Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not Proven
Randomized controlled trials (RCTs) are the gold standard for testing the safety and efficacy of new drugs and biologicals. The US Food and Drug Administration (FDA) has proactively improved the trial designs to make them scientifically rational while avoiding unnecessary human exposure. Several new...
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MDPI AG
2024-10-01
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| Series: | Pharmaceuticals |
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| Online Access: | https://www.mdpi.com/1424-8247/17/11/1424 |
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| author | Sarfaraz K. Niazi |
| author_facet | Sarfaraz K. Niazi |
| author_sort | Sarfaraz K. Niazi |
| collection | DOAJ |
| description | Randomized controlled trials (RCTs) are the gold standard for testing the safety and efficacy of new drugs and biologicals. The US Food and Drug Administration (FDA) has proactively improved the trial designs to make them scientifically rational while avoiding unnecessary human exposure. Several new guidelines by the FDA have come in 2024 that address consolidating the RCTs with the Real-World Evidence (RWE) trials, decentralizing the testing platforms, and allowing the point-of-use clinicians to participate. However, the issue of placebo control remains, which is part of RCTs, and it should be reduced or removed given the organic impact of placebo that compounds the efficacy evaluation (explanatory trials), as opposed to effectiveness trials (pragmatic trials), which measure the degree of beneficial effects in “real-world” clinical settings. Additionally, clinical trials with low study power should be allowed, and when the proof of bioavailability at the site of action is not present, it should be removed. It is advised that the FDA issue a comprehensive guideline to consolidate its several guidelines and consider the role of placebo in making drug development a more affordable exercise while meeting the requirement to minimize the abuse of humans in such trials. |
| format | Article |
| id | doaj-art-6a58d62fb1c9489db7e707029d7dafe6 |
| institution | Kabale University |
| issn | 1424-8247 |
| language | English |
| publishDate | 2024-10-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj-art-6a58d62fb1c9489db7e707029d7dafe62024-11-26T18:17:06ZengMDPI AGPharmaceuticals1424-82472024-10-011711142410.3390/ph17111424Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not ProvenSarfaraz K. Niazi0College of Pharmacy, University of Illinois, Chicago, IL 60612, USARandomized controlled trials (RCTs) are the gold standard for testing the safety and efficacy of new drugs and biologicals. The US Food and Drug Administration (FDA) has proactively improved the trial designs to make them scientifically rational while avoiding unnecessary human exposure. Several new guidelines by the FDA have come in 2024 that address consolidating the RCTs with the Real-World Evidence (RWE) trials, decentralizing the testing platforms, and allowing the point-of-use clinicians to participate. However, the issue of placebo control remains, which is part of RCTs, and it should be reduced or removed given the organic impact of placebo that compounds the efficacy evaluation (explanatory trials), as opposed to effectiveness trials (pragmatic trials), which measure the degree of beneficial effects in “real-world” clinical settings. Additionally, clinical trials with low study power should be allowed, and when the proof of bioavailability at the site of action is not present, it should be removed. It is advised that the FDA issue a comprehensive guideline to consolidate its several guidelines and consider the role of placebo in making drug development a more affordable exercise while meeting the requirement to minimize the abuse of humans in such trials.https://www.mdpi.com/1424-8247/17/11/1424clinical trialsFDAmulti-regional trialsintegrated RCT in clinical practiceadaptive trialsdecentralized trials |
| spellingShingle | Sarfaraz K. Niazi Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not Proven Pharmaceuticals clinical trials FDA multi-regional trials integrated RCT in clinical practice adaptive trials decentralized trials |
| title | Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not Proven |
| title_full | Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not Proven |
| title_fullStr | Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not Proven |
| title_full_unstemmed | Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not Proven |
| title_short | Advice to the FDA to Improve Its Proposed Guidelines to Rationalize Clinical Trials by Restricting Placebo Control, Preventing Low-Powered Studies, and Disallowing Studies Where Bioavailability Is Not Proven |
| title_sort | advice to the fda to improve its proposed guidelines to rationalize clinical trials by restricting placebo control preventing low powered studies and disallowing studies where bioavailability is not proven |
| topic | clinical trials FDA multi-regional trials integrated RCT in clinical practice adaptive trials decentralized trials |
| url | https://www.mdpi.com/1424-8247/17/11/1424 |
| work_keys_str_mv | AT sarfarazkniazi advicetothefdatoimproveitsproposedguidelinestorationalizeclinicaltrialsbyrestrictingplacebocontrolpreventinglowpoweredstudiesanddisallowingstudieswherebioavailabilityisnotproven |