FDA's Expedited Drug Approval Process Called Into Question
October 29, 2013

FDA’s Expedited Drug Approval Process Called Into Question

Brett Smith for redOrbit.com - Your Universe Online

If a new treatment is developed that is a major curative advancement and meets a therapeutic need for severe illness, the Food and Drug Administration will expedite the usual lengthy approval process.

However, fewer patients were studied as part of expedited reviews of new drugs in 2008, and some safety concerns about these drugs remain unanswered, according to a new study published in JAMA Internal Medicine.

Study researchers found that drugs expedited in 2008 were tested for effectiveness in a median 104 patients, compared to a median 580 patients through the normal review process. By 2013, many follow-up studies to collect additional evidence on the security of expedited drugs had not been finished, according to the study.

Eight drugs were approved under expedited review and 12 under standard review in 2008. The study researchers also found that expedited drugs took a median of 5.1 years of clinical development to get marketing approval, compared with 7.5 years for the drugs under the standard process.

"The testing of new drugs has shifted from a situation in which most testing was conducted prior to initial approval to a situation in which many innovative drugs are more rapidly approved after a small trial in a narrower patient population with extensive additional testing conducted after approval," the authors wrote. "Our findings suggest that the shift has made it more difficult to balance the benefits and risks of new drugs. Further systematic assessment of the standards and procedures for testing new drugs is needed."

In a commentary published alongside the study, Daniel Carpenter of Harvard University questioned the thoroughness of the expedited and follow-up processes.

"The findings… underscore the continuing importance of rigorous premarket studies of ample size,” Carpenter wrote. “ If the critical phrase 'serious or life-threatening conditions that would address an unmet medical need' is defined broadly enough (and there are lobbying efforts to define it as broadly as possible), the future of evidence for pharmaceuticals in the United States will look more like 100 patients for efficacy trials instead of 500 patients."

"If the FDA's requirements for new drugs, both premarket and postmarket, are weakened, trust in both the efficacy and safety of prescription drugs is likely to be weakened,” he added. “The stakes of the current policy debates could not be higher. There is scarcely a feature of the American health care system that does not depend on evidence-based trust in prescription drugs, ratified and enforced by the FDA.”

Carpenter’s critique comes just days after the FDA was criticized for approving a powerful, single-ingredient version of the painkiller hydrocodone, a widely-abused prescription drug.

Members of Congress who regularly battle prescription drug abuse in their home states quickly admonished the FDA decision to approve the drug referred to as Zohydro for "daily, around-the-clock, long-term treatment.”

"FDA not only approves this dangerous drug, but does so without requiring any abuse-deterrent features. This is outrageous," said Rep. Bill Keating, D-Mass., in a statement. "Abuse-deterrent technologies should not be the anomaly, they must be the norm."