Patients to Benefit from Novel Medicines

By: Margaret A. Hamburg, M.D.

At FDA, we face a careful balancing act between the need to bring innovative new drugs to patients quickly while taking care that these medicines are safe and effective.

Margaret Hamburg, M.D.A new FDA report shows that in the case of 35 novel drugs, called new molecular entities, we were able to strike that balance. These innovative drugs, many of which target devastating diseases, were approved by the agency in FY 2012, which ended September 30. That’s the same number of novel drug approvals as in FY 2011.  

Most of these 35 drugs were approved in theUnited Statesbefore they were approved in other countries, and all but one was approved within its target review date. This is thanks in part to the use of several authorities designed to enable quicker-than-usual action for drugs that treat serious or life-threatening diseases and that may fill an unmet medical need. FDA also permitted shorter, smaller, or fewer clinical studies when justified, which can reduce the length and cost of drug testing and bring drugs to market sooner.

If you go beyond the statistics, you will find the reality: elderly men and women whose sight may be saved, children who will breathe more easily, and cancer patients whose lives may be extended.

One of these 35 medicines is the first drug available to treat advanced basal cell carcinoma, a form of the most common skin cancer. Another is the first for the bone marrow disease myelofibrosis, which impairs the body’s ability to produce normal blood cells.

The list of novel drugs demonstrates numerous scientific advances and innovative approaches. For example, one is the first approved drug made from the blood of the human umbilical cord.  Another groundbreaking treatment targets a gene defect in certain cystic fibrosis patients rather than just treating the symptoms of the life-threatening disease. Another helps the body process a commonly used chemotherapy drug that can cause kidney failure and death in high and sustained concentrations.

It is also important to note that these faster approval times do not mean that the agency relaxed its review standards regarding patient safety. FDA continued its focus on assuring drug safety prior to approval as well as monitoring their safety after approval.  This included the implementation of the new drug adverse event system, FDA Adverse Event Reporting System (FAERS), which has modernized the agency’s ability to process more than one million adverse event reports received each year. It also included the agency’s Sentinel Initiative—FDA has been using a pilot of that program called Mini-Sentinel—to access electronic health information from 159 million patients and perform rapid assessments when there is an early signal that there may be a safety problem with a drug on the market.

For more about the novel medicines approved this fiscal year see the report at Most importantly, these new drugs will offer hope to some of the millions of patients awaiting critical treatments.

Margaret A. Hamburg, M.D., is Commissioner of the U.S. Food and Drug Administration

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