Regulatory challenges and incentives for repurposing – Lessons learned from a Swedish pilot

Repurposing is, according to the definition of the European pilot, a project where an academic organization or a not-for-profit entity (“champion”), aims to gather enough data for regulatory approval of a previously not approved indication for a medicinal product already on the market within the European Union ^1,2.

In addition to the “champion criteria”, another criterion is that the product should not be under regulatory or intellectual property protection. The indication to be approved should be within an area where important public health benefits are likely to be achieved.

According to the definition of the EU pilot, repurposing is not regarded as life cycle management where a Marketing Authorisation Holder (MAH) acts by its own initiative to get a new indication approved for its products.

In order to file a market authorisation application for a new indication, the champion should pair up with a company, which could be a generic or biosimilar company or alternatively any other interested company, to proceed with submission for approval.

There are regulatory tools available in the current pharmaceutical EU legislation for human medicines (2001/83/EC), but there are regulatory challenges, and the incentives and pathways are not clear. However, in the new legislative proposal from the EU Commission as of April 2023, the term “repurposing” is introduced along with regulatory incentives.

The Swedish Medical Products Agency has, in parallel to the EU-pilot, conducted a national pilot ³. This to reach out to the academia and not-for-profit organization nationally and gather information on e.g. support needs and current obstacles/challenges. A workshop with national stakeholders (academia, innovation hubs, branch organisation and regulators) was performed to obtain input to the pilot for such as but, not limited to, inclusion criterions. The experiences will be shared during the presentation.