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U.S. Department of Health and Human Services

About FDA

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Previous Section: Executive Summary

In Fiscal Year (FY) 2011,1 FDA approved 35 novel medicines, many of which are groundbreaking, including two new treatments for hepatitis C, a drug that prolongs survival in patients with late-stage prostate cancer, the first new drug to treat Hodgkin lymphoma in 30 years and the first new drug to treat lupus in 50 years. The 35 novel drugs are notable for their contributions to the health of Americans and their scientific innovation. There were breakthroughs in personalized medicine: two of the drugs, for lung cancer and melanoma, were developed and approved with diagnostic devices that will allow doctors to target the drug to those patients most likely to respond. Seven of the drugs represent major advances in cancer treatment. Still others provide important new therapies for treating drug resistant skin infections and pneumonia, and for preventing heart attack, stroke, and kidney transplant rejection. Ten of the approved treatments are for rare or "orphan" diseases, which have few or no drug treatment options because of their small patient populations (less than 200,000).

Many of these drugs were also notable for the speed with which they were approved: almost 70% of them were approved first in the US, before any other country in the world. For many of these approvals, FDA used expedited approval authorities and flexible clinical trial requirements to permit shorter, smaller, or fewer studies, which can reduce the high cost of drug testing. FY 2011 continued the agency's strong track record of approving drugs on or before the target dates for application review that were agreed to under the Prescription Drug User Fee Act (PDUFA). All but one (34/35) of the innovative drugs were approved on or before their target dates for review. FDA also continued to approve over half of these drugs on the "first cycle" of review, i.e., without requests for additional information that would lead to another cycle of review.

Over the last several years, FDA has improved the quality and speed of its drug approval process through:

  • The PDUFA program, which helps to provide the resources necessary to hire and train scientific reviewers, keep FDA scientists abreast of innovative technologies, allow FDA to meet with companies early in drug testing to provide advice on development programs, and develop guidance documents that clarify the drug development pathway for many diseases;
  • Expedited approval pathways including Fast Track, Accelerated Approval, Priority Review, and Expanded Access programs, which are designed to speed the testing, availability and approval of drugs in different ways;
  • Critical Path, a program started in 2004, focused on seeking the development of new test methods that can streamline drug development, testing and review; and
  • A strong focus on methods of efficiently identifying and resolving drug safety issues.

In this year of strong leadership by FDA in approving innovative drugs, it is important to recognize the achievements of the biopharmaceutical industry. None of FDA's accomplishments would be possible without the innovation and hard work of large and small biopharmaceutical companies alike. Not only did the drug applications that the industry submitted to FDA represent important medical advances, but their generally high quality permitted FDA to reach an approval decision, in many cases, after a single cycle of review.



It is FDA's job to make sure that new drugs entering the U.S. market are safe and effective. At the same time, FDA must seek to ensure that Americans have access to innovative and potentially life-saving medicines as soon as possible. Although maintaining a balance between these two responsibilities can be challenging, the public health demands that FDA actively pursue both goals. FDA has recently faced criticism that drug review times have slowed due to an increased emphasis on establishing drug safety before approval. FDA's record, including FY 2011, shows instead that the agency, while ensuring the safety and effectiveness of new therapies, continues to approve innovative drugs earlier and faster than anywhere else in the world, including the European Union (EU).

This report looks at FDA's approvals of "new molecular entities" (NMEs) in FY 2011. For purposes of this report, NMEs are drugs, including biological products, with novel chemical structures that have never been approved before to treat any disease, and often represent the most innovative drugs entering the market. These drugs include products approved by FDA's Center for Drug Evaluation and Research and FDA's Center for Biologics Evaluation and Research.

More "first approvals" than any other country.

Of the 35 innovative drugs approved in FY 2011, 24 (almost 70%) were approved by FDA before any other regulatory agency in the world, including the EMA (the EU's drug regulatory agency).

Since the enactment of the Prescription Drug User Fee Act (PDUFA) in 1992, FDA has steadily increased the speed of Americans' access to important new drugs compared to the EU and the world as a whole. As shown in Figure 1, the United States now leads the world in the first introduction of new active drug substances.2

Figure 1. US Share of New Active Substances (NAS) First Launched on the World Market

Figure 1 shows the percentage of the United States share of new active substances (NAS) first launched on the world market from 1982 to 2010.


If the performance of the US is compared with that of the EU, of 57 novel drugs approved by both FDA and the EU between 2006 and 2010, 43 (75%) were approved first in the US.

Length of reviews shorter than the EU.

In recent years, FDA's drug review times have also been, on average, significantly faster than those in the EU. It is difficult to compare length of approvals for FY 2011 because many of the drugs approved in the US have not yet been approved in the EU. A comparison of drugs approved in the US and EU between 2006 and 2010 is illustrative, however. For "priority" drugs3 approved between 2006 and 2010, FDA's median time to approval was 6 months (183 days), more than twice as fast as the EU, which took a median time of 13.2 months (403 days). For standard drug reviews, FDA's median time to approval was 13 months (396 days), 53 days faster than the EU time of 14.7 months (449 days).

A recent article in the journal Health Affairs also compared cancer drugs approved in the US and EU from 2003 through 2010. Thirty-five cancer drugs were approved by the US or the EU from October 2003 through December 2010. Of those, FDA approved 32—in an average time of 8.6 months (261 days). The EU approved only 26 of these products, and its average time was 12.2 months (373 days). All 23 cancer drugs approved by both agencies during this period were approved first in the United States.4

PDUFA has substantially reduced review times.

According to researchers at the Tufts Center for the Study of Drug Development, the time required for FDA's review of a new drug (i.e. time from submission until approval) has been cut by 60 percent since the enactment of PDUFA, from an average of 2.0 years at the start of PDUFA to an average of 1.1 years more recently.

Of the 35 the innovative drugs approved in FY 2011, 34 met their PDUFA target dates for review.

Expedited approval and flexible trial requirements used to speed access.

FDA has several programs to expedite the development and availability of important new drugs. The most important of these are Fast Track, Accelerated Approval, and Priority Review.5

Fast Track is designed to facilitate the development and expedite the review of drugs to treat serious diseases that fill an unmet medical need. Its purpose is to get important new drugs to the patient earlier. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients. Fast Track drugs are also eligible for rolling review, in which a company can submit portions of its application as they are completed, and FDA may begin review without waiting for the full application. More than a third (13/35) of the innovative drugs approved in 2011 were given a Fast Track designation. Of the 13 drugs that received a Fast Track designation, 12 (92%) were approved in the US before any other country, and 12 (92%) were approved on the first review cycle.

Nearly half (16 of 35) of the FY 2011 drugs received "Priority Review," which means that FDA gave itself a 6-month target date to review the drug. Priority reviews are given to drugs that may offer major advances in treatment or that provide a treatment when no adequate therapy exists. Under PDUFA, FDA has agreed to a 6-month review goal for priority drugs compared to a 10-month review goal for non-priority or "standard" drugs. Of the 16 drugs given a priority review, 11 (69%) were approved in the US before any other country, and 13 (81%) were approved on the first cycle.

Three of the novel drugs approved in FY 2011 were approved under FDA's "Accelerated Approval" authority. Accelerated approval allows the agency to approve a drug to treat a serious disease based on clinical data showing, for example, that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients (such as evidence that a drug shrinks cancer tumors), but does not demonstrate the clinical benefit itself (that patients actually live longer). Clinical trials to establish an effect on surrogate endpoints are frequently shorter and less costly than those establishing a longer-term clinical benefit. The program is designed to provide patients with earlier access to promising new drugs, followed by further studies to confirm the drug's clinical benefit conducted after approval.

Several of the drugs were also approved with flexible clinical trial requirements, for example, on the basis of a single study (in contrast to the usual requirement that a finding of effectiveness be replicated in a second study) and studies using small patient populations.

FDA has also taken steps to speed the development and approval of safe and effective drugs for Americans with rare diseases. Therapies for rare diseases—those affecting fewer than 200,000 people in the United States—represent the most rapidly expanding area of drug development. Although each disease affects a relatively small population, collectively rare diseases affect about 25 million Americans. Approximately 30% of the NMEs approved in the last five years have been drugs for rare diseases. Because of the small numbers of patients who suffer from each disease, FDA often allows non-traditional approaches to establishing safety and effectiveness. The National Organization for Rare Disorders has just released a report concluding that FDA has consistently allowed flexible clinical trial designs in approving drugs for rare diseases.6

Table of Contents: Innovative Drug Approvals

Next Section: Notable FY 2011 Approvals



1October 1, 2010-September 30, 2011.

2Source: Scrip NCE Review/Scrip Yearbook/Scrip Magazine (1982 -2005), PharmaProjects R&D Annual Review (2006-2010). New active substances include novel chemical or biological substances not previously approved to treat any disease. There is a close, but not complete overlap between NASs and NMEs: NASs exclude radiopharmaceuticals.

3Priority drugs are those designated by FDA for priority reviews because of their potential therapeutic value.

4"Despite Criticism Of The FDA Review Process, New Cancer Drugs Reach Patients Sooner In The United States Than In Europe," Samantha A. Roberts, Jeff D. Allen, and Ellen V. Sigal, Health Affairs, June 2011.

5For more information on these expedited approval processes, go to: http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm.

6Frank J. Sasinowski, National Organization for Rare Disorders, Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs: Cataloguing FDA's Flexibility in Regulating Therapies for Persons with Rare Disorders. Released October 11, 2011.