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Policy Implications of the Orphan Drug Designation for Remdesivir to Treat COVID-19

Educational Objective
To understand the policy implications of the Orphan Drug Designation for Remdesivir to treat COVID-19
1 Credit CME

On March 23, 2020, the US Food and Drug Administration (FDA) granted Gilead Science an orphan drug designation for remdesivir to treat coronavirus disease 2019 (COVID-19), even though this status is usually reserved for drugs that treat rare diseases.1 In explaining its decision, the agency stated that at the time of Gilead’s application for designation, there were sufficiently few confirmed cases of COVID-19 in the United States that remdesivir met criteria for designation under the Orphan Drug Act of 1983.1

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Article Information

Corresponding Author: Kao-Ping Chua, MD, PhD, Department of Pediatrics, University of Michigan, 300 N Ingalls St, SPC 5456, Rm 6E18, Ann Arbor, MI 48109-5456 (chuak@med.umich.edu).

Published Online: August 17, 2020. doi:10.1001/jamainternmed.2020.2759

Conflict of Interest Disclosures: None reported.

Funding/Support: Dr Chua is supported by a career development award from the National Institute on Drug Abuse (grant number 1K08DA048110-01). Dr Conti is supported by a grant from the American Cancer Society.

Role of the Funder/Sponsor: The funders had no role in the creation of this article; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.

References
1.
Lupkin  S. FDA Grants Experimental Coronavirus Drug Benefits for Rare Disease Treatments. National Public Radio. March 24, 2020.
2.
Public Citizen. Gilead Must Relinquish Monopoly on Potential Coronavirus Treatment. Accessed April 20, 2020. https://www.citizen.org/news/gilead-must-relinquish-monopoly-on-potential-coronavirus-treatment/
3.
Gilead Sciences. Gilead Sciences Statement on Request to Rescind Remdesivir Orphan Drug Designation. Accessed April 20, 2020. https://www.gilead.com/news-and-press/company-statements/gilead-sciences-statement-on-request-to-rescind-remdesivir-orphan-drug-designation
4.
Food and Drug Administration. Orphan Drug Act - Relevant Excerpts, 2018. Accessed May 12, 2020. https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-act-relevant-excerpts
5.
Chua  KP , Conti  RM . Revocation of Orphan Drug Designation for Extended-Release Buprenorphine Injection: Implications and Next Steps. Health Affairs Blog. March 5, 2020.
6.
Electronic Code of Federal Regulations. Part 316: Orphan Drugs. Accessed May 12, 2020. https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=316&showFR=1&subpartNode=21:5.0.1.1.6.3
7.
Johns Hopkins University and Medicine. Coronavirus Resource Center. Accessed May 12, 2020. https://coronavirus.jhu.edu/map.html
8.
Bagley  N , Berger  M , Chandra  A , Garthwaite  C , Stern  A . The orphan drug act at 30. In: Lerner  J , Stern  S , eds.  Innovation Policy and the Economy, 2018, Volume 19 (National Bureau of Economic Research Innovation Policy and the Economy). Chicago, IL: University of Chicago Press; 2019.
9.
Food and Drug Administration. Orphan drug designations and approvals. Accessed April 1, 2020. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm
10.
Daniel  MG , Pawlik  TM , Fader  AN , Esnaola  NF , Makary  MA .  The orphan drug act: restoring the mission to rare diseases.   Am J Clin Oncol. 2016;39(2):210-213. doi:10.1097/COC.0000000000000251PubMedGoogle ScholarCrossref
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