Delays in confirmatory trials for drug applications granted FDA’s accelerated approval raise concerns
- Collection:
- Health Policy and Services Research
- Series Title(s):
- Data snapshot (United States. Department of Health and Human Services. Office of Inspector General)
- Contributor(s):
- United States. Department of Health and Human Services. Office of Inspector General. Office of Evaluation and Inspections, issuing body.
- Publication:
- [Washington, D.C.] : U.S. Department of Health and Human Services, Office of Inspector General, September 2022
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Drug Approval Legislation, Drug United States United States. Food and Drug Administration
- Genre(s):
- Technical Report
- Abstract:
- The Food and Drug Administration’s (FDA) 2021 approval of aducanumab (Aduhelm), a drug intended to treat Alzheimer’s disease raised concerns about FDA’s accelerated approval pathway. Subsequently, the Office of Inspector General (OIG) announced plans to conduct evaluations of the accelerated approval pathway. This data snapshot focuses on confirmatory trials, given ongoing concerns that sponsors of drug applications granted accelerated approval fail to complete their statutorily required confirmatory trials on schedule, and concerns that FDA’s oversight of the trials is lax. The accelerated approval pathway allows FDA to approve certain drugs that treat serious or life-threatening diseases and offer meaningful therapeutic benefit to patients over existing treatments before confirmatory trials are completed. To gain approval in this pathway, drug sponsors must meet the same FDA standards for safety and efficacy as traditionally approved drugs. However, they may rely on a surrogate or intermediate endpoint and do not need to show clinical benefit before approval, rather that the predicted clinical benefit is reasonably likely. Federal law grants FDA the authority to require sponsors to conduct confirmatory trials after approval to verify that the drugs provide the predicted clinical benefit. Typically, sponsors conduct such trials while these drugs are available to the public on a timeline agreed to by FDA and the sponsor.
- Copyright:
- The National Library of Medicine believes this item to be in the public domain. (More information)
- Extent:
- 1 online resource (1 PDF file (12 pages))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918681584106676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918681584106676