[In print in the March 2004 issue of Drug Discovery and Development Magazine]
The US Food and Drug Administration reported in January what it called a rebound in approval of innovative drugs in 2003. They attributed the increase in approved new molecular entities (NMEs) to an innovation initiative introduced last year, soon after the arrival of FDA commissioner Mark McClellan in November 2002. NMEs, molecules not previously marketed in the United States, are among the most innovative new drugs. "FDA is making new treatments available more quickly," the agency quoted Department of Health and Human Services Secretary Tommy Thompson as saying.
The word "rebound" seems appropriate, because the number of NMEs approved the year before, at 17, had been the smallest in at least a decade. The 21 NMEs approved in 2003 was still the second smallest. Moreover, calling the increase from 17 to 21 "significant" may sound odd to researchers accustomed to strict statistical definitions of significance. The 72 new drug applications (NDAs) approved in 2003 represented a slight decrease in 2002, from 78.
The median approval time for priority NMEs decreased markedly, from 16.3 months in 2002 from 6.7 months in 2002, but the data for 2002 had been skewed by some unusually long approvals. For 2000 and 2001, priority NMEs were approved in a median of 6.0 months. For standard NMEs, the median time of 15.4 months in 2003 was almost unchanged from 2002.
Nonetheless, "it's hard to judge the FDA's effectiveness by looking at the numbers of approved compounds," says Glenn Gormley, MD, PhD, director of clinical development at AstraZeneca. (AstraZeneca and Roche each had two NMEs approved during 2003, more than any other company.) Both the number of approvals and the approval time depend on how well developed the applications submitted to the FDA are. Gormley says "what is important is the level of cooperation."
A major element of the initiative, which the FDA introduced in January 2003, is greater feedback to applicants about the agency's expectations, so that additional review cycles can be avoided. Gormley says he's impressed with the agency's willingness to have an open dialogue during reviews, which he says is particularly helpful at the end of phase II trials. "Under McClellan, the agency is doing an outstanding job."
Gormley emphasizes that more open communication may require applicants to adapt as well. There is a "responsibility on the sponsor's part to seek that communication and be willing to be open ourselves."