A recent study by researchers at Yale University and the Yale School of Medicine found that 46 medicines approved by the U.S. Food and Drug Administration as part of its Breakthrough Therapy program were sent to patients without large double-blind studies, direct measurement of benefits or placebo tests.
The study was published in the Journal of the American Medical Association, and did not specify whether the drugs were, indeed, as effective and safe as found in pre-approval testing, Reuters reports.
“Our research suggests that FDA approval of these breakthrough therapies is generally based on shorter and smaller clinical trials that those that support FDA approval of non-breakthrough therapy drugs,” Joseph Ross, coauthor of the study, told Reuters Health.
The FDA’s Breakthrough Therapy program was developed with a focus expedited approval for drugs targeting serious or life-threatening.
Expedited approvals, however, are not without some skepticism.
” … Expediting drug approvals raises concerns that important safety or effectiveness information will be missed, potentially heightening risk of patient harm,” Austin Frakt with the Veterans Health Administration reportedly said in a JAMA commentary.