• Drug repurposing can accelerate access to medicines for unmet medical needs. • Despite efforts, regulatory barriers still hinder Marketing Authorisation for repurposed drugs. • Without approved therapies, off-label use enables access but carries major risks. • Ten proposals to promote and discuss regulatory innovation in drug repurposing. Despite increasing efforts and incentives to address the critical lack of treatments for rare diseases, a substantial gap persists between patient needs and available therapies. Drug repurposing is a promising strategy in this field; nevertheless, it has not yet translated into a substantial increase in approved medicines. Although several EU regulatory pathways exist, none are tailored to drug repurposing, resulting in a complex landscape, particularly for nonprofit organisations. While there is no silver bullet, this article presents ten regulatory proposals to foster dialogue with decision-makers to tackle bottlenecks in areas of unmet medical need through a collaborative model with aligned incentives for all stakeholders. These proposals are based on published literature and the authors’ experience in the SIMPATHIC project.
Pintado et al. (2026) studied this question.