According to the report published in the Wall Street Journal today, the number of clinical holds by the FDA has increased over the last 10 years. These clinical holds are generally due to safety concerns and one reason is increasing number of new class of therapies such as gene therapy.

• 2011 to 2016: average of 557 clinical holds each year
• 2017 to 2021: average of 664 clinical holds each year
• During 2022 (Jan to mid-Dec): 747 clinical holds

Some examples highlighted in the article: clinical hold ordered for sickle-cell gene therapy from Bluebird Bio after a teenage subject developed anemia; clinical hold of Vertex Pharmaceuticals' diabetes cell therapy after the FDA said it needed more information on doses.

IMPLICATION OF CLINICAL HOLD

Sponsor cannot enroll new subjects or may have to stop treating ongoing subjects until the reasons for safety signal are resolved.

Under federal regulations, it [FDA] can halt clinical trials for reasons including to protect study volunteers from unreasonable risk of illness or injury or because a study’s design is deficient.

Interestingly, there is no long-term impact of clinical hold on the approvability of the drug.

There is however an impact on stock price, generally.

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Source: Whyte LE. FDA Increasingly Halting Human Trials as Companies Pursue Risky, Cutting-Edge Drugs. Wall Street Journal. 10 Jan 2023 [archive]

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https://www.wsj.com/articles/fda-increasingly-halting-human-trials-as-companies-pursue-risky-cutting-edge-drugs-11673322324