The introduction of Joint Clinical Assessment (JCA) marked one of the biggest changes to European Health Technology Assessment in decades. Designed to create greater consistency across Member States, the new framework has the potential to reshape how evidence is assessed and how innovative medicines reach patients. Yet for orphan medicines, its long-term implications remain far from certain.
This paper examines what JCA could mean for rare disease innovation and patient access, exploring both the opportunities and the unresolved questions surrounding implementation. It considers how the new system may interact with Europe’s wider policy environment for orphan medicines and asks whether harmonised clinical assessment will strengthen (or further challenge) the sustainability of orphan drug development.
The report reviews emerging guidance, implementation plans and stakeholder expectations while highlighting areas where uncertainty remains, including evidence requirements, patient and clinician involvement, confidentiality, national implementation and the particular challenges faced by small biotechnology companies developing orphan therapies. It also considers the practical realities of generating evidence for rare diseases, where small patient populations and limited clinical data make traditional assessment approaches more difficult.
As JCA begins to move from legislation to implementation, stakeholders across Europe will need to understand not only how the process works, but also how it could influence pricing, reimbursement and future investment decisions. This paper provides an informed overview of the issues likely to shape the next phase of European HTA.
Whether you are preparing for JCA implementation or looking to understand its wider strategic implications, this report offers valuable insight into one of the most significant policy changes affecting rare diseases in Europe.
Download the paper to explore the opportunities, challenges and unanswered questions surrounding Joint Clinical Assessment.
