Insufficient Clinical Trials Delay FDA Drug Approval Process
By Cyndi Root
A new study published in The Journal of the American Medical Association (JAMA) suggests that physicians and patients misunderstand how the Food and Drug Administration (FDA) makes decisions. They assume that drugs are safe and effective, but varying degrees of clinical evidence inform drug approval. The purpose of the new study was to examine efficacy trials that the FDA relies on to approve novel therapeutic agents. Authors concluded that drug companies can improve FDA approval rates by improving clinical study designs.
The FDA often denies new drug applications because sponsors have failed to include adequate clinical data. The drug discovery process takes an average of eight years, with only one in six drugs approved. Some of the delay is due to FDA bureaucracy, delays, and failures. The FDA processes cost businesses time and money, and patients remain underserved. Therefore, the study suggests that drug companies rework the studies and processes in order to facilitate faster and more efficient travel through the federal agency.
Study authors wrote, “It is advantageous to identify products that fail as early as possible in the development process to avoid these issues.” They identified one important reason for early failure, which was non-specificity of the optimal drug dose. FDA reviewers are more likely to approve a drug if sponsors can prove that a particular dose maximizes the treatment effect without increasing danger or side effects. Clinical trial end points are another sticking point for the FDA. Without a clear goal like a tumor shrinking or a person surviving, it is difficult for reviewers to affirm that the drug works. Some clinical trials are considered less valid if sponsors include subjects not appropriate for the trial.
Nicholas S. Downing and Joseph S. Ross, MD, MHS designed the study and completed it with help from other scientists and the Pew Charitable Trusts, the National Heart, Lung, and Blood Institute Cardiovascular Outcomes Center, the National Institute on Aging, and the American Federation for Aging Research.
The Associate Director for Clinical Methodology at the FDA's Office of Medical Policy, Leonard Sacks, also took a look at the approval process. He and colleagues examined applications for new drugs between 2000 and 2012. The research parameters included new molecular entities (NMES) or drugs not approved in the U.S. Researchers examined correspondence, reviews, and summaries between the FDA and drug sponsors. They found that 151 out of 302 applications failed initially. Many of those that failed applications received approval later, but 71 of those drugs took another year or more to earn approval.
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