EMA launches ‘adaptive licensing’ pilot

Europe is to launch a trial scheme aimed at getting potential breakthrough drugs to patients earlier.

The European Medicines Agency (EMA) has unveiled a pilot scheme for ‘adaptive licensing’ in which innovative new drugs will be released to patients before they have been given final regulatory approval.

The EMA is now inviting companies to participate in its adaptive licensing pilot project, and are requested to submit ongoing medicine development programmes for consideration as prospective pilot cases.

“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.

He added: “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.”

Adaptive licensing, sometimes called staggered approval or progressive licensing, is aimed at accelerating access for patients to new medicines. The EMA say it is a “prospectively planned process” and will start 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.

The regulator says 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. The EMA says all discussions will take place in a ‘safe harbour’ environment to allow free exploration of the strengths and weaknesses of all options for development, assessment, licensing, reimbursement, monitoring, and utilisation pathways in a confidential manner and without commitment from either side.

Similar goal to FDA’s Breakthrough Therapy Designation

The scheme 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 EU regulator’s plans has a similar goal to the FDA’s own accelerated approval system the ‘Breakthrough Therapy Designation’ – although the initial description suggests the EMA scheme is less radical in re-designing the regulator-pharma relationship, and relies more on adapting existing mechanisms.

Nevertheless, the news has been welcomed by the industry. UK industry association the ABPI said the pilot scheme was ‘big step forward’.

In a blog posting on the ABPI’s website, its head of regulatory affairs Esteban Herrero-Martinez commented: “We expect adaptive licensing to help support the development of medicines where there is high unmet need, such as novel antibiotics and medicines for rare diseases, and this pilot is a big step forward.

“Adaptive licensing could be a significant tool in the armoury to support more efficient medicine development and licensing, and ultimately ensure faster patient access to innovative medicines. We encourage all medicine developers to engage with this pilot where appropriate.”

The EMA says it wants to include as many programmes as necessary in the pilot phase in order to gather sufficient knowledge and experience, address a range of technical and scientific questions and further refine how the new pathway should be adapted for different products and indications.

The regulator wants companies to propose new medicines which are the early stage of clinical development, i.e., prior to the initiation of confirmatory studies, which could mean drugs currently in phase I or phase II being selected.

The European Commission will examine the legal and policy aspects related to adaptive licensing as the project progresses, and will work with EU Member States and consul with relevant stakeholders as necessary.

The EU-wide programme follows just days behind a similar UK-specific scheme, the Early Access to Medicines Scheme (EAMS).


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