Characteristics of Single Pivotal Trials Supporting Regulatory Approvals of Novel Non‐orphan, Non‐oncology Drugs in the European Union and United States from 2012−2016
| Autor: | Vivien Jagalski, Anne Vinther Morant, Henrik Tang Vestergaard |
|---|---|
| Rok vydání: | 2019 |
| Předmět: |
medicine.medical_specialty
MEDLINE Article General Biochemistry Genetics and Molecular Biology Food and drug administration Primary outcome Drug approval Humans Medicine media_common.cataloged_instance European Union General Pharmacology Toxicology and Pharmaceutics European union Intensive care medicine Drug Approval media_common Clinical Trials as Topic business.industry lcsh:Public aspects of medicine Research General Neuroscience lcsh:RM1-950 Gold standard lcsh:RA1-1270 Articles General Medicine United States Social Control Formal Clinical trial lcsh:Therapeutics. Pharmacology business Oncology drugs |
| Zdroj: | Clinical and Translational Science Clinical and Translational Science, Vol 12, Iss 4, Pp 361-370 (2019) |
| ISSN: | 1752-8062 1752-8054 |
| Popis: | For regulatory approval of a new medicine, the gold standard for demonstration of efficacy has traditionally been a minimum of two positive, adequate, and well‐controlled clinical trials. Nevertheless, drugs to treat cancer and rare diseases are usually approved based on a single and often uncontrolled pivotal trial. In contrast, little is known about single pivotal trial approvals for non‐orphan, non‐oncology drugs. Between 2012 and 2016, 23 novel therapeutic drugs were approved by the US Food and Drug Administration (FDA) and/or the European Medicines Agency (EMA) for 27 non‐orphan, non‐oncology indications each based on a single pivotal trial. Although there was considerable variation in the nature and strength of the efficacy evidence supporting these drug approvals, the majority (85%) of the pivotal trials were randomized and controlled. For all superiority trials, the primary outcome was met with a statistical significance of P ≤ 0.005. Most approvals were supported by additional efficacy data from nonpivotal studies. |
| Databáze: | OpenAIRE |
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