JOINT CLINICAL ASSESSMENTS AND THE CHALLENGE OF EVALUATING MEDICINES WITH NON-TRADITIONAL EVIDENCE
Author(s)
Matias Olsen, -1, Matteo SCARABELLI, -2, Lydia Shotton, -3, Paul Ferreira, PharmD, MSc4, Victor Thomas, PharmD, MSc4, Robin Puzenat, PharmD, MSc4, Sebastien Faure, -4, Ingo Hantke, PhD5, Letizia Rossi, PharmD, MD6, Asís Ariznavarreta, -7.
1European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Brussels, Belgium, 2EFPIA, Brussels, Belgium, 3Alliance for Regenerative Medicine (ARM), Brussels, Belgium, 4Nextep, A PLG Company, Paris, France, 5Kintiga, Hannover, Germany, 6Intexo Società Benefit, A PLG Company, Rome, Italy, 7Outcomes'10, A PLG Company, Castellon, Spain.
1European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Brussels, Belgium, 2EFPIA, Brussels, Belgium, 3Alliance for Regenerative Medicine (ARM), Brussels, Belgium, 4Nextep, A PLG Company, Paris, France, 5Kintiga, Hannover, Germany, 6Intexo Società Benefit, A PLG Company, Rome, Italy, 7Outcomes'10, A PLG Company, Castellon, Spain.
OBJECTIVES: Regulation (EU)2021/2282 introduced Joint Clinical Assessments for oncology and ATMPs in January 2025, with first reports emerging in 2026 and scope expanding to orphan drugs in 2028. This study assesses how national HTA bodies across six countries (France, Germany, Italy, Poland, Spain, Sweden) evaluated medicines supported by unconventional evidence and explores how the emerging framework might affect future national assessments and patient access.
METHODS: The research is conducted on three assets volunteered by companies in the rare oncology, orphan, and advanced therapies space, recently approved in the EU, assessed in at least two countries and supported by non-randomized evidence. Submitted clinical package, HTA outcome and reimbursement decision were documented. Simulated JCA reports were generated across four domains (study design and internal validity; endpoint relevance; population and transferability; evidence maturity), based on EU HTA guidance and the first published JCA report. National and simulated outcomes were compared to identify divergences.
RESULTS: All national bodies flagged identical methodological limitations, such as single-arm designs, absent comparators, indirect comparisons and immature follow-up. Yet reimbursement decisions ranged from full coverage to denial. Decisive factors were disease severity, absence of alternatives and national flexibility mechanisms, not the evidence package alone. The JCA simulation introduced no new concerns but reframed existing ones, embedding implicit value judgments that could shift baseline toward "uncertainty dominant, access threatened".
CONCLUSIONS: Findings suggest that JCA frameworks should place greater emphasis on the transparent and proportionate management of uncertainty. As the latest is an intrinsic feature of assessment, particularly in areas of high unmet need and rapid innovation, it should be addressed through appropriate methodological approaches rather than interpreted as a reason to restrict access. This does not imply lower standards of evidence, but rather smarter and context-sensitive assessment approaches that recognize the realities of evidence generation while minimizing implicit judgments in JCA reports and guidance.
METHODS: The research is conducted on three assets volunteered by companies in the rare oncology, orphan, and advanced therapies space, recently approved in the EU, assessed in at least two countries and supported by non-randomized evidence. Submitted clinical package, HTA outcome and reimbursement decision were documented. Simulated JCA reports were generated across four domains (study design and internal validity; endpoint relevance; population and transferability; evidence maturity), based on EU HTA guidance and the first published JCA report. National and simulated outcomes were compared to identify divergences.
RESULTS: All national bodies flagged identical methodological limitations, such as single-arm designs, absent comparators, indirect comparisons and immature follow-up. Yet reimbursement decisions ranged from full coverage to denial. Decisive factors were disease severity, absence of alternatives and national flexibility mechanisms, not the evidence package alone. The JCA simulation introduced no new concerns but reframed existing ones, embedding implicit value judgments that could shift baseline toward "uncertainty dominant, access threatened".
CONCLUSIONS: Findings suggest that JCA frameworks should place greater emphasis on the transparent and proportionate management of uncertainty. As the latest is an intrinsic feature of assessment, particularly in areas of high unmet need and rapid innovation, it should be addressed through appropriate methodological approaches rather than interpreted as a reason to restrict access. This does not imply lower standards of evidence, but rather smarter and context-sensitive assessment approaches that recognize the realities of evidence generation while minimizing implicit judgments in JCA reports and guidance.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA188
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Systems & Structure, Value Frameworks & Dossier Format
Disease
Oncology, Rare & Orphan Diseases