Perceived Impact and Actionability of Barriers to Drug Repurposing: Insights From a Multistakeholder Policy Survey
Author(s)
Kristóf Gyöngyösi, PharmD1, Zsuzsanna Ida Petykó, MSc, MD, PhD1, Dalma Hosszú, MA2, Pan Pantziarka, PhD3, Helene G. van der Meer, PharmD, PhD4, Donald Lo, PhD5, Marcell Csanádi, MSc, PhD6, George Dennis Obeng, Msc, MPH6, Zoltán Kaló, MD, MSc, PhD1, Andras Inotai, PharmD, 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, 3Anticancer Fund, Meise, Belgium, 4ZonMw, The Hague, Netherlands, 5European Infrastructure for Translational Medicine (EATRIS), Amsterdam, Netherlands, 6Syreon Research Africa, Accra, Ghana.
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, 3Anticancer Fund, Meise, Belgium, 4ZonMw, The Hague, Netherlands, 5European Infrastructure for Translational Medicine (EATRIS), Amsterdam, Netherlands, 6Syreon Research Africa, Accra, Ghana.
OBJECTIVES: Exploring new therapeutic areas for existing medicines continues to provide important impact. Although drug repurposing (DR) offers a faster and more affordable alternative to de novo drug development, barriers to successful DR remain pervasive across the drug development ecosystem. A systematic literature review (SLR) undertaken by the REMEDi4ALL Horizon Europe project identified 33 policy-related barriers hindering the success of repurposed medicines (RMs). This study presents the process by which the identified barriers were prioritized with the involvement of multiple stakeholder groups.
METHODS: An online policy survey was conducted among stakeholder groups across the DR ecosystem. Participants were asked to weigh the impact and actionability of all barriers from their perspectives, using 5-point categorical scales. A weighted scoring method was used to prioritize the identified policy barriers by combining scores across domains, while ensuring that top-ranked barriers from each stakeholder group were retained in the final shortlist.
RESULTS: 60 individual responses were collected from experts. The final shortlist contained 22 barriers, including 4 barriers related to exclusivity rights for RMs, 2 to pricing of RMs, 5 to market authorization of RMs, 2 to perception of repurposing of off-patent medicines, 2 to business case for repurposing off-patent medicines, 2 to non-industry funded DR, 2 to health technology assessment, 2 to ecosystem for non-profit or small-medium sized enterprises-driven DR, and 1 to business case for repurposing on-patent compounds. Interestingly, the highest ranked barriers varied across different stakeholder groups, with the patient representatives’ preference showing the greatest divergence from other groups’ preferences.
CONCLUSIONS: Prioritization of barriers can facilitate the development of solutions by focusing on the most critical challenges first. Recognition of stakeholder groups’ different perceptions on the impact and actionability of barriers is essential in creating a shared multi-stakeholder understanding when developing policy recommendations to address the most pressing obstacles to DR.
METHODS: An online policy survey was conducted among stakeholder groups across the DR ecosystem. Participants were asked to weigh the impact and actionability of all barriers from their perspectives, using 5-point categorical scales. A weighted scoring method was used to prioritize the identified policy barriers by combining scores across domains, while ensuring that top-ranked barriers from each stakeholder group were retained in the final shortlist.
RESULTS: 60 individual responses were collected from experts. The final shortlist contained 22 barriers, including 4 barriers related to exclusivity rights for RMs, 2 to pricing of RMs, 5 to market authorization of RMs, 2 to perception of repurposing of off-patent medicines, 2 to business case for repurposing off-patent medicines, 2 to non-industry funded DR, 2 to health technology assessment, 2 to ecosystem for non-profit or small-medium sized enterprises-driven DR, and 1 to business case for repurposing on-patent compounds. Interestingly, the highest ranked barriers varied across different stakeholder groups, with the patient representatives’ preference showing the greatest divergence from other groups’ preferences.
CONCLUSIONS: Prioritization of barriers can facilitate the development of solutions by focusing on the most critical challenges first. Recognition of stakeholder groups’ different perceptions on the impact and actionability of barriers is essential in creating a shared multi-stakeholder understanding when developing policy recommendations to address the most pressing obstacles to DR.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR158
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