FDA clinical trial pauses are increasing year by year, and advanced cell and gene therapy clinical pauses require more transparency

As the pharmaceutical industry gradually turns to exploring promising but unproven frontier technologies, the US FDA is increasingly asking pharmaceutical companies to stop trials
of investigational drugs.

Permission from the FDA is often required before companies begin human trials, and the FDA has long used its statutory authority to require companies to suspend trials
for safety reasons.
According to a recent Wall Street Journal review of the FDA Review of clinical pause data, as biotech companies pursue more cutting-edge cell and gene therapy approaches, the FDA has issued more clinical trial pauses than ever before
.

The FDA reportedly suspended clinical trials of experimental drugs an average of 664 times a year from 2017 to 2021, up from 557 per year in the previous five years
.
Last year as of 12 In the middle of the month, the FDA had suspended 747 clinical trials
.

New gene and cell technologies now promise breakthroughs in challenging diseases, but their development also carries risks
.
The deaths of some young subjects and other safety fears have cast a shadow
over trials of gene therapies aimed at replacing defective genes.
New technologies require regulators to invest more time and effort to keep up with complex technological advances
.

Under federal regulations, FDA may stop clinical trials for the following reasons: to protect study subjects from unreasonable risks of illness or injury, or for defective
study design.
FDA A clinical trial suspension order issued may organize the start of a study or the addition of new subjects, or may require volunteers to stop continuing to participate in the study
.
FDA The clinical suspension will not be publicly disclosed when the clinical suspension order is issued, and the sponsor is not required to do so
.
But sometimes the sponsor will voluntarily disclose
.

In a study[1] published in preprint last June, 39 were analyzed anticancer drug trials
.
The researchers believe that the moratorium does not necessarily hinder the final approval of the drug, and may lead to better drugs
.
"But the clinical trial moratorium could lead to a significant decline in investor interest, especially for small biotech companies
.
"

Some clinical trials have been suspended or even sparked shareholder lawsuits accusing companies of withholding information about the suspension or delaying sharing information
about the suspension.
Some investors have criticized certain companies for misrepresenting the FDA's
reasons for requesting the suspension.
FDA A transparency task force called on the FDA in 2010 to disclose clinical trial suspensions and liftings, but that recommendation has not yet been implemented
.

According to the Wall Street Journal analysis, CDER, the FDA's division that approves new drugs, and CBER, which approves new biologics, including vaccines, have both issued more trial moratoriums
in recent years.
FDA The spokesperson said the increase in clinical trial pauses issued by CBER, the FDA's division that reviews macromolecules, may be due to more new cell and gene therapy clinical trials
.
The FDA said, "FDA We will continue to work with product developers and researchers to facilitate the development of new potential treatments, including ensuring that study participants are not exposed to unreasonable risks in early-stage clinical trials and that study design is consistent with their goals
.
”

But analysts say the increase in test moratoriums may also be partly due to an increase
in the number of new applications in recent years.
According to the analysis of FDA data, from 2014 to 2021 The number of requests for clinical trials with experimental drugs increased by 43% year, while clinical trial pauses increased by 66%
during that period.

[1] Snyder, A.
, De Alwis, D.
, Goonewardene, A.
, & Hegde, P.
(2022).
Clinical Holds in Early Oncology Drug Development.
medRxiv.