Drugs

Review of Drug Trials with External Controls

Researchers in pharmaceutical and academic settings say FDA regulatory guidance should be updated to acknowledge the utility of external controls in drug trials, particularly retrospective natural history data. Writing in an open-access article in Therapeutic Innovation and Regulatory Science, the authors discuss their review of 45 FDA approvals between 2000 and 2019 in which the agency accepted external control data for the benefit/risk assessment.

The researchers found that retrospective natural history data, including retrospective reviews of patient records, was the most common source of external control (44%), followed by baseline control (33%), published data (11%), and data from a previous study (11%).

The study notes that none of the cases where external controls were used were sourced from prospectively collected natural history data. The authors say this is not surprising given the difficulty of performing a meaningful prospective natural history study in a realistic time frame.

“This is a critical point,” the authors say, “given that the regulatory precedent is contrary to the FDA guidance that identifies prospective natural history as the gold standard while discouraging the use of retrospective natural history. While prospective natural history studies may be ideal, such an approach is often impractical, would lead to significant delays in the availability of life-saving therapies, and could ultimately stifle the development of products for rare diseases.”

FDA has recently communicated, the study says, that it is less swayed by the size of natural history studies than by the rigor of data collection and clarity on the course of the disease, and has recommended the use of longitudinal rather than cross-sectional data sources, as they yield more comprehensive information about disease onset and progression over time.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome