The FDA has released a final guidance document regarding the benefits, risks, planning, and implementation of adaptive clinical study designs used in medical device development. These study designs—which can change as the data rolls in—can be more efficient than traditional, fixed clinical designs, can provide more responsive treatment for enrolled patients, and can better protect the welfare of the test subjects, according to the guidance document, Adaptive Designs for Medical Device Clinical Studies.
“When properly implemented, adaptive design can reduce resource requirements and/or increase the chance of study success,” the FDA wrote in a bulletin about the new guidance.
The FDA defines an adaptive design for a medical device clinical study as one that “allows for prospectively planned modifications based on accumulating study data without undermining the study’s integrity and validity.” The FDA said such modifications should be planned beforehand “in nearly all situations.” Modifications, if unplanned, could weaken the validity of a study by introducing bias, mismatching results, or increasing complexity, the agency said.
However, the FDA added that modifications made after the study begins “can be scientifically valid if they are made without knowledge of the outcome results by treatment.” Adaptive study designs can use interim analyses to check early on if a trial is working, reassess sample size, assign patients to better treatment options, and improve decision making during product development.
The guidance provides tips on how to preserve the integrity of clinical studies, such as using a Data Monitoring Committee, independent statistician, contract research organization, or another independent clinical trial body to safeguard trial participants and make adaptive decisions.
The guidance applies to premarket medical device submissions, including:
- Premarket approval applications
- Premarket notification (510(k)) submissions
- De novo submissions (Evaluation of Automatic Class III Designation)
- Humanitarian Device Exemption applications
- Investigation Device Exemption submissions
In comments submitted to the FDA last year, AdvaMed, a trade organization for medical device manufacturers, had welcomed the initial draft guidance as “comprehensive and helpful,” and described itself as supportive of the agency’s efforts on this front.
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