As the European Union prepares to extend Joint Clinical Assessments (JCA) to orphan medicines, stakeholders face important questions about evidence requirements and feasibility.
The EU’s JCA framework is intended to streamline clinical evaluations and create a more consistent foundation for health technology assessments (HTAs) across Member States. But for orphan medicinal products (OMPs), which will be subject to the JCA process beginning in January 2028, the transition may be more complex than it first appears.
CRA’s Life Sciences team worked independently to produce this white paper for Alexion, AstraZeneca, Rare Disease, exploring how challenges could play out under the JCA framework, and what stakeholders can do to prepare. After completing in-depth research across Germany, France, Italy, Spain, and Sweden, the authors conclude that:
1. PICO complexity could become a significant challenge for orphan medicines
At the heart of the JCA process is the development of PICOs (Population, Intervention, Comparator, and Outcomes), which define the scope of clinical assessment. For rare diseases, differences in patient populations, treatment practices, and comparator options across Member States can add up to hundreds of required PICOs, and thousands of analyses needed to support them.
2. Harmonization offers real opportunities, but only if implementation remains pragmatic
The JCA has the potential to reduce duplication, improve consistency across national assessments, and support faster patient access to innovative therapies. However, realizing these benefits will require a balanced approach that recognizes the realities of rare disease evidence generation. Excessive complexity could undermine the efficiencies that the framework is designed to create.
3. Learnings from early JCAs should be applied to optimize future processes
Tailored approaches, supported by continuous feedback and learnings from early JCAs, can help ensure the JCA framework evolves in a way that supports equitable access for rare disease patients. JCA PICO rules should build on what already works in member states to create a more operational, predictable, and effective framework for expert involvement in OMP assessments.
As implementation of the EU HTA Regulation continues, organizations developing therapies for rare diseases will need to understand how JCA requirements may affect evidence planning, market access strategies, and stakeholder engagement.
Download the full white paper to learn more about navigating JCA for OMPs.
