Comparing Clinical Benefit Criteria in Health Technology Assessment of Oncology Drugs in the Netherlands and Europe: Driving Real Value or Inequal Access?
Author(s)
Marnix Uyterlinde, MSc1, Laura van der Linde, MSc2, David Maarten Smalbrugge, MSc2, Ha Anh Pham, MSc2.
1Research Associate, Leiderdorp, Netherlands, 2Cytel, Rotterdam, Netherlands.
1Research Associate, Leiderdorp, Netherlands, 2Cytel, Rotterdam, Netherlands.
OBJECTIVES: The PASKWIL-criteria are used by the Dutch Healthcare Institute (Zorginstituut) to assess the added clinical benefit of novel oncology therapies and inform recommendations for reimbursement. The criteria consist of minimum thresholds for absolute and relative survival gains (progression-free [PFS] and overall [OS]), depending on the median survival with standard of care, and therapeutic intent (curative vs palliative). The PASKWIL-criteria differ from the European Society of Medical Oncology (ESMO) Magnitude of Clinical Benefit Score (MCBS) in outcomes assessed and criteria applied, raising concerns about discrepancies in evaluations in the Netherlands and Europe. This study compares both frameworks to evaluate their impact on access to new oncology therapies in the Netherlands.
METHODS: A review of European Public Assessment Reports was conducted to identify assessments related to new therapeutic indications approved in 2024. Clinical relevance was assessed based on the PASKWIL and ESMO-MCBS criteria. ESMO-MCBS recommendations grade A (curative treatments) and grade 4/5 (palliative treatments) were considered clinically meaningful.
RESULTS: A total of 32 assessments were identified across the adjuvant (n=5) and palliative settings (n=27): 15 met PASKWIL and ESMO-MCBS criteria, 2 met only the ESMO-MCBS criteria, 1 only met PASKWIL criteria and 13 met neither. Differences in recommendations were attributable to stricter thresholds being applied for OS (n=2) with PASKWIL and to a lack of differentiation on toxicities and quality of life (n=1).
CONCLUSIONS: The application of the PASKWIL criteria leads to different outcomes than with ESMO-MCBS, although the impact on access to therapies in practice may be limited. The discrepancies in recommendations are primarily attributable to the endpoints considered and the number and nature of thresholds applied. The emphasis on primary clinical endpoints by ESMO-MCBS may lead to more favorable assessments at the European level for trials with PFS as a primary endpoint, while resulting in negative assessments by PASKWIL.
METHODS: A review of European Public Assessment Reports was conducted to identify assessments related to new therapeutic indications approved in 2024. Clinical relevance was assessed based on the PASKWIL and ESMO-MCBS criteria. ESMO-MCBS recommendations grade A (curative treatments) and grade 4/5 (palliative treatments) were considered clinically meaningful.
RESULTS: A total of 32 assessments were identified across the adjuvant (n=5) and palliative settings (n=27): 15 met PASKWIL and ESMO-MCBS criteria, 2 met only the ESMO-MCBS criteria, 1 only met PASKWIL criteria and 13 met neither. Differences in recommendations were attributable to stricter thresholds being applied for OS (n=2) with PASKWIL and to a lack of differentiation on toxicities and quality of life (n=1).
CONCLUSIONS: The application of the PASKWIL criteria leads to different outcomes than with ESMO-MCBS, although the impact on access to therapies in practice may be limited. The discrepancies in recommendations are primarily attributable to the endpoints considered and the number and nature of thresholds applied. The emphasis on primary clinical endpoints by ESMO-MCBS may lead to more favorable assessments at the European level for trials with PFS as a primary endpoint, while resulting in negative assessments by PASKWIL.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA81
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Oncology