trial designsThe U.S. Food and Drug Administration (FDA) provides various ways to speed new drugs and medical devices to market. Even though some contest that FDA is too slow when it comes to product approvals, data suggests otherwise. However, in spite of quicker approval turnaround times, some studies raise questions on trial designs for new products sped to market, RAPS reports.

One “fast-track” option, FDA’s accelerated approval pathway, came under question recently due to the use of surrogate measures as outcomes in both confirmatory and preapproval trials. The co-author of one critical article published in JAMA, Huselyn Naci of the department of health policy at the London School of Economics and Political Science, expressed concerns to RAPS. Naci explained: “A key question raised by our study findings is whether the surrogate measures used in the confirmatory trials of drugs granted accelerated approvals are adequate to compensate for the data limitations at the time of approval. Since these drugs enter the market on the basis of surrogate measures that are only ‘reasonably likely’ to predict clinical benefit, it is essential that the required confirmatory trials evaluate clinically meaningful outcomes.”1

The study found that among 22 drugs with 24 indications granted accelerated approval by FDA from 2009 – 2013, efficacy was often confirmed in postapproval trials a minimum of three years after approval. However, “confirmatory trials and approval trials had similar design elements, including reliance on surrogate measures as outcomes.”1 Critics of the study’s findings argue that “the use of surrogate endpoints to predict clinical benefit, as agreed upon by both a sponsor organization and the FDA, has allowed many patients with no other treatment options to sooner reap the benefit of innovative new drugs and biologics. Recent studies have demonstrated that such medicines provide substantial health gains.”1

A second article published in JAMA evaluated trial designs used for FDA approval of high-risk device supplements. The study found that “fewer than half were randomized, blinded, or controlled, and most primary outcomes were based on surrogate end points… suggest[ing] that the quality of studies and data evaluated to support approval by the FDA of modifications of high-risk devices should be improved.” Robert Califf, former acting commissioner of FDA, provided his thoughts on the two studies in an editorial. Califf suggests that both studies “raise concerns about whether the US regulatory system has become too permissive by not requiring traditional randomized controlled trials (RCTs) for postmarketing evaluation of drugs approved through the accelerated approval pathway and for supplemental design medications of medical devices thought to be sufficiently risky to warrant human studies… [and] provide empirical evidence that the current regulatory system does not always impose the most rigorous standard of clinical evidence.”1

Pearl Pathways routinely helps drug and device companies take advantage of FDA’s accelerated approval pathways. Contact us today to speak with our nimble team of experts about delivering your product to patients faster.