The US Food and Drug Administration has approved 35 new innovative drugs during the past 12 months ended September 2011. This is among the highest number of approvals in the past decade, surpassed only during 2009 of 37 approvals. 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.
Margaret Hamburg, M.D., Commissioner of Food and Drugs, said, “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. 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.”
The report for the fiscal year ending September 30, 2011 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.
Among the new drugs approved in FY 2011, a number of them 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 and 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 programme 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 September 30, 2012.
“Before the PDUFA programme, 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.