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
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 uncontrol...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2019-07-01
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| Series: | Clinical and Translational Science |
| Online Access: | https://doi.org/10.1111/cts.12617 |
| _version_ | 1818381549188415488 |
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| author | Anne Vinther Morant Vivien Jagalski Henrik Tang Vestergaard |
| author_facet | Anne Vinther Morant Vivien Jagalski Henrik Tang Vestergaard |
| author_sort | Anne Vinther Morant |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-14T02:36:20Z |
| format | Article |
| id | doaj.art-5c742bd3825e4803aa3a267aaa370037 |
| institution | Directory Open Access Journal |
| issn | 1752-8054 1752-8062 |
| language | English |
| last_indexed | 2024-12-14T02:36:20Z |
| publishDate | 2019-07-01 |
| publisher | Wiley |
| record_format | Article |
| series | Clinical and Translational Science |
| spelling | doaj.art-5c742bd3825e4803aa3a267aaa3700372022-12-21T23:20:08ZengWileyClinical and Translational Science1752-80541752-80622019-07-0112436137010.1111/cts.12617Characteristics of Single Pivotal Trials Supporting Regulatory Approvals of Novel Non‐orphan, Non‐oncology Drugs in the European Union and United States from 2012−2016Anne Vinther Morant0Vivien Jagalski1Henrik Tang Vestergaard2H. Lundbeck A/S Valby DenmarkH. Lundbeck A/S Valby DenmarkH. Lundbeck A/S Valby DenmarkFor 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.https://doi.org/10.1111/cts.12617 |
| spellingShingle | Anne Vinther Morant Vivien Jagalski Henrik Tang Vestergaard 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 Clinical and Translational Science |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| url | https://doi.org/10.1111/cts.12617 |
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