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Fast Track Routes for Medicines that address Unmet Medical Needs
dateJuly 27, 2015
datePatricia Baede, Regulatory Expert

Launch of two-month public consultations on revised guidelines on accelerated assessment and conditional marketing authorization

The European Medicines Agency (EMA) has revised its guidelines on the implementation of accelerated assessment and conditional marketing authorization, two key tools in the European legislation to accelerate patients’ access to medicines that address unmet medical needs. Accelerated assessment and conditional marketing authorization are intended for innovative medicines that target a disease for which no treatment is available, or that provide patients with a major therapeutic advantage over existing treatments.

EMA’s accelerated assessment procedure allows for a faster assessment of eligible medicines by EMA’s scientific committees. CHMP opinion will be reach within the 150 days compared to 210 days in non-accelerated procedures. The proposed revision of the guideline includes a more detailed guidance on how to justify fulfilment of major public health interest, which is the basis for a request for an accelerated assessment. Early dialogue with EMA is emphasized.

Conditional marketing authorization allows for the early approval of a medicine on the basis of less complete clinical data than normally required, if the medicine addresses an unmet medical need and targets a seriously debilitating or life-threatening disease, a rare disease or is intended for use in emergency situations in response to a public health threat. While less complete, the available data must still demonstrate that the medicine’s benefits outweigh its risks and the applicant should be in a position to provide the comprehensive clinical data after authorization.

The EMA emphasizes on the importance of planning a conditional marketing authorization prospectively and engaging in early dialogue with and other stakeholders, for example health technology assessment bodies. This is expected to help translate conditional marketing authorizations into early access to medicines for patients. The revisions include clarification on fulfilment of unmet medical needs, i.e. medicines providing major improvements in patient care over existing therapies can be eligible in certain cases and clarification of how a positive benefit-risk balance is to be substantiated where there are less complete data.

The EMA expects the updated guidelines to optimize the use of these tools by medicine developers and consequently allow more medicines that address unmet medical needs to reach patients earlier. The public consultations on the revised guidelines are open until 30 September 2015.