New EMA/HMA pilot on medicines repurposing

The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) launched a pilot to support the repurposing of medicines. The pilot is being carried out 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 initiative aims to support not-for-profit organisations and academia, referred to as “the Champion”, to gather or generate sufficient evidence on the use of an established medicine in a new indication to support an application from the current marketing authorisation holder (MAH) for a new use that can then be formally authorised by a participating regulatory authority.

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. Applicants should present a scientific rationale for their repurposing programme and will be able to receive Scientific Advice from a regulatory authority. EMA and 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. The pilot is planned to run until the completion of Scientific Advice for the selected repurposing candidate projects and possibly until the filing of an application by a pharmaceutical company for the new indication.

The Marketing Authorisation Holder (MAH) is envisaged to be involved in two stages of the process, either before or after “the Champion” has obtained scientific advice:

• “The Champion” may contact one or more of the existing MAHs for the product or active ingredient to seek their views on the proposed new use. The input may range from none at all, to commentary or extend to data sharing or collaboration with “the Champion” in seeking Scientific Advice;

• “The Champion” is encouraged to contact the MAHs to share the outputs from the Scientific Advice, with the aim of at least one MAH seeking to obtain regulatory approval for the new indication via a Marketing Authorisation variation. If no collaboration has taken place, or if it has been unsuccessful “the Champion” is encouraged to take forward the development programme as far as possible on their own, and other regulatory or Member State-specific measures might apply.

EUCOPE has provided input to the drafting of the proposed STAMP framework and is a participant in the multistakeholder Repurposing Observatory Group, which is overseeing the roll-out of the pilot. If you have any questions, please contact Laurent (louette@eucope.org) or Matias (olsen@eucope.org).

More information can be found on the EMA’s website.