Bridging the Gap: Evidence Challenges for Advanced and Orphan Therapies in the EU HTA Landscape

The landscape of Health Technology Assessment (HTA) in Europe is evolving, particularly concerning advanced therapy medicinal products (ATMPs) and orphan medicinal products (OMPs) in oncology. The introduction of the EU HTA Regulation and the Joint Clinical Assessment (JCA) process aims to harmonize evaluations across EU Member States. However, disparities in national evidence requirements persist, creating challenges for developers and patients alike.

Key highlights include:

Evidence flexibility vs. rigor: randomized controlled trials remain the preferred evidence standard, but single-arm trials and validated surrogate endpoints are increasingly accepted in rare oncology indications.
Real-world evidence: countries such as France, Denmark, and the Netherlands incorporate registry and observational data to validate target populations, while others (Belgium and Spain) rely primarily on trial data.
Indirect treatment comparisons (ITCs): accepted where head-to-head trials are lacking, ITCs must be methodologically robust and transparent to support HTA decisions.
Patient-centered outcomes and quality of life: patient-centered outcomes and measures of patients’ quality of life are valued in some Member States (e.g., the Netherlands and France) but are not consistently considered across all HTA evaluations.

As Europe moves toward a unified HTA process, bridging national differences in evidence expectations is crucial. The JCA has the potential to streamline access, reduce duplication, and ensure that patients benefit from innovative oncology therapies without compromising scientific rigor.

Read the full white paper for an analysis of HTA processes, country-specific evidence requirements, and insights on how the JCA might reshape the evaluation of ATMPs and OMPs in Europe.

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OPEN Health authors:

Three headshots with names and titles: Shruti Pandey, Senior Research Analyst; Amrita Sandhu, Senior Market Access Consultant; Victoria Brennan, Director of Strategic Market Access—all at OPEN Health.

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