Navigating Evidence Requirements for Joint Clinical Assessment of Orphan and Advanced Therapy Medicinal Products in Europe

Author(s)

Shruti Pandey, MPH1, Victoria K Brennan, BSc, MSc, PhD2, Amrita Sandhu, BSc, MSc2, Emanuele Arca`, Sr., MSc3.
1OPEN Health, Mumbai, India, 2OPEN Health, London, United Kingdom, 3OPEN Health, Rotterdam, Netherlands.
OBJECTIVES: From January 2025, all oncology drugs and advanced therapy medicinal products (ATMPs) will be subject to the new Joint Clinical Assessment (JCA) process in order to achieve reimbursement in Europe. The aim of this research was to understand country-specific data requirements for health technology assessments (HTAs) for orphan drugs and ATMPs and the potential implications for the JCA.
METHODS: The European Medicines Agency (EMA) website was searched to identify oncology OMPs and ATMPs receiving marketing authorization between July 2019 to July 2024. Products most commonly assessed across the EU were identified and their final HTA appraisals were assessed.
RESULTS: Of 75 EMA-approved OMPs (2019-2024), eight of the most frequently assessed oncology OMPs and ATMPs were included based on HTAs from six EU countries (France, Germany, Spain, Belgium, Denmark, and the Netherlands). The assessed indications included glioma, multiple myeloma, uveal melanoma, lymphomas and chronic myeloid leukemia. HTA agencies favored comparative data from randomized controlled trials (RCT) but increasingly accepted single-arm studies and surrogate endpoints. There was an increase in the use and acceptance of both real-world evidence (RWE) and data obtained through indirect treatment comparisons (ITCs). There was limited integration of patient-centered outcomes (PCO) and societal values/preferences in the submissions.
CONCLUSIONS: The findings indicate that a holistic approach to evidence generation, incorporating diverse evidence from RCTs, RWE, ITCs, PCOs and societal values/preferences, will be important for JCA submissions, with such evidence being increasingly accepted by HTA bodies; however, the impact on national HTAs and access outcomes remains uncertain. Although this research focuses on the European HTA landscape, the identified challenges and considerations for OMPs and ATMPs are likely to resonate with HTA bodies globally, given the standard complexities in appraising innovative therapies for conditions with limited evidence and unique therapeutic impacts.

Conference/Value in Health Info

2025-05, ISPOR 2025, Montréal, Quebec, CA

Value in Health, Volume 28, Issue S1

Code

HTA96

Topic

Health Technology Assessment

Topic Subcategory

Decision & Deliberative Processes

Disease

SDC: Oncology, SDC: Rare & Orphan Diseases

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