Based on the initial experience gained from its adaptive licensing pilot project, the European Medicines Agency
(EMA) has released a document to address the most frequently asked questions from companies and to clarify the terms of engagement and expected outputs for prospective applicants.
The document clarifies in particular: the criteria used by the agency to select suitable candidates for the pilot project; the concrete steps involved in the process and expected outcomes; the context and aim of the exploratory discussions between the agency and selected candidates.
The EMA launched its adaptive licensing pilot project in March 2014 to explore how adaptive licensing can be implemented with real medicine development programs. The adaptive licensing approach, sometimes called staggered approval or progressive licensing, is part of the EMA's efforts to improve timely access for patients to new medicines that address serious conditions with unmet medical needs.
It is a prospectively planned process, starting with the early authorization of a medicine in a restricted patient population, followed by iterative phases of evidence gathering and adaptations of the marketing authorization to expand access to the medicine for increasingly broader patient populations. The approach seeks to maximize the positive impact of new medicines on public health by balancing timely access for patients with the need to provide adequate evolving information on the benefits and risks.
So far the EMA has received and assessed 26 applications as part of its adaptive licensing pilot project, seven of which have been selected for a discussion with the applicant.
The EMA continues to welcome applications from interested companies as it intends to include as many programs as necessary in the pilot to gather sufficient knowledge and experience, address a range of technical and scientific questions, and further refine how the adaptive licensing approach should be designed for different types of products and indications. Applications are reviewed on a monthly basis. The Agency will review the findings of the adaptive licensing pilot project at the end of 2014 and will then decide on the next steps.