Repurposing of authorised medicines: pilot to support not-for-profit organisations and academia
EMA and the Heads of Medicines Agencies (HMA) are launching a pilot project to support the repurposing of medicines as a follow-up to the European Commission’s Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) discussions on a proposal for a medicines repurposing framework.
The aim of this initiative is to support not-for-profit organisations and academia to gather or generate sufficient evidence on the use of an established medicine in a new indication with the view to have this new use formally authorised by a regulatory authority. This is a way of making new treatment options available to patients.
As part of the pilot, EMA and the national medicines agencies will provide regulatory support, primarily scientific advice, to help these stakeholders generate a data package robust enough to support a future application by a pharmaceutical company.
Candidate medicines for the pilot should fulfil the following criteria:
- contain a well-established active substance;
- be an authorised medicine (containing the concerned active substance) out of data exclusivity and market protection periods and out of basic patent / supplementary protection certificate (SPC) protection;
- target an indication in a condition distinct from the currently authorised indication(s);
- target an indication in an area where important public health benefits are likely to be achieved. Conditions for which no or few medicines are currently authorised or which are associated with high morbidity and / or mortality despite available medicines, will be the focus of the pilot.
Eligible academia sponsors developing orphan medicines will automatically benefit from a fee waiver. Additional fee waivers will be granted to a subset of selected applications taking into account the extent of the expected public health benefits and the strength of the evidence to substantiate the promise held by the proposal.
While marketing authorisation holders may develop medicines for uses in other indications, sometimes they lack the incentives or the commercial interest to pursue the necessary research and development and complete the regulatory process needed for the authorisation of a new indication for old medicines which are no longer protected by a patent or data exclusivity. This could be a wasted opportunity for public health. At the same time, academic institutions and/or patient organisations may be interested in carrying out this development for the benefit of public health. However, they may not have the necessary regulatory experience and have no intention of becoming a marketing authorisation holder themselves.
The pilot is open to not-for-profit stakeholders and academia (institutions and individuals) who have a particular interest in repurposing an authorised medicine for a new indication in an area of public health interest, have a scientific rationale for their repurposing programme and would like to seek scientific advice from a regulatory authority.
Further information on the pilot project is available in a question-and-answer document .
Sponsors wishing to seek EMA scientific advice should complete the drug repurposing submission form and submit it to email@example.com by 28 February 2022. For submissions to national competent authority contacts, please refer to the additional information in the annex to the question and answer document.
The pilot will run until the completion of scientific advice for the selected repurposing candidate projects and optimally until the filing of an application by a pharmaceutical company for the new indication.
A report will be published after the pilot.
The medicines repurposing framework proposal was developed by the European Commission’s STAMP Expert Group composed of representatives of EU Member States together with EMA and stakeholders from not-for-profit organisations, patients, healthcare professionals, industry, health technology assessment bodies and payers.
EMA proposes to support the development and implementation of a repurposing framework in its Regulatory Science Strategy to 2025, which is its plan for advancing engagement with regulatory science over the next five to ten years.
Repurposing of medicines for COVID-19 falls outside the scope of this pilot project. The development and authorisation of treatments for COVID-19 is coordinated by the COVID-19 EMA pandemic Task Force (COVID-ETF) and should follow the steps outlined in the following document: EMA initiatives for acceleration of development support and evaluation procedures for COVID-19 treatments and vaccines . Repurposing programmes for medicines intended for COVID-19 will therefore not be considered for this pilot.