How should we design future mechanistic and/or efficacy clinical trials?
Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology 2024 DOI: 10.1038/s41386-023-01600-9 (opens in new tab)
Study at a glance
AI-extracted from the abstract| Characteristics | Review Peer reviewed |
|---|---|
| Citations | 22 |
| Key findings | The review identifies limitations in current neuropsychiatric clinical trials and presents methodological approaches, such as the sequential parallel comparison design, to improve their efficiency and precision. |
Abstract
The emergence of new molecular targets, together with the development of new approaches to neuropsychiatric diseases, involving psychedelics as well as gene and cell therapies, are creating the need to improve the efficiency of mechanistic and/or efficacy clinical trials. This review article will discuss a number of issues that have hampered our ability to detect therapeutic signals, from excessive placebo/sham response rates to the imprecision of diagnostic and outcome assessments. In addition to reviewing the limitations of current efficacy and mechanistic neuropsychiatric clinical trials, this review presents some of the methodological approaches that may improve the overall performance of our neuropsychiatric trials, including the adoption of novel study designs such as the sequential parallel comparison design and independent confirmation of the appropriateness of subjects' enrollment. In addition, this review will discuss several designs that make mechanistic clinical trials more precise.