Every five years the pace of FDA evaluations and approvals increases in advance of PDUFA renewal in congress. This year has proven no exception to that trend.
Over the past few months, there have been numerous reports and media stories surrounding the ability of the Food and Drug Administration (FDA) to approve new drugs and devices. Industry and patient groups have raised concerns that FDA may be too slow in approving certain products and that the process for approval may be too unpredictable, thus stifling investors and funding. Some have even pointed to other countries in Europe or Asia, where products were first approved or approved faster.
Perhaps dispelling some of these concerns, FDA recently announced it has approved 35 new molecular entities (NMEs) in the past 12 months, including two new treatments for hepatitis C, the first new drug for Hodgkin lymphoma in 30 years, drug resistant skin infections, pneumonia, and the first new drug for lupus in 50 years. In 2009, FDA approved 37 new drugs.
Additionally, as an article from MedPage Today noted, “the U.S. was first to approve 24 of the 35 new approvals in fiscal 2011, which ended Sept. 30, “continuing a trend of the U.S. leading the world in first approval of new medicines,” the FDA said in a press release announcing the new report.
The approval rate is “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” FDA Commissioner Margaret Hamburg remarked.
The agency also pointed out that for all but one of the 35 new drugs, the FDA issued an approval decision within the time frame it had agreed upon with the drug company, under current PDUFA legislation.
In a letter to FDA staff, it was noted that, “FDA expedited the approval of many of these products by streamlining clinical trial requirements to permit smaller, shorter, or fewer studies wherever possible. FDA approved nearly half — 16 — of the innovative drugs under the agency’s “priority review” program for drugs that may offer major advances in treatment; priority reviews carry a six-month target date for review.” Moreover, “FDA approved the majority of these innovative drugs on the “first cycle,” that is, without requests for additional information that would trigger a second review cycle.”
MedPage Today pointed out that, “Seven of the new medications approved provide “major advances” in cancer treatment, according to the FDA. They include: abiraterone acetate (Zytiga) for prostate cancer; ipilimumab (Yervoy) for metastatic melanoma; brentuximab vedotin (Adcetris) for Hodgkin lymphoma and systemic anaplastic large-cell lymphoma; vandetanib (Caprelsa) for metastatic medullary thyroid cancer; and eribulin mesylate (Halaven) for metastatic breast cancer.”
Two of the cancer drugs are targeted therapies: crizotinib (Xalkori) for late-stage non-small cell lung cancer and vemurafenib (Zelboraf) for metastatic melanoma. Both drugs come with a companion diagnostic test to help determine if a patient has the abnormal genes for the specific disease and will therefore benefit most from treatment with the cancer medications. In addition, 10 of the 35 drugs are for rare, so-called “orphan” diseases, which have few or no treatment options.
Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research (CDER), said during a call with reporters that the high number of approvals shows the agency is flexible in working with drug companies seeking approval.
“For instance, when the company CSL Behring was conducting a full-scale trial for its factor XIII concentrate (Corifact), a drug to prevent bleeding in people with a rare genetic defect, the agency told the company that a much smaller trial would suffice, considering how rare the disorder is and how positive early results were. The agency granted accelerated approval to the company in February based on a clinical trial of just 14 patients.”
“Woodcock also pointed to the user fees collected from drug companies as a key reason for the large number of approvals. Woodcock said user fees collected have enabled CDER to hire more staff, which has improved the drug approval process.”
“Richard Pazdur, leader of the Office of Oncology drug products within CDER, said approvals may accelerate as part of a September reorganization of his office that focuses drug evaluation teams on afflicted body parts instead of the treatment’s composition.”
In a prepared statement, the Pharmaceutical Research and Manufacturers of America (PhRMA) said the FDA’s report underscores the “shared commitment of biopharmaceutical research companies and the agency to medical progress and patient care,” and underscores the importance of approving a “clean” reauthorization of the Prescription Drug User Fee Act (PDUFA).
Congress must reauthorize the program every five years, and the current PDUFA is set to expire in September 2012. A draft agreement between drugmakers and the FDA would increase industry user fees by 6% through 2017, in exchange for improved communication with the agency and greater consistency in the drug approval process.
This would amount to roughly $712.8 million in fees in fiscal 2013. PDUFA fees covered 62% of the nearly $932 million the FDA spent on reviewing new drugs in 2010.
While this report shows FDA is making progress, it is important that during PDUFA negotiations, all stakeholders continue to press FDA on important issues to ensure the predictability and transparency necessary for the approval process to continue having successful rates as this report shows, such as changes to the FDA advisory committee process.
It is also important that in the coming years the FDA continue a brisk pace of drug evaluations.