Drug repurposing pilot to support not-for-profit organisations and academia

The European Medicines Agency (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 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.

While marketing authorisation holders may develop medicines for uses in other indications, they can 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. Meanwhile, academic institutions and patient organisations may be interested in carrying out this development for the benefit of public health but they may not have the necessary regulatory experience and have no intention of becoming a marketing authorisation holder themselves.

The pilot is therefore 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 wish to seek scientific advice from a regulatory authority.

As part of the pilot, EMA and the national medicines agencies will provide regulatory support and primarily scientific advice to help stakeholders generate a data package robust enough to support a future drug application by a pharmaceutical company.

EMA state that the candidate medicines 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.

According to EMA, eligible academia sponsors developing orphan medicines will automatically benefit from a fee waiver and additional fee waivers will be granted to a subset of selected applications.

The pilot will run until the completion of scientific advice for the selected drug 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.