FDA grants priority review for applications that it  expects, if approved, would provide significant therapeutic benefits,  compared to available drugs, in the treatment, diagnosis, or prevention of  a disease. FDA Met Most Performance Goals for Original NDAs and BLAs for FYs 2000 through 2010

FDA met most of its performance goals for priority and standard original  NDA and BLA submissions during our analysis period by issuing the  proportion of action letters specified in the performance goals within the  goal time frames. For Class 2 NDA and BLA resubmissions, FDA  met the performance goals for 10 of the 11 completed cohorts we  examined. Average FDA Review Time Increased Slightly for Original NDAs and BLAs from FY 2000 through FY 2010

Overall, average FDA review time—the time elapsed from when FDA  received a submission until it issued an action letter—increased slightly  from FY 2000 through FY 2010 for both priority and standard NDAs and  BLAs. Percentage of FDA First-Cycle Approvals Generally Increased from FY 2000 through FY 2010 but Decreased for Priority NDAs and BLAs Since FY 2007

The percentage of priority NDAs and BLAs receiving an approval letter at  the end of the first review cycle exhibited a sharp 1-year decline from   FY 2000 to FY 2001, then increased substantially from FY 2001 through  FY 2007, before decreasing again from FY 2007 through FY 2010 (see  fig. 3). Trends for FYs 2000 through 2010 in the percentage of first-cycle  approvals were similar for the subset of NDAs and BLAs that were for  innovative drugs when compared to trends for all priority or standard  NDAs and BLAs. However, the average FDA review time generally  increased during this period for both priority and standard efficacy  supplements. Specifically, FDA  met the performance goals for both priority and standard efficacy  supplements for 10 of the 11 completed cohorts we examined (see fig. Although the FY 2011 cohort was still incomplete at the time we received  FDA’s data, based on efficacy supplements on which it had taken action,  FDA was meeting the goal for both priority and standard efficacy  supplements. Percentage of FDA First-Cycle Approvals Fluctuated for Priority Efficacy Supplements but Generally Increased for Standard Efficacy Supplements from FYs 2000 through 2010

The percentage of priority efficacy supplements receiving an approval  decision at the end of the first review cycle fluctuated for FYs 2000  through 2010, ranging between 47 percent and 80 percent during this  time (see fig. The results for standard efficacy supplements showed a  steadier increase than for priority submissions. Stakeholders Noted Issues with the Prescription Drug Review Process and FDA Is Taking Steps That May Address Many of Those Issues

The industry groups and consumer advocacy groups we interviewed  noted a number of issues related to FDA’s review of prescription drug  applications. The most commonly mentioned issues raised by industry  and consumer advocacy stakeholder groups were actions or  requirements that stakeholders believe can increase review times and  insufficient communication between FDA and stakeholders throughout the  review process. Industry stakeholders also noted a lack of predictability  and consistency in reviews. Industry Stakeholders Report a Lack of Predictability and Consistency in Reviews

The two industry stakeholders that we interviewed also told us that there  is a lack of predictability and consistency in FDA’s reviews of drug  applications. Consumer Advocacy Group Stakeholders Suggest That FDA May Provide Inadequate Assurance of the Safety and Efficacy of Approved Drugs

Three of the five consumer advocacy group stakeholders that we spoke  with raised issues about whether FDA is adequately ensuring the safety  and efficacy of the drugs it approves for marketing. While FDA has met most of the performance goals we examined,  stakeholders we spoke with point to a number of issues that the agency  could consider to improve the drug review process; FDA is taking or has  agreed to take steps that may address these issues, such as issuing new  guidance, establishing new communication-related performance goals,  training staff, and enhancing scientific decision making. HHS generally agreed with our findings and  noted that they reflect what the agency reported for the same time period. HHS also called attention to activities FDA has undertaken to improve the  prescription drug review process. It highlighted FDA’s performance in  approving innovative drugs in FY 2011. Finally, HHS discussed enhancements to the drug review  program that were included in the proposed recommendations for the  2012 reauthorization of the prescription drug user fee program, such as  establishing a new review program for innovative drugs, enhancing  benefit-risk assessment, and requiring electronic submissions and  standardization of electronic application data to improve efficiency. At that time, we will send copies of this report to the  Secretary of Health and Human Services, the Commissioner of the Food  and Drug Administration, and other interested parties. FYs 2008 through 2011 calculations exclude submissions for which FDA had not yet issued an  action letter.
Why GAO Did This Study

The Food and Drug Administration (FDA) within the Department of Health and Human Services (HHS) is responsible for overseeing the safety and efficacy of drugs and biologics sold in the United States. New drugs and biologics must be reviewed by FDA before they can be marketed, and the Prescription Drug User Fee Act (PDUFA) authorizes FDA to collect user fees from the pharmaceutical industry to support its review of prescription drug applications, including new drug applications (NDA), biologic license applications (BLA), and efficacy supplements that propose changes to the way approved drugs and biologics are marketed or used. Under each authorization of PDUFA since 1992, FDA committed to performance goals for its drug and biologic reviews.
In preparation for the next PDUFA reauthorization, GAO was asked to examine FDAs drug and biologic review processes. In this report, we (1) examine trends in FDAs NDA and BLA review performance for fiscal years (FY) 2000 through 2010, (2) examine trends in FDAs efficacy supplement review performance for FYs 2000 through 2010, and (3) describe issues stakeholders have raised about the drug and biologic review processes and steps FDA is taking that may address these issues. To do this work, GAO examined FDA drug and biologic review data, reviewed FDA user fee data, interviewed FDA officials, and interviewed two industry groups and five consumer advocacy groups. All of the stakeholder groups participated in at least half of the meetings held by FDA to discuss the reauthorization of the prescription drug user fee program.

What GAO Found

FDA met most performance goals for priority and standard NDAs and BLAs received from FY 2000 through FY 2010. FDA meets its performance goals by completing its review and issuing an action lettersuch as an approval or a response detailing deficiencies that are preventing the application from being approvedfor a specified percentage of applications within a designated period of time. FDA designates NDAs and BLAs as either priorityif the product would provide significant therapeutic benefits when compared to available drugsor standard. FDA met the performance goals for both priority and standard NDAs and BLAs for 10 of the 11 fiscal years GAO examined; FDA did not meet either of the goals for FY 2008. Although FDA had not yet issued an action letter for all of the applications it received in FY 2011 and results are therefore preliminary, FDA was meeting the goals for both priority and standard NDAs and BLAs on which it had taken action. Meanwhile, FDA review time for NDAs and BLAsthe time elapsed between FDAs receipt of an application and issuance of an action letterincreased slightly from FY 2000 through FY 2010. In addition, the percentage of NDAs and BLAs receiving an approval letter at the end of the first review cycle generally increased, although that percentage has decreased for priority NDAs and BLAs since FY 2007.
FDA met most of its performance goals for efficacy supplements from FY 2000 through FY 2010. Specifically, FDA met the performance goals for both priority and standard efficacy supplements for 10 of the 11 fiscal years GAO examined. FDA review time generally increased during the analysis period for both priority and standard efficacy supplements. The percentage of priority efficacy supplements receiving an approval letter at the end of the first review cycle fluctuated from FY 2000 through FY 2010, ranging between 47 percent and 80 percent during this time. The results for standard efficacy supplements showed a steadier increase with the percentage of first-cycle approval letters rising from 43 percent for FY 2000 applications to 69 percent for FY 2010 applications.
The industry groups and consumer advocacy groups we interviewed noted a number of perceived issues related to FDAs review of drug and biologic applications. The most commonly mentioned issues raised by industry and consumer advocacy stakeholder groups were actions or requirements that can increase review times (such as taking more than one cycle to approve applications) and insufficient communication between FDA and stakeholders throughout the review process. Industry stakeholders also noted a perceived lack of predictability and consistency in reviews. Consumer advocacy group stakeholders noted issues related to inadequate assurance of the safety and effectiveness of approved drugs. FDA is taking steps that may address many of these issues, including issuing new guidance, establishing new communication-related performance goals, training staff, and enhancing scientific decision making.
In commenting on a draft of this report, HHS generally agreed with GAOs findings and noted that they reflect what the agency reported for the same time period. HHS also called attention to activities FDA has undertaken to improve the prescription drug review process.