Catalogue of Actions to Address Policy Barriers of Drug Repurposing
Author(s)
Andras Inotai, PharmD, PhD1, Dalma Hosszú, MA2, Kristóf Gyöngyösi, PharmD1, Zsuzsa Reka Pozsar, MA1, Menke Winckers, MSc3, Donald Lo, PhD4, Pan Pantziarka, PhD5, Patricia Vandamme, MPharmSc5, Helene G. van der Meer, PharmD, PhD6, Dunja Huijbers, MSc6, Raluca Radu, PharmD7, Maria Merkou, LLM7, Zoltan Kalo, MD, MSc, PhD1.
1Center for Health Technology Assessment, Semmelweis University, Center for Pharmacology and Drug Research & Development, Semmelweis University, Syreon Research Institute, Budapest, Hungary, 2Syreon Research Institute, Institute of Psychology, University of Pécs, Budapest, Hungary, 3Syreon Research Institute, Utrecht University, Erasmus University Rotterdam, Rotterdam, Netherlands, 4European Infrastructure for Translational Medicine (EATRIS), Amsterdam, Netherlands, 5Anticancer Fund, Meise, Belgium, 6ZonMw, The Hague, Netherlands, 7Medicines for Europe, Brussels, Belgium.
1Center for Health Technology Assessment, Semmelweis University, Center for Pharmacology and Drug Research & Development, Semmelweis University, Syreon Research Institute, Budapest, Hungary, 2Syreon Research Institute, Institute of Psychology, University of Pécs, Budapest, Hungary, 3Syreon Research Institute, Utrecht University, Erasmus University Rotterdam, Rotterdam, Netherlands, 4European Infrastructure for Translational Medicine (EATRIS), Amsterdam, Netherlands, 5Anticancer Fund, Meise, Belgium, 6ZonMw, The Hague, Netherlands, 7Medicines for Europe, Brussels, Belgium.
OBJECTIVES: Exploring new therapeutic areas for established medicines has been gaining recognition, as it offers potentially faster, and more affordable alternatives to de novo drug development. Previous research in the REMEDi4ALL Horizon Europe project had identified policy-related barriers hindering the success of repurposed medicines (RMs). This study aims to identify push and pull mechanisms that could incentivize drug repurposing (DR) by addressing these barriers.
METHODS: Facilitating mechanisms were retrieved i) from the scientific literature, ii) prior good practices of R&D incentives e.g. in rare, neglected and tropical diseases, and iii) successful reference cases of DR. Mechanisms were compiled into a catalogue of actions (CoA) until saturation was reached. Identified mechanisms were divided into two categories. Push incentives aim to reduce the cost, time, and risk of R&D, while pull incentives aim to improve the financial and societal return for investors.
RESULTS: We identified 10 types of push incentives, including specific regulatory pathways for RMs, increased regulatory know-how and accelerated timelines and regulatory fee reductions or waived fees, enabling label extension initiated by third parties, exploring complementary evidence generation options for RMs, providing education of supply-side stakeholders, increase provision of different funding methods- and types of funders for R&D of RMs. We also identified 9 types of pull incentives, including exclusivity-type regulatory incentives for RMs, monitoring off-label use and bringing off-label use to on-label where possible, developing appropriate health technology assessment frameworks for RMs, differentiating pricing of RMs from pricing rules of on-patent and generic medicines, reducing time and cost of post authorization processes for RMs, providing education of demand-side stakeholders, compensation for successful R&D of RMs and increasing demand for RMs.
CONCLUSIONS: As a next step, the CoA will be validated by different stakeholder groups, aligned with previously identified policy barriers, and transformed into actionable policy recommendations.
METHODS: Facilitating mechanisms were retrieved i) from the scientific literature, ii) prior good practices of R&D incentives e.g. in rare, neglected and tropical diseases, and iii) successful reference cases of DR. Mechanisms were compiled into a catalogue of actions (CoA) until saturation was reached. Identified mechanisms were divided into two categories. Push incentives aim to reduce the cost, time, and risk of R&D, while pull incentives aim to improve the financial and societal return for investors.
RESULTS: We identified 10 types of push incentives, including specific regulatory pathways for RMs, increased regulatory know-how and accelerated timelines and regulatory fee reductions or waived fees, enabling label extension initiated by third parties, exploring complementary evidence generation options for RMs, providing education of supply-side stakeholders, increase provision of different funding methods- and types of funders for R&D of RMs. We also identified 9 types of pull incentives, including exclusivity-type regulatory incentives for RMs, monitoring off-label use and bringing off-label use to on-label where possible, developing appropriate health technology assessment frameworks for RMs, differentiating pricing of RMs from pricing rules of on-patent and generic medicines, reducing time and cost of post authorization processes for RMs, providing education of demand-side stakeholders, compensation for successful R&D of RMs and increasing demand for RMs.
CONCLUSIONS: As a next step, the CoA will be validated by different stakeholder groups, aligned with previously identified policy barriers, and transformed into actionable policy recommendations.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
P51
Topic
Health Policy & Regulatory, Methodological & Statistical Research, Study Approaches
Topic Subcategory
Approval & Labeling, Pricing Policy & Schemes, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas