Researchers Examine FDA Decision Making Processes

(HealthDay News) – The rationale behind the decision making processes of the U.S. Food and Drug Administration is discussed in three articles published in the Jan. 22/29 issue of the Journal of the American Medical Association.

Nicholas S. Downing, from the Yale University School of Medicine in New Haven, CT, and colleagues characterized pivotal efficacy trials that serve as the basis for FDA approval for novel therapeutic agents. The researchers found that, between 2005 and 2012, the FDA approved 188 novel therapeutic agents for 206 indications, based on 448 pivotal efficacy trials. The majority of trials were randomized (89.3%), double-blinded (79.5%), and used an active or placebo comparator (87.1%). Among 201 indications, 74 (37%) were approved on the basis of a single trial, 77 (38%) on 2, and 50 (25%) on 3 or more. Trials with surrogate end points as the primary outcome formed the exclusive basis of approval for 91 indications.

Benjamin N. Rome, from Harvard Medical School in Boston, and colleagues used the FDA premarket approval (PMA) database to characterize changes to cardiac implantable electronic devices from 1979–2012. During that period, the researchers identified 77 original and 5,829 supplement FDA-approved PMA applications (median of 50 supplements per original PMA). Seventy-nine percent of the original PMAs approved during the study period were the subject of one or more supplements in 2012. In a third study, Leonard V. Sacks, MBBCh, from the FDA in Silver Spring, MD, noted that significant delays in the approval of new molecular entities could be explained by potentially preventable deficiencies, including failure to select optimal drug doses and suitable study end points.

“Understanding the reasons for previous failures is helpful to improve the efficiency of clinical development for new drugs,” Sacks and colleagues write.

Several authors from the Downing study disclosed financial ties to the health care and medical device industries. Several authors from the Rome study disclosed ties to the FDA.

Abstract – Downing
Full Text (subscription or payment may be required)
Abstract – Rome
Full Text (subscription or payment may be required)
Abstract – Sacks
Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)