EMA to fast track much-needed therapies

by Gary Finnegan: The European Medicines Agency wants to get new medicines to patients quicker where there is a clear unmet medical need. The move could also see some medicines given provisional approval while additional evidence is gathered.

The medicines regulator has launched a consultation on new guidelines on accelerated assessments and conditional marketing authorisation, with a deadline of September 30.

The fast-track approval system is 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.

The new guidelines are based on the Agency’s recent experience with fast-tracked assessment as well as feedback from the European Commission Expert Group on Safe and Timely Access to Medicines for Patients (STAMP).

Products eligible for the scheme would still be assessed by scientific committees but commits the agency to a response time of 150 days – rather than the 210 target for typical medicines).

The granting of conditional marketing authorisation would allow patients to access innovative therapies even where the level of available clinical data is lower than normally required.

This could be offered where medicines address 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 authorisation within a timeframe agreed with the EMA.

In addition, the benefit to public health must outweigh the risk due to the limited availability of clinical data at the time of marketing authorisation.