GAO Report Says That FDA Has Met Most PDUFA Performance Goals; Agency Plans to Take Steps to Address Lingering Stakeholder Concerns

By Kurt R. Karst –      

A report released earlier this week by the Government Accountability Office (“GAO”) says 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, although in each case FDA review times have increased slightly.  The GAO’s analysis covers applications in the Fiscal Year 2000 to 2010 cohorts, as well as preliminary information for applications submitted in Fiscal Year 2011. 

The GAO report was sent to Senators Richard Burr (R-NC) and  Tom Coburn (R-OK).  Both Senators have long criticized FDA, saying that the Agency‘s “regulatory malaise” harms patients and manufacturers.  Sen. Burr has also threatened to delay the passage of PDUFA and other user fee legislation unless FDA speeds up application approval times.  In addition, as we previously reported, Sen. Burr was successful in getting an amendment added to the FDA appropriations bill passed last year that seeks to “improve the transparency and accountability of the FDA in order to encourage regulatory certainty and innovation on behalf of America’s patients.”  That amendment requires the submission of information to Congress on, among other things,

• “the average number of calendar days that elapsed from the date that drug applications (including any supplements) were submitted to such Secretary under [FDC Act § 505] until the date that the drugs were approved under such section 505;” and
• “the average number of calendar days that elapsed from the date that [BLAs] (including any supplements) were submitted to such Secretary under [PHS Act § 351] until the date that the biological products were licensed under such section 351.”  [(Emphasis added)]

The GAO’s report responds to the requests in Sen. Burr’s appropriations bill amendment – at least insofar as NDAs and BLAs are concerned (ANDAs are not covered in the GAO report).  The report also provides a wealth of additional information on FDA approval statistics and addresses some stakeholder concerns that were raised as GAO conducted its analysis. 

The GAO’s analysis (reflected in the tables below) shows that except for Fiscal Year 2008, FDA met PDUFA goals in all of the Fiscal Year 2000 to 2010 cohorts.  (FDA recently provided similar statistics in testimony before Congress.)  Moreover, the GAO found that an average of 44% of all original NDAs and BLAs submitted to FDA in Fiscal Years 2000 to 2010 were approved during the first review cycle and 75% were ultimately approved.  FDA and industry stakeholders the GAO interviewed suggested that FDA failed to meet Fiscal Year 2008 goals as a result of implementation of the Risk Evaluation and Mitigation Strategy (“REMS”) requirements added to the FDC Act by the 2007 FDA Amendments Act.

Although FDA met most PDUFA goals for the Fiscal Year 2000 to 2010 application cohorts (and is on track to meet Fiscal Year 2011 goals for the applications submitted in that cohort), the GAO’s analysis (reflected in the tables below) shows that average FDA review times (i.e., the time elapsed from when FDA received a submission until it issued an action letter) hve increased slightly from Fiscal Year 2000 through Fiscal Yeat 2010 for both priority and standard NDAs and BLAs and priority and standard original efficacy supplements to approved NDAs and BLAs.

With respect to Sen. Burr’s request for the average number of calendar days that elapsed from the date of NDA or BLA (including supplement) submission to final FDA action, the GAO says that it was unable to calculate average FDA review times in any meaningful way because most cohorts were still open; that is, “fewer than 90 percent of submissions had received a final action such as approval, denial, or withdrawal.”  Specifically, for priority original NDAs and BLAs, only four cohorts had at least 90% of submissions closed (Fiscal Years 2001, 2002, 2005, and 2006), and for standard original NDAs and BLAs, only one cohort had at least 90% of submissions closed (Fiscal Year 2002).  For priority efficacy supplements, only four cohorts had at least 90% of submissions closed (Fiscal Years 2000, 2001, 2004, and 2007), and for standard efficacy supplements, only one cohort had at least 90% of submissions closed (Fiscal Year 2005).

Stakeholders the GAO interviewed identified some issues that they believe hamper the the NDA and BLA approval process, including REMS implementation, the use of outside expertise for reviewing applications, insufficient communication between FDA and stakeholders, and a lack of predictability and consistency in FDA reviews.   FDA commented in the Agency’s response to the GAO report that it is taking or has agreed to take steps (as part of PDUFA V) that may address these issues, including issuing new guidance, establishing new communication-related performance goals, training staff, and enhancing scientific decision making.