FDA Commissioner Explains The Importance of Flexibility In Drug Development
By Cyndi Root
Margaret A. Hamburg, M.D., the Commissioner of the Food and Drug Administration (FDA), wrote an opinion on drug development in the FDA Voice. She stated that, just like each individual is different, so are disease states and the drugs that treat them. Therefore, she was not surprised or upset by a recent study in the journal of the American Medical Association (JAMA), which pointed out the varying standards used by the FDA to approve drugs. She is referring to some reactions to the study that put the varying standards in a negative light. Dr. Hamburg concurs that not all FDA approvals are created equally, but they are evaluated by their individual merits without sacrificing quality.
Entitled, “Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012,” the study seeks to explain the importance of clinical trial evidence and its role in new drug approvals. Study authors stress that physicians and patients assume that a drug is safe after FDA approval, but often do not understand how the FDA makes those decisions. Investigators used data on new drugs approved by the federal agency from 2005 to 2012. Trial designs were compared including trial type, number of patients, duration, and other measures. The results showed that the FDA used a wide range of evidence to make decisions.
Dr. Hamburg believes that this is a plus for the agency, and not a negative as some detractors might say. About the study she said, “These results are entirely consistent with our regulatory mandate. We believe varying approaches to clinical studies to support drug approval is good news, not bad.” She added that she would be more concerned about a rigid process.
She wants people to know that patients with life-threatening diseases or conditions are willing to consider all alternatives and would make sacrifices to gain access to new drugs. Dr. Hamburg added that criticism framing the varied approvals misses the mark because a comprehensive clinical trial design is often subjective. One drug may do well with a small number of participants, while another should be a large group in multi-centers. The Commissioner pointed to the Congress in the FDA Modernization Act of 1997 and the FDA Safety and Innovation Act of 2012 as evidence that the agency’s mandate is written in law.
Dr. Hamburg gave a few examples. Imbruvica (ibrutinib) was approved after a trial of 111 people. Elelyso (taliglucerase alfa) was approved after two trials of 56 enrollees. Studies of heart disease drugs often need large numbers in the clinical study. She believes that having flexibility does not indicate a lack of thoroughness but an adherence to high standards.