Dr. Margaret Hamburg, commissioner of the Food and Drug Administration, defended FDA support of a proposal to create a controversial expedited approval process for drugs for serious, often untreatable, conditions.
“People with serious or life-threatening illnesses, particularly those who lack good alternatives, have told us repeatedly that they are willing to make some trade-offs in order to gain access,” Hamburg said in a blog posted Thursday.
“Thoroughness, such as whether a clinical trial is large enough, is in the eyes of the beholder. There is no reason to expect drugs to be tested on similar numbers of patients, regardless of the disease,” she wrote.
Her post came in response to release of a recent study which claimed the FDA had “flexible standards” for approving new therapies. Investigators found that of 188 novel therapies approved between 2005 and 2012, 37% were approved on the basis of a single clinical trial, 38% on the basis of two trials and 25% had been tested in three or more trials. Results were published in January in the Journal of the American Medical Association.
“A number of commentators framed this as criticism. But I would be more troubled if FDA used a rigid, one size fits all approach,” Hamburg said.
Still critics worry fast-tracking would lower standards and pose a threat to patient safety and product quality.
In a letter addressed to Hamburg, Rep. Rosa DeLauro (D-Conn.) expressed concern that the new guidelines would require one small trial, instead of two well-designed controlled clinical trials, which DeLauro says may not necessarily be a measure of a meaningful health outcome.
“The FDA has a high level of regulatory authority and discretion over medical device classification and drug approval methods,” DeLauro said. “The public trusts in FDA to ensure that drugs and medical devices are both safe and effective. Public health and wellness has been, and should continue to be, the primary focus of the FDA.” The letter said permitting smaller sample sizes would make it more difficult to determine safety, efficacy or the proper dosages across sexes, racial and ethnic groups or all age groups. “This information is essential for prescribers and patients,” she said.
The FDA proposal would require drugs approved through the expedited process to contain labeling making it clear the drug is narrowly indicated for use in limited, well-defined subpopulations for which the drug's benefits have been shown to outweigh its risks. But speed is not the answer, others say.
“There's so much emphasis on drugs being the latest, the most innovative and novel—but unfortunately this usually means it's just new, not necessarily better,” Diana Zuckerman, president of the National Research Center for Women & Families, told Modern Healthcare when the JAMA study was released. “It's better to have an old treatment that is proven to be safe and effective than a new treatment that we don't know is safe and may not improve health.”
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