Applying real-world data from expanded-access ("compassionate use") patients to drug development.
| Autor: | Wasser JS; From the Clinical and Translational Science Institute, Tufts Medical Center and Tufts University School of Medicine, Boston, MA, USA., Greenblatt DJ; From the Clinical and Translational Science Institute, Tufts Medical Center and Tufts University School of Medicine, Boston, MA, USA. |
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| Jazyk: | angličtina |
| Zdroj: | Journal of clinical and translational science [J Clin Transl Sci] 2023 Aug 07; Vol. 7 (1), pp. e181. Date of Electronic Publication: 2023 Aug 07 (Print Publication: 2023). |
| DOI: | 10.1017/cts.2023.606 |
| Abstrakt: | Our drug development process has produced many life-saving medications, but patients experiencing rare diseases and similar conditions often are left with limited options for treatment. For an approved treatment to be developed, research on a new candidate or existing drug must validate safety and efficacy based on contemporary research expectations. Randomized clinical trials are conducted for this purpose, but they are also costly, laborious, and time-consuming. For this reason, The 21 st Century Cures Act mandates that the US Food and Drug Administration look for alternative methods for approving drugs, in particular exploring the uses of real-world data and evidence. Expanded access ("compassionate use") is a pathway for the clinical treatment of patients using drugs that are not yet approved for prescribing in the United States. Using real-world evidence generated from expanded-access patients presents an opportunity to provide critical data on patient outcomes that can serve regulatory approval in conjunction with other observational datasets or clinical trials, and in limited circumstances may be the best data available for regulatory review. In doing so, we may also support and encourage patient-centered care and a personalized medicine approach to drug development. Competing Interests: The authors have no conflicts of interest to declare. (© The Author(s) 2023.) |
| Databáze: | MEDLINE |
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