Barriers and Policy Solutions in Off-Patent Drug Repurposing: A Full-Life-Cycle Perspective
Author(s)
Mario García-Díaz, PhD student1, David Epstein, PhD2, Jaime Espin, MSc, PhD3.
1Research Assistant, Escuela Andaluza de Salud Pública, Granada, Spain, 2University of Granada, Granada, Spain, 3EASP, Granada, Spain.
1Research Assistant, Escuela Andaluza de Salud Pública, Granada, Spain, 2University of Granada, Granada, Spain, 3EASP, Granada, Spain.
OBJECTIVES: Drug repurposing leverages the development of medicines that have already been studied to reduce clinical R&D time and healthcare costs. However, off-patent medicines do not provide incentives for the private sector, and prescribing by active ingredient is a major barrier. In addition, academic researchers/NGOs face regulatory barriers.
METHODS: Through a review of the literature and a questionnaire to drug repurposing experts, we capture the entire problematic of off-patent drug repurposing and the different solutions offered by governments from the moment a medicine candidate for repurposing is found until that medicine obtains a new indication (lifecycle perspective).
RESULTS: There is sufficient public funding for the early stages of R&D, where opportunities can also be identified from off-label uses, RWE studies and computational models. Even so, funding is limited for robust clinical trials and, added to the fact that R&D is led by academics/NGOs with academic objectives rather than regulatory objectives, the probability of success in obtaining marketing authorisation is usually less than 30%. As solutions, research should focus on areas such as rare diseases and unmet health needs. Governments should play a more prominent role in public investment and promote public-private partnerships to provide financial and regulatory support (with Marketing Authorisation Holders support) to academics/NGOs. Finally, a major problem is pharmacy substitution in dispensing at pharmacies, which could be solved by tools for differentiation by indication together with improved control of dispensing. In addition, there are certain public intervention mechanisms (although very limited experience), such as social impact bonds and the InterVentional PharmacoEconomics (IVPE) proposal.
CONCLUSIONS: In conclusion, off-patent drug repurposing faces significant financial and regulatory barriers, in which academics/NGOs must play a central role and be supported by governments throughout the entire life cycle. Although steps have been taken in recent years, efforts are still insufficient and structural and political challenges remain.
METHODS: Through a review of the literature and a questionnaire to drug repurposing experts, we capture the entire problematic of off-patent drug repurposing and the different solutions offered by governments from the moment a medicine candidate for repurposing is found until that medicine obtains a new indication (lifecycle perspective).
RESULTS: There is sufficient public funding for the early stages of R&D, where opportunities can also be identified from off-label uses, RWE studies and computational models. Even so, funding is limited for robust clinical trials and, added to the fact that R&D is led by academics/NGOs with academic objectives rather than regulatory objectives, the probability of success in obtaining marketing authorisation is usually less than 30%. As solutions, research should focus on areas such as rare diseases and unmet health needs. Governments should play a more prominent role in public investment and promote public-private partnerships to provide financial and regulatory support (with Marketing Authorisation Holders support) to academics/NGOs. Finally, a major problem is pharmacy substitution in dispensing at pharmacies, which could be solved by tools for differentiation by indication together with improved control of dispensing. In addition, there are certain public intervention mechanisms (although very limited experience), such as social impact bonds and the InterVentional PharmacoEconomics (IVPE) proposal.
CONCLUSIONS: In conclusion, off-patent drug repurposing faces significant financial and regulatory barriers, in which academics/NGOs must play a central role and be supported by governments throughout the entire life cycle. Although steps have been taken in recent years, efforts are still insufficient and structural and political challenges remain.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR34
Topic
Health Policy & Regulatory, Organizational Practices, Study Approaches
Topic Subcategory
Approval & Labeling, Pricing Policy & Schemes, Reimbursement & Access Policy
Disease
Biologics & Biosimilars, Generics, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases