CPE has joined a coalition focused on the advanced therapies and rare diseases, including patient organisations, clinicians, medical societies, academic centres and therapy developers, in endorsing a joint stakeholder statement responding to the first Joint Clinical Assessment (JCA) report published under the EU Health Technology Assessment (HTA) Regulation.
The statement reflects on the implementation of the new European JCA framework from the perspective of advanced therapy medicinal products (ATMPs), highlighting key considerations to ensure that it fulfils its intended purpose: supporting evidence-based decision-making while enabling timely and equitable patient access to innovative therapies across Europe.
The coalition welcomes the introduction of the Joint Clinical Assessment as an important milestone for European cooperation in health technology assessment. At the same time, it stresses that the first reports provide valuable lessons for strengthening the process from the outset.
The statement identifies three priorities for improving the JCA framework:
- Evidence requirements should reflect the realities of rare diseases and other evidence-constrained settings. Innovative therapies developed for small patient populations often rely on evidence that differs from traditional clinical development programmes. The assessment framework should recognise these realities and appropriately contextualise uncertainty.
- Patient experience and clinical expertise should play a more meaningful role in interpreting evidence. The coalition calls for earlier, broader and more transparent involvement of patients, carers and clinical experts, ensuring that outcomes important to patients and the realities of living with complex diseases are fully considered throughout the assessment process.
- The JCA should support faster and more consistent access to innovation across Europe. By making better use of the available evidence and reducing unnecessary duplication at national level, the JCA can help deliver on the objectives of the EU HTA Regulation while improving access to innovative treatments for patients.
As a signatory to the statement, CPE reiterates that patient perspectives must be meaningfully integrated throughout the Joint Clinical Assessment process. A transparent, patient-centred and scientifically robust assessment framework is essential to ensuring that people affected by cancer and other serious conditions can benefit from timely access to innovative therapies.
The coalition also encourages European policymakers and HTA decision-makers to build on the experience of the first JCA reports by strengthening methodological guidance, expanding opportunities for patient and clinical expert involvement, and ensuring that the framework can effectively assess innovative therapies across all disease settings.
Read the full Joint Stakeholder Statement here.
