The Preferences for Advanced Therapy Medicinal Products: A Multicriteria Decision Analysis Framework
Author(s)
Hui-Chu Lang, MPH, PhD1, ChiaChun Wu, Master2, Pin-Yu Huang, Master2.
1Professor, National Yang Ming Chiao Tung University, Taipei, Taiwan, 2Institute of Hospital and Health Care Administration, Taipei, Taiwan.
1Professor, National Yang Ming Chiao Tung University, Taipei, Taiwan, 2Institute of Hospital and Health Care Administration, Taipei, Taiwan.
OBJECTIVES: Advanced therapy medicinal products (ATMPs) represent revolutionary treatments for previously untreatable or inadequately managed conditions. However, ATMPs present unprecedented challenges for healthcare systems due to their exceptional costs, limited long-term efficacy data, complex manufacturing requirements, and uncertain durability of effect. The study aims to deliver an ATMP-specific MCDA framework that balances affordability, clinical effectiveness, and long-term value considerations.
METHODS: Our study employs a mixed-methods approach beginning with a systematic literature review examining established MCDA frameworks in healthcare, specifically analyzing EVIDEM, Advanced Value Framework, Hungary's National Framework, and VALIDATE for their applicability to ATMPs. We also reviewed recent HTA decisions for approved ATMPs to identify evaluation patterns and challenges. A comprehensive criteria list will be developed and refined through a 2-round modified Delphi process involving a multidisciplinary panel of 25 experts (clinicians, health economists, patient representatives, payers, industry specialists, and bioethicists). Finally, the Analytic Hierarchy Process (AHP) was applied to establish stakeholder preferences for the refined framework.
RESULTS: After comprehensive reviews, we identified five core value domains relevant for ATMPs: disease burden, therapeutic outcomes, economic impacts, innovation level, and evidence quality, with 18 potential criteria. After experts reviewed the definitions of each factor, we kept 15 criteria and deleted the last three with the lowest scores. The rankings are as follows: 1. Improvement of efficacy/effectiveness 2. Unmet Medical Needs 2. Improvement of adverse events and tolerability 4. Evidence on Efficacy and Comparative Effectiveness 5. Disease severity 5. Size and Design of Trials 5. Disease Progression and Long-Term Effect 8. Public health interest 9. Affordability 10. Ethical analysis 11. Cost-effectiveness 11. Innovation 13. Family and Societal Impact 14. Equity 15. Budget impact.
CONCLUSIONS: AHP was further applied to establish stakeholder preferences for the refined framework. We expect this framework to provide decision-makers with a structured approach to evaluate ATMPs.
METHODS: Our study employs a mixed-methods approach beginning with a systematic literature review examining established MCDA frameworks in healthcare, specifically analyzing EVIDEM, Advanced Value Framework, Hungary's National Framework, and VALIDATE for their applicability to ATMPs. We also reviewed recent HTA decisions for approved ATMPs to identify evaluation patterns and challenges. A comprehensive criteria list will be developed and refined through a 2-round modified Delphi process involving a multidisciplinary panel of 25 experts (clinicians, health economists, patient representatives, payers, industry specialists, and bioethicists). Finally, the Analytic Hierarchy Process (AHP) was applied to establish stakeholder preferences for the refined framework.
RESULTS: After comprehensive reviews, we identified five core value domains relevant for ATMPs: disease burden, therapeutic outcomes, economic impacts, innovation level, and evidence quality, with 18 potential criteria. After experts reviewed the definitions of each factor, we kept 15 criteria and deleted the last three with the lowest scores. The rankings are as follows: 1. Improvement of efficacy/effectiveness 2. Unmet Medical Needs 2. Improvement of adverse events and tolerability 4. Evidence on Efficacy and Comparative Effectiveness 5. Disease severity 5. Size and Design of Trials 5. Disease Progression and Long-Term Effect 8. Public health interest 9. Affordability 10. Ethical analysis 11. Cost-effectiveness 11. Innovation 13. Family and Societal Impact 14. Equity 15. Budget impact.
CONCLUSIONS: AHP was further applied to establish stakeholder preferences for the refined framework. We expect this framework to provide decision-makers with a structured approach to evaluate ATMPs.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA327
Topic
Health Policy & Regulatory, Health Technology Assessment, Methodological & Statistical Research
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure, Value Frameworks & Dossier Format
Disease
No Additional Disease & Conditions/Specialized Treatment Areas