 4 LIST OF ABBREVIATIONS ............................................................................................................. 5 EXECUTIVE SUMMARY ................................................................................................................ 6 1 Introduction .............................................................................................................................. 7 2 Methods ................................................................................................................................... 8 2.1 Identification of Completed EU-Funded Projects ............................................................... 8 2.2 Inclusion and Exclusion Criteria ........................................................................................ 8 2.3 Data Extraction and Database Development .................................................................... 9 3 Results..................................................................................................................................... 9 3.1 Overview of the Database ............................................................................................... 10 4 Discussion ............................................................................................................................. 11 5 Next Steps ............................................................................................................................. 13 References ................................................................................................................................... 15 D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 3/16 © Copyright 2025 SUSTAIN-HTA Consortium DOCUMENT HISTORY Version Date Description V1.0 6 March 2025 First Draft V2.0 11 March 2025 Second Draft for Steering Committee Review V3.0 31 March 2025 Final Version D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 4/16 © Copyright 2025 SUSTAIN-HTA Consortium DEFINITIONS Partners of the SUSTAIN-HTA Consortium are referred to herein according to the following codes: 1. UU Universiteit Utrecht (Netherlands) 2. UB Universita Commerciale Luigi Bocconi (Italy) 3. EMC Erasmus Universitair Medisch Centrum Rotterdam (Netherlands) 4. ZIN Zorginstituut Nederland (Netherlands) 5. RUMC Stichting Radboud Universitair Medisch Centrum (Netherlands) 6. SRI Syreon Kutato Intezet Korlatolt Felelossegu Tarsasag (Hungary) 7. OSTEBA Fundacion Vasca de Innovacion e Investigacion Sanitarias (Spain) 8. GetReal I GetReal Institute (Netherlands) 9. SYNAPSE Research Management Partners (Spain) 10. NIPN Nemzeti Nepegészségügyi és Gyógyszerészeti Központ (Hungary) 11. AGE.NA.S Agenzia Nazionale per i Servizi Sanitari Regionali (Italy) 12. AQUAS Agencia de Qualitat i Avaluació Sanitaries de Catalunya (Spain) 13. NOMA Statens Legemiddelverk (Norway) 14. UCSC Universita Cattolica del Sacro Cuore (Italy) 15. NICE National Institute for Health and Care Excellence (United Kingdom) Grant agreement The agreement signed between the beneficiaries and the European Health and Digital Executive Agency for the undertaking of the SUSTAIN-HTA project (101136318). Project The sum of all activities carried out in the framework of the Grant Agreement. Consortium The SUSTAIN-HTA Consortium, comprising the above-mentioned legal entities. Consortium agreement Agreement concluded amongst SUSTAIN-HTA partners for the implementation of the Grant Agreement. Such an agreement shall not affect the parties’ obligations to the Community and/or to one another arising from the Grant Agreement. D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 5/16 © Copyright 2025 SUSTAIN-HTA Consortium LIST OF ABBREVIATIONS Acronym Description CSA Coordination and Support Action EU European Union H2020 Horizon 2020 HRQoL Health-Related Quality of Life HTA Health Technology Assessment HTACG Member State Coordination Group on HTA IHI Innovative Health Initiative IMI Innovative Medicines Initiative RUM Resource Use Measurement RWD Real-World Data RWE Real-World Evidence SUSTAIN-HS SUSTAIN-HTA Horizon Scanning SUSTAIN-OBS SUSTAIN-HTA Methods Observatory SUSTAIN-SB SUSTAIN-HTA Sandbox Environment WP1 Work Package 1 D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 6/16 © Copyright 2025 SUSTAIN-HTA Consortium EXECUTIVE SUMMARY The European Union (EU) has made substantial investments in advancing health technology assessment (HTA), yet the adoption of innovative HTA methods and tools by HTA bodies remains limited. The reasons behind this limited uptake of methods and tools are not fully understood but may stem from insufficient dissemination, implementation barriers, concerns about applicability in HTA practice, or misalignment with the needs of HTA bodies. SUSTAIN-HTA aims to bridge this gap by assessing the methodological needs of European HTA bodies and mapping them to relevant innovative HTA methods. To this end, Work Package 1 is spearheading two key initiatives: 1) a horizon-scanning tool (SUSTAIN-HS) to monitor the evolving methodological needs of European HTA bodies (Task 1.3); and 2) a methods observatory (SUSTAIN-OBS) to identify and categorise innovative HTA methods and tools (Task 1.5). This report constitutes the initial phase of Task 1.5, presenting an inventory of HTA methods and tools developed in projects funded under Horizon Europe and its predecessor, Horizon 2020, as well as under the Innovative Health Initiative (IHI) and its predecessor, the Innovative Medicines Initiative (IMI). The inventory focuses specifically on projects completed within the past five years to ensure relevance to current HTA practices. A multi-step approach was employed to identify relevant research projects. Consortium members, drawing on their involvement in multiple EU-funded initiatives, reported projects known to them that had contributed to the development of HTA methods and tools. This internal effort was complemented by consultations with members of the Expert and Stakeholder forums. Project outputs were sourced from the EU Funding & Tenders Portal and official project websites. Predefined inclusion and exclusion criteria were applied to ensure the methodological relevance of selected outputs to HTA. The identified methods and tools were catalogued in an Excel-based database, capturing project-level details, methodand tool-specific attributes, and classification according to the SUSTAIN-HTA taxonomy developed in Task 1.2. The review identified five Horizon 2020 projects and two IMI-funded projects that contributed to the development of HTA methods. Of the 348 outputs (e.g., deliverables and publications) originating from these projects, 99 met the inclusion criteria, resulting in 55 distinct methods and tools. Among these, 78% were derived from the Horizon 2020 projects, while 22% were developed within the IMI initiatives. The methodological focus of the identified methods and tools varied, with 47% addressing clinical effectiveness, 35% relating to costs and economic evaluation, and the remainder covering organisational aspects, safety, and broader issues extending beyond HTA. These methods and tools spanned a range of methodological areas, including real-world evidence (35%), predictive modelling (13%), health-related quality of life (11%), costing and resource use (11%), and heterogeneity in treatment effects and cost-effectiveness (7%). Several of the identified methods and tools align with the methodological needs of European HTA bodies outlined in Task 1.3. This alignment underscores the potential of SUSTAIN-OBS as a structured and accessible resource, enabling HTA bodies to identify methods and tools that meet their needs and supporting the integration of innovative methods into real-world settings. Looking ahead, the observatory will be expanded to incorporate methods and tools emerging from ongoing Horizon Europe/2020 and IMI/IHI projects; EU projects funded under other schemes; HTA bodies; international HTA-related organisations; and peer-reviewed literature. An online tool will also be developed to enhance accessibility, allowing users to locate and retrieve information on relevant methods efficiently. Engagement with HTA bodies and the Expert Forum will continue to ensure the observatory remains a dynamic and evolving resource for methodological advancements in HTA. D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 7/16 © Copyright 2025 SUSTAIN-HTA Consortium 1 Introduction Health technology assessment (HTA) is ‘a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle’. This value may be assessed by examining the intended and unintended consequences of using a health technology compared to existing alternatives, across several dimensions including clinical effectiveness, safety, cost and economic implications, as well as ethical, social, cultural, legal, organisational, and environmental aspects (O’Rourke et al., 2020). The European Union (EU) has made considerable investments in HTA through various research projects aimed at advancing scientific methods. These investments are intended to enhance decision-making in healthcare by improving the evaluation of health technologies. However, despite the development of numerous innovative HTA methods by EU projects and other learned consortia and academia, their adoption by HTA bodies remains limited. The reasons for this limited uptake are not well understood, though several factors may contribute to the challenge. Potential explanations include insufficient visibility and dissemination of new methods and tools, practical barriers to implementation, methodological concerns regarding their applicability in HTA practice, or a mismatch between the methods/tools developed and the actual needs of HTA bodies. To address these challenges, SUSTAIN-HTA—a Horizon Europe Coordination and Support Action (CSA)—has been established to assess the methodological needs of European HTA bodies and align them with innovative HTA methods and tools, particularly those developed through EUfunded projects. By systematically identifying HTA bodies’ needs and mapping them to methodological innovations, SUSTAIN-HTA strives to facilitate the adoption of methods that address these needs, thereby improving the rigor, consistency, and impact of HTA practices across Europe. Work Package 1 (WP1) is dedicated to exploring these issues. Task 1.3 will establish a continuous horizon-scanning tool (SUSTAIN-HS) to identify and monitor the evolving methodological needs of European HTA bodies. In parallel, Task 1.5 will develop a methods observatory (SUSTAINOBS) to systematically identify and categorise innovative HTA methods and tools. In Task 1.6, the methodological needs identified through horizon scanning will be considered alongside the innovative methods catalogued in the observatory to prioritise those most relevant to HTA bodies. Subsequently, the prioritised methods will be tested for implementation as case studies within the SUSTAIN-HTA Sandbox Environment (SUSTAIN-SB). This report represents the first step in Task 1.5, focusing on identifying the methods and tools developed by EU-funded research projects that concluded within the past five years (i.e., between D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 8/16 © Copyright 2025 SUSTAIN-HTA Consortium 2020 and 2024). Specifically, the report focuses on projects funded under Horizon Europe and its predecessor Horizon 2020, or under the Innovative Health Initiative (IHI) and its predecessor, the Innovative Medicines Initiative (IMI). The goal is to provide a comprehensive inventory of these methods and tools, ensuring their visibility and accessibility to HTA bodies. In subsequent annual reports, this mapping exercise will be expanded to include ongoing Horizon Europe and IHI projects, EU projects funded under other schemes, as well as peer-reviewed literature and relevant outputs from HTA bodies, HTA bodies’ collaborations, HTA/payer collaborations, and international (professional) HTA-related organisations. This continuous, multi-source approach will help ensure that the observatory remains an up-to-date repository of methodological advancements in HTA. 2 Methods 2.1 Identification of Completed EU-Funded Projects The first step involved systematically identifying Horizon Europe/2020 and IMI/IHI projects that had produced methods or tools relevant to HTA. As mentioned earlier, the scope of this report was limited to projects that concluded within the past five years to ensure the relevance to current HTA practices. A multi-step approach was employed to identify these projects. First, during WP1 meetings, consortium members—drawing on their broad involvement in numerous EU-funded initiatives—reported projects known to them that had contributed to the development of HTA methodologies. This internal identification was complemented by input from members of the project’s Expert and Stakeholder forums, collected during forum meetings. This coordinated approach aimed to ensure a comprehensive mapping of relevant projects and to minimise the risk of omitting significant initiatives. 2.2 Inclusion and Exclusion Criteria Following the identification of relevant projects, a set of predefined inclusion and exclusion criteria was applied to assess the eligibility of their outputs (e.g., deliverables and publications) for inclusion in this assessment. These criteria were formulated to ensure that only outputs of methodological relevance and applicability to HTA were retained for analysis. • Inclusion Criteria  The output must present or describe a method or tool relevant to HTA.  The output must be accessible via the EU Funding & Tenders Portal or the official website of the project/initiative. D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 9/16 © Copyright 2025 SUSTAIN-HTA Consortium  For academic publications, the output must include a funding statement or other explicit reference linking it to the corresponding project/initiative. • Exclusion Criteria  Outputs related exclusively to project management, administrative reporting, or coordination activities.  Outputs addressing topics that do not concern methods or tools applicable to HTA. 2.3 Data Extraction and Database Development The data extraction process was designed to systematically collect and document information on identified HTA methods and tools. An Excel-based database was developed to serve as a structured repository for data collection and analysis. This database will be used as the background to the SUSTAIN-OBS. To ensure comprehensive and systematic documentation, the database captured both project-related information and details specific to the method or tool reported, which may be searched upon in SUSTAIN-OBS. Project-related information included the project name, funding source, and a summary of the project’s main achievements. Information related to the reported method or tool encompassed the type of technology to which it is applicable and a description of its purpose and characteristics. Additionally, links to project outputs in which the method or tool is documented were recorded, and supplementary information relevant to its application (e.g., country of application or clinical condition) was noted when relevant. The database was structured with a project-centric ordering to facilitate systematic data extraction, ensuring that each entry corresponded to a specific project. The reported methods and tools were also categorised according to the methodological domains and areas of HTA they address. The categorisation was underpinned by the SUSTAIN-HTA taxonomy and expanded to be in accordance with the HTA Core Model (Elayan et al., 2024; Lampe et al., 2009). To improve usability, the database was designed with integrated filtering functions, enabling dynamic reorganisation and targeted searches based on projectand method or tool-level characteristics. 3 Results A total of five Horizon 2020 projects and two IMI projects were identified as contributing to the development of HTA methods and tools (see Table 1). From these projects, 348 documents D1.7 – First yearly overview HTA methods WP1 – Identification, Synthesis & Prioritisation Version: 3.0 Authors: Saif Elayan, Karen Facey, Wim Goettsch Dissemination: PU 16/16 © Copyright 2025 SUSTAIN-HTA Consortium Kristensen, F. B. (2009). The HTA Core Model: A novel method for producing and reporting health technology assessments. International Journal of Technology Assessment in Health Care, 25(S2), 9–20. https://doi.org/10.1017/S0266462309990638 O’Rourke, B., Oortwijn, W., Schuller, T., & Group, the I. J. T. (2020). The new definition of health technology assessment: A milestone in international collaboration. International Journal of Technology Assessment in Health Care, 36(3), 187–190. https://doi.org/10.1017/S0266462320000215 Pokhilenko, I., Janssen, L. M. M., Paulus, A. T. G., Drost, R. M. W. A., Hollingworth, W., Thorn, J. C., Noble, S., Simon, J., Fischer, C., Mayer, S., Salvador-Carulla, L., Konnopka, A., Hakkaart van Roijen, L., Brodszky, V., Park, A.-L., & Evers, S. M. A. A. (2023). Development of an Instrument for the Assessment of Health-Related Multi-sectoral Resource Use in Europe: The PECUNIA RUM. Applied Health Economics and Health Policy, 21(2), 155–166. https://doi.org/10.1007/s40258-022-00780-7 Sculpher, M. (2008). Subgroups and Heterogeneity in Cost-Effectiveness Analysis. PharmacoEconomics, 26(9), 799–806. https://doi.org/10.2165/00019053-200826090-00009