A new research topic on Frontiers in Medicine Regulatory Science journal has just been published! It is dedicated to repurposing medicines to cover unmet therapeutic needs for small populations, like paediatric and rare disease patients.

Viviana Giannuzzi, from Fondazione Gianni Benzi, is one of the co-editors.

Drug repurposing or repositioning is defined as the process of finding new uses outside the scope of the original medical indication for existing medicines. It might relate to new patient populations, dosage forms or routes of administration. The drug candidate for a repurposed use could be a medicine already marketed, including old medicines with an expired intellectual property protection, or a medicine under development for its first intended medical use, when it is discovered to be effective for the treatment of another condition. Drug repositioning represents an emerging and dynamic drug development strategy, and it is expected to reduce time, costs, and development risks. In settings with special needs, like paediatric and rare diseases characterised by well-known challenges, repurposing is considered as an opportunity to cover unmet medical needs.

Key themes of this research topic are:

• Industry and academic approaches as a R&D opportunity;
• Scientific evaluations and regulatory decision-making;
• Successes and failures of off-label uses gone into a repurposing path;
• Commercial perspectives and strategies, including public-private partnerships;
• Formulations to comply with the specific patient needs;
• Covering needs for ultra-rare diseases;
• Tools like Real World Data and pharmacometrics providing evidence to endorse drug use;
• international and national initiatives to exploit repurposing pathways.

Submission deadline for abstracts is 31 October 2023 and for manuscripts 29 February 2024.

Manuscripts as original research, review, mini review, policy and practice review, hypothesis and theory, perspective, clinical trial, case report, policy brief, brief research report, general commentary, opinion, on the developments, applications, and policies on repurposing for small populations will be considered.

Discover more about the Research Topic by visiting the Frontiers website where you can also find the author guidelines.