European Medicines Agency launches adaptive licensing pilot project

The European Medicines Agency (EMA) is inviting pharmaceutical companies to participate in its adaptive licensing pilot project. Those interested are requested to submit ongoing medicine development programmes for consideration as prospective pilot cases.

A framework to guide discussions of individual pilot studies has been published.

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 starts with the early authorisation of a medicine in a restricted patient population, followed by iterative phases of evidence gathering and adaptations of the marketing authorisation 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, organisations issuing clinical treatment guidelines and patient organisations. All discussions will be confidential and without commitment from either side to allow free exploration of the strengths and weaknesses of all options for development, assessment, licensing, reimbursement, monitoring, and utilisation pathways.

'The approach seeks to maximise 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,' said Hans-Georg Eichler, the EMA's Senior Medical Officer.

Adaptive licensing builds on existing regulatory processes

Adaptive licensing builds on existing regulatory processes and intends to extend the use of elements that are already in place, including scientific advice, centralised compassionate use, the conditional marketing authorisation mechanism (for medicines addressing life-threatening conditions), patients’ registries and pharmacovigilance tools that allow collection of real-life data and development of risk management plans.

The Agency intends to include as many programmes as necessary in this pilot phase so that it can 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 programmes submitted by companies 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 the EU members and by consultation with relevant stakeholders, as necessary.

The BioIndustry Association (BIA) has welcomed the launch of the pilot project.

Steve Bates, BIA Chief Executive, said: 'The BIA has campaigned for the introduction of adaptive licensing for a number of years and we will encourage our members to respond to the Agency's call for participation in the pilot project.

'We note that the introduction of an adaptive licensing pilot project was a recommendation of the Expert Group on innovation in the regulation of healthcare. We pay tribute to the leading role the Medicines and Healthcare Products Regulatory Agency has taken on this agenda.'