Drug Repurposing for Rare Diseases: A Role for Academia

Abstract

Background: The European Commission highlights in its Pharmaceutical Strategy the role of academic researchers in drug repurposing, especially in the development of orphan medicinal products (OMPs). This study summarizes the contribution of academia over the last 5 years to registered repurposed OMPs and describes barriers to success, based upon three real world cases.Methods: OMPs granted marketing authorization between January 2016 and December 2020 were reviewed for repurposing and whether the idea originated from academia or industry. Three cases of drug repurposing were selected from different therapeutic areas and stages of development to identify obstacles to success.Results: Thirteen of the 68 OMPs were the result of drug repurposing. In three OMPs, there were two developments such as both a new indication and a modified application. In total, twelve developments originated from academia and four from industry. The three cases showed as barriers to success: lack of outlook for sufficient return of investments (abatacept), lack of regulatory alignment and timing of interaction between healthcare professionals and regulators (etidronate), failure to register an old drug for a fair price, resulting in commercialization as a high priced orphan drug (mexiletine).Conclusion: While the majority of repurposed OMPs originates in academia, a gap exists between healthcare professionals, regulators and industry. Future strategies should aim to overcome these hurdles leading to more patient benefit through sustainable access of repurposed drugs. Potential solutions include improved regulatory and reimbursement knowledge by academia and the right for regulators to integrate new effectiveness data into product labels.

Repurposing empagliflozin in individuals with glycogen storage disease Ib: A value‐based healthcare approach and systematic benefit‐risk assessment

“…The role of the academia in drug repurposing for rare diseases is gaining attention and a framework for the repurposing of established medicines has been developed in the European Union. 22,23 Empagliflozin is wellestablished and safe in its current areas of usage. [24][25][26] According to an international questionnaire study, empagliflozin treatment was reimbursed in 78% of the cases, but mostly on an individual basis, depending on T A B L E 3 Budget impact analysis for 25 mg/day empagliflozin in persons with GSDIb in two nationwide scenarios.…”

A Cross-Sectional Study of Price and Affordability of Drugs for Rare Diseases in Shandong Province, China

“…In China, the health insurance system prefers to include more drugs indicated for multiple diseases because such drugs can be used to treat various diseases and more patients, including rare diseases. Repurposed or repositioned drugs have been used to treat rare diseases and to significantly improve the affordability of rare disease drugs [ 49 , 50 ]. Some drugs with multiple indications can treat rare diseases, although off-label.…”

Development of medicines for rare diseases and inborn errors of metabolism: Toward novel public–private partnerships

“…Following the identification of promising therapeutic compounds, academia traditionally needed to involve pharmaceutical or biotech companies for the further development and commercialization of its inventions to pharmaceutical or biotech companies 12 . This process is facilitated by the academic institute's knowledge transfer offices (KTOs).…”

“…In traditional medicine development, the role of academia has mainly been at its start, propelling innovation—for instance by elucidating pathophysiological pathways and identifying early‐stage (repurposed) medicine candidates 10–12 . The translational stage of development that follows is commonly privatized, which sometimes includes commissioning academics to perform late‐stage trials 10,13 .…”