Abstract
Drug repurposing is promoted as a cost- and time-effective mechanism for providing new medicines. Often, however, there is insufficient consideration by academic researchers of the processes required to ensure that a repurposed drug can be used for a new indication. This may explain the inability of drug repurposing to fulfill its promise. Important aspects, often overlooked, include financial and intellectual property considerations, the clinical and regulatory path, and clinical equipoise, which provides ethical justification for randomized controlled trials. The goal of drug repurposing is to obtain a new regulator-approved label for an existing drug, and so, the trajectory for drug repurposing and traditional drug development is similar. Here, we discuss factors critical for a successful repurposed medicine to help academic investigators better identify drug repurposing opportunities.
| Original language | English |
|---|---|
| Article number | eabd5524 |
| Number of pages | 13 |
| Journal | Science Translational Medicine |
| Volume | 13 |
| Issue number | 612 |
| DOIs | |
| Publication status | Published - 22 Sept 2021 |
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In: Science Translational Medicine, Vol. 13, No. 612, eabd5524, 22.09.2021.
Research output: Contribution to journal › Review Article › Research › peer-review
TY - JOUR
T1 - Drug repurposing
T2 - Misconceptions, challenges, and opportunities for academic researchers
AU - Begley, C. Glenn
AU - Ashton, Mark
AU - Baell, Jonathan
AU - Bettess, Michael
AU - Brown, Michael P.
AU - Carter, Brett
AU - Charman, William N.
AU - Davis, Christopher
AU - Fisher, Simon
AU - Frazer, Ian
AU - Gautam, Anand
AU - Jennings, Michael P.
AU - Kearney, Philip
AU - Keeffe, Eloise
AU - Kelly, Darren
AU - Lopez, Angel F.
AU - McGuckin, Michael
AU - Parker, Michael W.
AU - Rayner, Craig
AU - Roberts, Brett
AU - Rush, James S.
AU - Sullivan, Mark
N1 - Funding Information: We thank the reviewers for their thoughtful comments. J.B., M.P.B., M.P.J., I.F., D.K., M.M., M.W.P., and A.F.L. are supported by research grants from the Australian National Health and Medical Research Council. M.W.P. is supported by research grants from the Australian Research Council. Funding Information: A good example of drug repurposing by a not-for-profit organization is moxidectin, which was discovered in the 1980s and used as a veterinary anti-helminthic drug. Clinical development was driven by Tropical Diseases Research at the WHO and Medicines Development for Global Health, with funding from the Global Health Investment Fund. This collaboration used the FDA’s Priority Review Voucher, a mechanism allowing expedited review of a new drug product. Vouchers are transferable, and their sale provided the funds for moxidectin’s development. This resulted in moxidectin’s approval in 2018 for treating the disease river blindness caused by the helminthic worm Onchocerca (20). Further research led to an extension of use for moxidectin in the treatment of five other neglected tropical diseases. Moxidectin’s success demonstrates the value of collaboration and partnership between academic groups and philanthropies, and how working together with regulatory authorities resulted in new therapies for neglected diseases. The successful repurposing of moxidectin from a veterinary drug to a human therapeutic should not be taken as a general example of the ease of repurposing an approved veterinary medicine. The approved veterinary anti-helminthic drug, monepantel, was reported to have anticancer action (21), but evaluation of monepantel’s possible repurposing as a human cancer therapeutic required a standard clinical development program beginning with a first-in-human clinical trial (22). Funding Information: A number of regulatory mechanisms have been established that acknowledge both the importance of drug repurposing efforts and the central role of academic investigators. These include periods of guaranteed market exclusivity, FDA Priority Review Vouchers that allow expedited review of a new drug product, and the FDA’s 505(b) (2) regulatory pathway. Some important repurposing studies will only be undertaken by academic investigators and particularly for drugs where there is no opportunity to recoup investment. An excellent example is the study of all-cause mortality of aspirin in the healthy elderly. Researchers studied more than 19,000 individuals, supported by government grants from the National Institute on Aging, National Cancer Institute, National Institutes of Health, the Australian National Health and Medical Research Council, and Monash University. Although that study might not have resulted in a change in the approved label for aspirin, its publication provided evidence that physicians now can use when balancing risk and benefit for their patients (31). Publisher Copyright: © 2021 The Authors, some rights reserved Copyright: Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2021/9/22
Y1 - 2021/9/22
N2 - Drug repurposing is promoted as a cost- and time-effective mechanism for providing new medicines. Often, however, there is insufficient consideration by academic researchers of the processes required to ensure that a repurposed drug can be used for a new indication. This may explain the inability of drug repurposing to fulfill its promise. Important aspects, often overlooked, include financial and intellectual property considerations, the clinical and regulatory path, and clinical equipoise, which provides ethical justification for randomized controlled trials. The goal of drug repurposing is to obtain a new regulator-approved label for an existing drug, and so, the trajectory for drug repurposing and traditional drug development is similar. Here, we discuss factors critical for a successful repurposed medicine to help academic investigators better identify drug repurposing opportunities.
AB - Drug repurposing is promoted as a cost- and time-effective mechanism for providing new medicines. Often, however, there is insufficient consideration by academic researchers of the processes required to ensure that a repurposed drug can be used for a new indication. This may explain the inability of drug repurposing to fulfill its promise. Important aspects, often overlooked, include financial and intellectual property considerations, the clinical and regulatory path, and clinical equipoise, which provides ethical justification for randomized controlled trials. The goal of drug repurposing is to obtain a new regulator-approved label for an existing drug, and so, the trajectory for drug repurposing and traditional drug development is similar. Here, we discuss factors critical for a successful repurposed medicine to help academic investigators better identify drug repurposing opportunities.
UR - http://www.scopus.com/inward/record.url?scp=85116814876&partnerID=8YFLogxK
U2 - 10.1126/scitranslmed.abd5524
DO - 10.1126/scitranslmed.abd5524
M3 - Review Article
C2 - 34550729
AN - SCOPUS:85116814876
SN - 1946-6234
VL - 13
JO - Science Translational Medicine
JF - Science Translational Medicine
IS - 612
M1 - eabd5524
ER -