Quality of FDA Device Approval Studies Examined

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Approving high-risk medical device modifications and granting accelerated drug approval requires high-quality evidence.
Approving high-risk medical device modifications and granting accelerated drug approval requires high-quality evidence.

HealthDay News — Many studies used to support US Food and Drug Administration approval of high-risk medical device modifications are not controlled; and efficacy of drugs granted accelerated approval is often confirmed 3 years after approval, according to 2 studies published in the issue of the Journal of the American Medical Association.

Sarah Y. Zheng, MD, from the University of California in San Francisco, and colleagues characterized the quality of the clinical studies and data used to support FDA approval of panel-track supplements.1 The researchers found that 83 clinical studies supported the approval of 78 panel-track supplements, with 91% supported by a single study. There were 150 primary end points, and 38% were compared with controls. Of the primary end points with controls, 6 and 51 were retrospective and active controls, respectively.

Huseyin Naci, PhD, MHS, from the London School of Economics, and colleagues characterized preapproval and confirmatory clinical trials of drugs granted accelerated approval.2 The researchers found that the FDA granted approval to 22 drugs for 24 indications between 2009 and 2013. Thirty preapproval studies supported the 24 indications. The median number of participants enrolled in preapproval studies was 132. Fifty percent of the 38 required confirmatory studies were completed at a minimum 3 years of follow-up.

"It is now time to seriously consider how to use these increasingly robust data and analytic capabilities and more efficient prospective research systems to deal with the issues raised in these articles," write the authors of an accompanying editorial.3

References

  1. Zheng SY, Dhruva SS, Redberg RF. Characteristics of clinical studies used for US Food and Drug Administration approval of high-risk medical device supplements. JAMA. 2017;318(7)619-625. doi:10.1001/jama.2017.9414
  2. Naci H, Smalley KR, Kesselheim AS. Characteristics of preapproval and postapproval studies for drugs granted accelerated approval by the US Food and Drug Administration. JAMA. 2017;318(7):626-636. doi:10.1001/jama.2017.9415
  3. Califf RM. Balancing the need for access with the imperative for empirical evidence of benefit and risk. JAMA. 2017;318(7):614-616. doi:10.1001/jama.2017.9412

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