Over the past 12 months, the U.S. Food and Drug Administration approved 35 new medicines. This is among the highest number of approvals in the past decade, surpassed only by 2009 (37). Many of the drugs are important advances for patients, including: two new treatments for hepatitis C; a drug for late-stage prostate cancer; the first new drug for Hodgkin’s lymphoma in 30 years; and the first new drug for lupus in 50 years.
In a report released today, FY 2011 Innovative Drug Approvals, the FDA provided details of how it used expedited approval authorities, flexibility in clinical trial requirements and resources collected under the Prescription Drug User Fee Act (PDUFA) to boost the number of innovative drug approvals to 35 for the fiscal year (FY) ending Sept. 30, 2011. The approvals come while drug safety standards have been maintained.
The report shows faster approval times in the United States when compared to the FDA’s counterparts around the globe. Twenty-four of the 35 approvals occurred in the United States before any other country in the world and also before the European Union, continuing a trend of the United States leading the world in first approval of new medicines.
“Thirty-five major drug approvals in one year represents a very strong performance, both by industry and by the FDA, and we continue to use every resource possible to get new treatments to patients,” said Margaret Hamburg, M.D., Commissioner of Food and Drugs. “We are committed to working with industry to promote the science and innovation it takes to produce breakthrough treatments and to ensure that our nation is fully equipped to address the public health challenges of the 21st century.”
Among the new drugs approved in FY 2011, a number are notable for their advances in patient care and for the efficiency with which they were approved:
• Two of the drugs – one for melanoma and one for lung cancer – are breakthroughs in personalized medicine. Each was approved with a diagnostic test that helps identify patients for whom the drug is most likely to bring benefits;
• Seven of the new medicines provide major advances in cancer treatment;
• Almost half of the drugs were judged to be significant therapeutic advances over existing therapies for heart attack, stroke and kidney transplant rejection;
• Ten are for rare or “orphan” diseases, which frequently lack any therapy because of the small number of patients with the condition, such as a treatment for hereditary angioedema;
• Almost half (16) were approved under “priority review,” in which the FDA has a six month goal to complete its review for safety and effectiveness;
• Two-thirds of the new approvals were completed in a single review cycle, meaning sufficient evidence was provided by the manufacturer so that the FDA could move the application through the review process without requesting major new information;
• Three were approved using “accelerated approval,” a program under which the FDA approves safe and effective medically important new drugs quickly, and relies on subsequent post-market studies to confirm clinical benefit. For example, Corifact, the first treatment approved for a rare blood clotting disorder, was approved under this program; and
• Thirty-four of 35 were approved on or before the review time targets agreed to with industry under PDUFA, including three cancer drugs that FDA approved in less than six months.
The Prescription Drug User Fee Act was established by Congress in 1992 to ensure that the FDA had the necessary resources for the safe and timely review of new drugs and for increased drug safety efforts. The current legislative authority for PDUFA expires on Sept. 30, 2012.
“Before the PDUFA program, American patients waited for new drugs long after they were available elsewhere,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “As a result of the user fee program, new drugs are rapidly available to patients in the United States while maintaining our high standards for safety and efficacy.”
In October 2011, the FDA released a new plan, Driving Biomedical Innovation: Initiatives to Improve Products for Patients, to assist companies engaged in new product development, particularly smaller, entrepreneurial companies.
In a separate action, the agency also released a report this week on drug shortages, expanded its current actions to address the problem, and, at the direction of the President, will broaden early notification of drug shortages and work with the Department of Justice to prevent price gouging.
Report on FY 2011 Innovative Drug Approvals
Driving Biomedical Innovation: Initiatives to Improve Products for Patients