“People should ask their doctors about the evidence for drugs and devices being prescribed or use, and they should support research and participate,” he says. “In addition, they should encourage their doctors to participate; too many doctors just ‘go with the flow’ rather than demanding high-quality evidence about what they are prescribing and implanting, and actively joining into appropriate clinical trials.”
Dr. Walid Fouad Gellad from the University of Pittsburgh, in Pennsylvania, who recently wrote about accelerated approval and expensive drugs, tells Reuters Health by email, “It’s important for the public to know that the FDA already has a process in place to make it easier to approve drugs that fill a substantial medical need—the FDA already does this. This report shows that in cases of accelerated approval, many drugs are approved based on studies with fewer than 200 people, using designs which don’t even compare the drug to placebo or some other drug.”
“Another major take-away is that many of the so-called confirmatory trials that are meant to prove the efficacy of these drugs are not confirmatory at all—they show a benefit on ‘surrogate’ outcomes but not on the clinical measures that often matter to patients, like quality of life or overall survival,” he says. “This is important because these medications, approved based on small studies, cost big money.”
Dr. James D. Chambers, an investigator at the Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston, tells Reuters Health by email, “I don’t think that the study’s findings should be interpreted as the FDA letting unsafe drugs on the market. What is concerning is that a key aspect of the accelerated approval pathway is not being fulfilled, which could have potentially important consequences for patients taking these drugs.”
He says, “If confirmatory studies are not carried out correctly (with clinical outcomes) or in a timely manner, then one has to question the appropriateness of the FDA granting Accelerated Approval in the first place.”
“Certainly, it seems that the public should wonder why the FDA does not have greater authority to ensure not only that confirmatory studies are performed in a timely manner, but also that they are designed to confirm a drug’s safety using clinical outcomes, not surrogates,” Dr. Chambers says.
References
- Naci H, Smalley KR, Kesselheim AS. Characteristics of pre-approval and post-approval studies for drugs granted accelerated approval by the U.S. Food and Drug Administration. JAMA. 2017 Aug 15;318(7):626–636. doi: 10.1001/jama.2017.9415.
- Zheng SY, Dhruva SS, Redberg RF. Characteristics of clinical studies used for U.S. Food and Drug Administration approval of high-risk medical device supplements. JAMA. 2017 Aug 15;318(7):619–625. doi: 10.1001/jama.2017.9414.
- Califf RM. Balancing the need for access with the imperative for empirical evidence of benefit and risk. JAMA. 2017 Aug 15;318(7):614–616. doi: 10.1001/jama.2017.9412.
