Studies Find No Consistency in FDA Drug Approval Methods

Friday, January 24, 2014

Before approving a new drug or medical device, the Food and Drug Administration (FDA) ensures it is “safe and effective” for Americans. But the FDA comes to this conclusion by applying differing standards during its approval process, according to new studies published in a leading medical journal.


“Not all FDA approvals are created equally,” Nicholas Downing at Yale University’s School of Medicine and the studies’ lead author told The Washington Post.


Downing and his colleagues examined nearly 200 new drug approvals between 2005 and 2012. They reported in the Journal of the American Medical Association (JAMA) that the FDA doesn’t apply the same standard of evidence to all drugs going through clinical trial, and instead approve medications based on criteria that vary widely from case to case.


The researchers also found that the agency often approves new versions of existing heart devices without requiring a clinical trial.


Federal regulators also have ignored early problems in drug trials that later resulted in significant delays in the approval of new medications, according to the studies.


“The FDA walks a tightrope, and until now we haven't had a huge amount of information about how they're doing that balancing act,” Dr. Steven Goodman, a Stanford professor of medicine who wrote an accompanying editorial in JAMA, told HealthDay.


“The criteria they are using could be perfect, in that they are being flexible and allowing innovation and getting lifesaving drugs through to market. The other side is they could be making decisions too precipitously and allowing potentially dangerous medications through. It's a difficult balancing act that requires constant monitoring,” Goodman added.


FDA officials responded to the studies by saying their standard for approval does not change, even though they do tailor clinical trials depending on the drug and the disease it is intended to treat.


“The agency applies the same statutory approval standards of safety and efficacy to all drugs, but uses regulatory flexibility in applying those standards,” spokeswoman Stephanie Yao said in a statement. “Some drugs may be tested in clinical trials that enroll hundreds of participants while others, particularly those seeking to treat rare diseases, may be tested in trials that enroll only a handful of participants.”


Downing does not believe the FDA should use a one-size-fits-all approval process, nor does he want to imply that the agency has approved the wrong drugs.


“It’s very understandable that regulators have a flexible standard for approval, but patients and doctors need to be aware the standard is flexible,” he told the Post. “It’s important for doctors and patients to have a discussion about how much we know about the potential benefits or risks of taking a drug before they take it…[and] it underscores the need to continue to study these drugs after approval.”

-Noel Brinkerhoff


To Learn More:

FDA Approval Process Comes Under Scrutiny in Studies (by Dennis Thompson, HealthDay)

FDA’s ‘Safe and Effective’ Drug Approvals Based on Widely Varied Data, Study Finds (by Brady Dennis, Washington Post)

Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012 (Nicholas S. Downing, Jenerius A. Aminawung, Nilay D. Shah, Harlan M. Krumholz, and Joseph S. Ross, Journal of the American Medical Association)


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