“Based on our analysis, some drugs are approved on the basis of large, high-quality clinical trials, while others are approved based on results of smaller trials,” said senior author Dr. Joseph Ross, assistant professor of internal medicine at Yale School of Medicine.
“There was a lack of uniformity in the level of evidence the FDA used.”
The research, published in the journal JAMA, is the first systematic analysis to look at the FDA’s standards in making drug approval decisions.
“We found that during the study period, more than one-third of the drugs were approved on the basis of a single trial, without replication, and many other trials were small, short, and focused on lab values, or some other surrogate metric of effect, rather than clinical endpoints like death,” said first author and Yale School of Medicine student Nicholas S. Downing.
For the study, the researchers evaluated the strength of clinical trial evidence supporting FDA approval decisions for new drugs. They used publicly available FDA documents to identify 188 new medications between the years of 2005-2012.
These medical review documents summarized in great detail the rationale behind FDA approvals.
“We also found that only 40 percent of drug approvals involved a clinical trial that compared a new drug to existing treatment offerings. This is an important step for determining whether the new drug is a better option than existing, older drugs,” said Ross.
Downing added that survey data shows that patients expect drugs approved by the FDA to be both safe and effective.
“Based on our study of the data, we can’t be certain that this expectation is necessarily justified, given the quantity and quality of the variability we saw in the drug approval process,” he said.
Other authors on the study include Jenerius A. Aminawung, M.D., Nilay D. Shah, and Harlan M. Krumholz, M.D.
Source: Yale University