New Reports on FDA Drug Approval Performance Emerge as House Committee Considers User Fee Reauthorization Legislation

May 10, 2012

By Kurt R. Karst –      

Two new reports were published this week analyzing FDA’s performance under the Prescription Drug User Fee Act (“PDUFA”).  The reports – one from the California Health Institute (“CHI”) and The Boston Consulting Group (“BCG’), and another from the Tufts Center for the Study of Drug Development (“CSDD”) – come on the heels of a report from the Government Accountability Office saying that FDA has met most of the Agency’s PDUFA performance goals for priority and standard original NDA and BLA submissions and for priority and standard original efficacy supplements to approved NDAs and BLAs.  The reports also surfaced just a day before the House Energy and Commerce Committee takes up consideration of legislation (H.R. 5651) to ensure the continuation of existing FDA user fee programs for drugs and devices, to create new user free prograns for biosimilars and generic drugs, and that includes a host of other provisions including drug and device regulatory reforms (see here).  (The U.S. Senate’s version of the bill is S. 2516 – the FDA Safety and Innovation Act.) 

The CHI/BCG report, titled “Managing Priorities: Therapeutic Area Variation in FDA Drug Regulation,” updates a February 2011 CHI/BCG report, titled “Competitiveness and Regulation: The FDA and the Future of America’s Biomedical Industry,” that examined FDA’s drug and biologic approval performance over the past several years.  The new report also presents new data on FDA performance differences based on therapeutic area.

According to the CHI/BCG analysis, “[i]n 2009, the average drug approval time marginally improved from the PDUFA III period (by 1.3 percent) to 14.5 months.”  Although a review of submissions through Fiscal Year 2011 “suggests improvement in approval times,” according to the report, “[l]ooking at new drugs submitted to the Agency in 2008 . . . shows approval timelines getting longer; 45 percent of those submissions remained in process more than a year before the Agency took regulatory action.”

Moving on to therapeutic area variations in FDA performance, the CHI/BCG report says that while oncology, infectious disease, and orphan diseases are “bright spots,” there are significant review time deviations depending on a product’s therapeutic area.  For example, “between 2000 and 2011, oncology and antiinfective drugs . . . experienced the fastest reviews, on the order of 10-15 months,” while “[f]or other categories – cardiovascular, central nervous system, gastro-intestinal, respiratory, etc. – average review times stretched from 20 to 30 months” (see the figure below from the report). 


“No single factor explains differences in performance among therapeutic areas,” says the report, but rather, a confluence of factors.  “Some fields may be scientifically more complicated, with fewer biomarkers or with poorly understood mechanisms of action for novel drugs.”  In addition, regulatory pathways in the areas of diabetes, obesity, and cardiovascular disease, which “exert enormous, and growing damage on health, . . . are fraught with uncertainty.”  This leads the report authors to recognize that “PDUFA V is essential to provide the Agency the core resources it needs to regulate drugs,” but that more work is needed to “[bring] drug approvals into better alignment with public health needs [with] continuous improvements in management, infused with science and common sense.”

The CSDD report, titled “User fee era in U.S. currently poses mixed regulatory burden for sponsors,” shows the results of a top-tier industry working group that was asked to consider two questions in light of the various regulatory enhancements that were made as part of the 2007 FDA Amendments Act (“FDAAA”):  “1) Has there been a detectable increase in the regulatory burden pre-FDAAA vs. post-FDAAA? and 2) What specific requirements/actions in the approval process are particularly problematic?”  According to the Tufts CSDD Impact Report, among other things:

  • The regulatory burden from the pre- to post-FDAAA period largely decreased for New Molecular entity (“NME”) NDA and new BLA approvals in the number of Complete Response letters, share of multi-cycle approvals, average number of FDA-sponsor meetings, number of countries in which product was launched, and increase in annual regulatory budget.
  • The regulatory burden from the pre- to post-FDAAA period increased for supplemental NDA and BLA approvals in the number of Complete Response letters and regulatory outsourcing. 
  • The difference between optimal and sub-optimal sponsor experiences with the FDA review process was less for NME NDAs and new BLAs than for NDA and BLA supplements.  FDA information requests and first cycle actions were shown to have the most consistent impact on a sponsor’s regulatory experience. 

Christopher-Paul Milne, an associate director at Tufts CSDD and who conducted the assessment, commented in a press release that “[w]hile user fees have helped to streamline and speed up the drug review and approval process in the Untied States over the last two decades, certain requirements on developers remain especially problematic . . . PDUFA V provides an opportunity to resolve some of these issues and concerns.”


  • On May 10th, the House Energy and Commerce Committee passed H.R. 5651 by a roll call vote of 46-0.  An amendment submitted by Rep. Joe Barton (R-TX) to reduce the amount of PDUFA, MDUFA, GDUFA, and BSUFA fees by 20% if FDA does not make “adequate progress” toward achieving the agreed upon application review goals was withdrawn.