European Medicines Agency Announces Adaptive Licensing Pilot Project

The European Medicines Agency (EMA) has announced that it is inviting companies to participate in an adaptive licensing pilot project. EMA is asking companies to submit ongoing drug development programs for consideration as prospective pilot cases. Ongoing medicine development programs submitted by companies should be experimental drugs in the early stage of clinical development (i.e., prior to the initiation of confirmatory studies) to enable actionable input from relevant stakeholders.

The adaptive licensing approach, sometimes called staggered approval or progressive licensing, is a prospectively planned process, starting with the early authorization of a drug in a restricted patient population, followed by iterative phases of evidence gathering and adaptations of the marketing authorization to expand access to the medicine to broader patient populations. According to EMA, adaptive licensing requires the involvement of all stakeholders who have a role in determining patient access, including the EMA, the industry, health technology assessment (HTA) bodies, organizations issuing clinical treatment guidelines, and patient organizations. EMA expects all discussions to allow for free exploration of the strengths and weaknesses of all options for development, assessment, licensing, reimbursement, monitoring, and utilization pathways in a confidential manner and without commitment from either side.

“With the adaptive licensing pilot project we intend to explore with real medicines in development a progressive licensing approach that would allow timely access for patients to new medicines that address serious conditions with unmet medical needs,” explains Hans-Georg Eichler, the Agency’s Senior Medical Officer, in a press release. “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 their benefits and risks.”

Building on regulatory processes, adaptive licensing intends to extend the use of existing scientific advice, centralized compassionate use, the conditional marketing authorization mechanism (for drugs addressing life-threatening conditions), patients’ registries, and pharmacovigilance tools that allow collection of real-life data and development of risk management plans. EMA intends to include as many programs as necessary in the pilot phase to gather sufficient knowledge and experience, address technical and scientific questions, and refine the design of the adaptive licensing pathway for different types of products and indications.

The European Commission plans to examine the legal and policy aspects related to adaptive licensing in collaboration with the EU Member States and by consultation with relevant stakeholders, as necessary, as the project progresses.