On 19 March 2014, the EMA announced the launch of its adaptive licensing pilot project along with an invitation to interested companies to submit their ongoing medicine development programmes to be considered as pilot cases.
The term “adaptive licensing” (also known as “staggered approval” or “progressive licensing”) describes a process which allows patients to have early access to new medicinal products by starting with an early authorisation of a product for a specific, restricted patient subset and gradually adapting the marketing authorisation to include broader patient populations as additional clinical evidence is gathered.
The adaptive licensing approach is expected to ensure timely access for patients to new medicines, especially those addressing serious conditions with unmet medical needs, while at the same time safeguarding the principle of risk-benefit assessment.
All stakeholders involved in and impacted by patient access to medicines are invited to participate in discussions, including the EMA and other regulatory authorities, such as Health Technology Assessment (HTA) bodies, the industry as well as patient organisations. Discussions will take place in a “safe harbour” environment, in confidentiality and with no commitment from either side.
The EMA envisages that adaptive licensing can be utilized within the current EU regulatory framework, without the need for new or amended legislation. The adaptive licensing project will therefore be based on existing regulatory processes and tools, such as:
- the offer of scientific advice by the EMA;
- the centralised compassionate use option;
- the mechanism of conditional marketing authorisation for medicines treating life-threatening conditions;
- patient registries and pharmacovigilance tools for the collection of real-life data and the development of risk management plans.
All interested companies are invited to submit ongoing medicine development programmes, which should concern experimental medicines in the early stage of clinical development, i.e. prior to the initiation of confirmatory studies, in order to allow for more practically useful input from stakeholders. The application form as well as a framework document to guide the discussions on individual pilot studies are available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/general/general_content_000571.jsp&mid=WC0b01ac0580665b62
The EMA plans to include as many programmes as necessary in the pilot project, with the aim to obtain experience from exchanges with stakeholders, to address scientific or technical issues as well as to progressively adjust adaptive licensing procedures to different types of medicines or conditions. The Commission, along with Member States and other stakeholders, will address legal and policy issues as the project evolves.