In the vast landscape of medicine, innovation is often associated with groundbreaking discoveries and novel treatments. However, there is an often unexplored area of potential. Existing medicines which are used in different context that could give fresh hope for patients’ conditions with limited or no treatment options –  we refer to this as drug repurposing.

This week sees the first international drug repurposing conference, co-organised by REMEDi4ALL, Beacon and MeRIT and taking place on 6-7 March 2024 in Barcelona. In this global event, key opinion leaders from both the research and patient communities, funders, regulators and representatives from the pharmaceutical industry will come together to advance innovative drug repurposing in Europe and beyond. 

This blog will explore, from the innovative pharmaceutical industry’s perspective, some of the important aspects in the exciting world of drug repurposing and its potential to positively impact healthcare.

Terminology matters

While no common definition exists in literature, innovative industry understands ‘Repurposing’ as the process of bringing on label new therapeutic uses for already known medicines which are out of patent and regulatory data protection, where for example a third party (academia, non-profit organisations, etc.) generates regulatory grade evidence of a potential new use. This definition excludes abandoned compounds, new uses for drugs in development, and recently approved drugs where development activities are still being carried out by the originator company. The last two in particular are part of the normal life cycle activities of a product and already contemplated in the pharmaceutical regulatory framework in the form of development of new formulation routes (e.g. turning a tablet form into oral syrup to be more easily taken by children or elderly patients) for existing products and new indications.

A tech-supported second Chance for Older Medicines at Times of Crises

Drug repurposing can provide avenues to explore new uses of older, off-patent medicines, for example in areas where no treatments are available, or research gaps exist, thus benefitting patients affected by conditions for which limited, or no therapeutic options are approved and/or development is economically challenging. The COVID-19 pandemic showcased the power of drug repurposing. Despite only a fraction of drugs proving effective, the efforts demonstrated the speed at which repurposing explores treatment options in uncharted territories. Technologies like Artificial Intelligence (AI) and Machine Learning (ML), along with data-sharing platforms, enhance the efficiency of drug repurposing. The future holds promise for even more breakthroughs.

Navigating Regulations – Proposal for Principles

Supported by EFPIA members, we propose principles to guide policies and legislations that will ensure safety and efficacy of repurposed drugs.

• A dedicated pathway for repurposing must meet the same rigorous standards as any new medicine. The EU regulatory system is designed to uphold public health and any changes for repurposed medicines needs careful consideration.
• In repurposing, the expertise of marketing authorisation holders, who deeply understand their medicinal product, is vital. Labelling changes on new indications require collaboration and consultation with MAHs to ensure patient safety and informed prescribing.
• While repurposing holds great potential, incentives must balance healthcare system interests and foster innovation. Economic challenges, cross-label use, and pricing hurdles must be addressed for a vibrant innovation ecosystem.
• A new framework for repurposing should focus on conditions with limited treatment options or high morbidity/mortality despite available medicines.

Striking a balance between innovation and access to medicines is crucial. Drug repurposing is a beacon of hope, offering a second chance to existing medicines and providing solutions for conditions with limited treatment options. Through drug repurposing, we can unlock a path to a healthier, more innovative future.

As we navigate the intricate web of current and future regulations, collaborations, and incentives, the ultimate goal remains clear: to improve access to medicines for European patients. To ensure successful implementation, it is crucial to adhere to stringent scientific and regulatory standards, involve marketing authorisation holders throughout the process, establish appropriate incentives and, in particular, remove disincentives and prevent repurposing from becoming a mere economic substitute for developing new treatments. We are committed to contributing to the development of a regulatory framework that supports and facilitates collaborative repurposing initiatives.

The authors are/have been EFPIA representatives in the Repurposing Observatory Group led by the EMA and the Heads of Medicines Agencies (HMA).  This group advices on various aspects of an on-going 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.