The EMA is inviting companies to participate in its adaptive licensing pilot project. Interested companies should submit ongoing medicine development programs for consideration as prospective pilot cases.
The adaptive licensing approach, sometimes called staggered approval or progressive licensing, is part of the agency’s efforts to improve timely access for patients to new medicines. 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 to broader patient populations.
As a holistic approach, 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. All discussions will take place in a “safe harbor” environment to allow 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,” said Hans-Georg Eichler, EMA senior medical officer. “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.”
Adaptive licensing builds on existing regulatory processes and intends to extend the use of elements already in place, including scientific advice, centralized compassionate use, the conditional marketing authorization mechanism (for medicines addressing life-threatening conditions), patient registries and pharmacovigilance tools that allow collection of real-life data and development of risk management plans.
The EMA intends to include as many programs as necessary in this pilot phase to gather sufficient knowledge and experience, address a range of technical and scientific questions and further refine how the adaptive licensing pathway should be designed for different types of products and indications.
Ongoing medicine development programs submitted should be experimental medicines in the early stage of clinical development, i.e., prior to the initiation of confirmatory studies, to enable actionable input from relevant stakeholders.
As the project progresses, the European Commission will examine the legal and policy aspects related to adaptive licensing in collaboration with E.U. member states and by consultation with relevant stakeholders, as necessary.