FDA Articles

They scuttle product launches and send company stocks through the floor. They steal precious years from patents and require pricey new trials. They have the entire drug industry on edge, from struggling one-hit biotechs to struggling large-cap pharmas. They're FDA approvable letters—that odd regulatory response that isn't quite an approval and isn't quite a rejection—and in the past three or four years, they've become a major part of the FDA's arsenal for dealing with (or, as many say, not dealing with) new drug applications (NDAs).

You have to admit: Mark McClellan, MD, has quite a resumé. But after you've been the commissioner of FDA and the administrator of the Centers for Medicare and Medicaid Services (CMS), what comes next?

For McClellan, who was awarded a fellowship at the AEI-Brookings Joint Center for Regulatory Studies after leaving CMS, it's about finishing what he started. Under his leadership, the think tank launched the new Engelberg Center for Health Care Reform, which seeks to find practical solutions to the very big questions surrounding the safety, affordability, and quality of medical care.

Industry insiders see McClellan as a "less talk, more action" kind of guy and hope his new post will add horsepower to moving discussions about public policy reforms into action. (After all, Medicare Part D was enacted under his watch.) Here, Pharm Exec talks to McClellan about what it will take to improve the drug safety system and the challenges that face industry and regulators alike.

The FDA is weighing whether to allow a system for electronic insert information for most prescription drugs, replacing paper Rx inserts.
Is it time to make prescribing information electronic and forgo paper package inserts for most prescription drugs? Manufacturers have long led an effort to move toward "paperless labeling," which they say could save millions of dollars. Now the Food & Drug Administration is exploring the idea with a call for comments and a recent hearing at its Rockville, Md., offices.

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Temsirolimus (Torisel, Wyeth), the first mTOR (mammalian target of rapamycin) inhibitor, was recently approved for the treatment of advanced renal cell carcinoma (RCC). "Torisel provides a new option for physicians for the treatment of renal cell carcinoma," said Cindy O'Bryant, Pharm.D., BCOP, assistant professor at the University of Colorado Health Sciences Center School of Pharmacy, Denver.

"Torisel shows promise in treating RCC—a hard tumor to treat," added James Trovato, Pharm.D., MBA, BCOP, associate professor of pharmacy practice at the University of Maryland School of Pharmacy. "It will probably be used as a first-line agent for patients with poor prognostic factors such as advanced or metastatic disease."

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The Food & Drug Administration's Medication Guide program, set up nine years ago as a way to inform patients about the risks or special issues with certain drugs, has a host of problems, according to pharmacists and other witnesses at a recent hearing. For specific drugs with serious public health concerns, the agency requires pharmacists to distribute—with each prescription and refill—written patient information that is approved by FDA and provided by the manufacturer.

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The US Food and Drug Administration plans to shut down its regional offices and some additional field facilities, a move that reflects an ever-tightening squeeze on agency funding and staff. FDA also seeks to make more efficient use of its limited resources by establishing risk-based approaches for selecting those drug-manufacturing sites most in need of frequent oversight, while reducing inspections for less risky facilities. More highly trained inspectors will be able to assess modern manufacturing systems with greater efficiency, and field staff will collaborate better with reviewers in FDA Centers to understand company quality-control systems better.

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The 2006 Guidance reveals the advantages of using electronic modalities, such as IVR, when collecting patient-reported data.

The FDA's draft guidance, "Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims," marks a formal positioning of the agency's stance regarding patient reported outcomes (PROs) in clinical trials. In publishing its guidance, the FDA has implicitly acknowledged the role of PROs and does so explicitly with statements such as: "Patients provide a unique perspective on treatment effectiveness," "Some treatment effects are known only to the patient," "Self-completed questionnaires...are often preferable to the clinician administered interview and rating." In addition, much like the EMEA,1 the FDA has acknowledged the importance and advantages of the collection of electronic patient reported outcomes (ePRO).

Regulating and monitoring clinical trials has become an increasingly complex and challenging business for the Food and Drug Administration and other government agencies that fund and oversee biomedical research. There are more clinical studies in the United States and abroad, many involving multiple sites, complex study designs, and more research participants. Many studies deal with complex biotech therapies, medical devices, and combination products and call for greater participation of vulnerable subjects, including children.

Efforts to better manage the flood of data from these research programs is generating a number of electronic record keeping and processing methods and systems that, in turn, require new rules. These developments impose new challenges for IRBs and research institutions, which continue to evolve to meet new requirements.

Who should make decisions about drug safety—FDA or patients and doctors? In this excerpt from his important new book Overdose, the renowned (and ever controversial) legal scholar Richard A. Epstein argues that the current system overvalues risk, ignores individual differences, and needlessly deprives patients of valuable treatments.

It is well known that the FDA has authority to regulate not only for purity, but also for safety and effectiveness. On the first of these questions no one challenges the role of the FDA, either inside the industry or beyond it. But the issues about safety and effectiveness raise serious questions of institutional design, which are not well handled under the current regulatory regime. The key question is this: Where should decisions about drug safety and efficacy be made, upstream by the FDA or downstream by the individual drug user, aided by professional assistance?

The development of more complex therapies to treat chronic illnesses and complex diseases warrants a whole new approach to testing and regulating prescription drugs, according to a much anticipated report from the Institute of Medicine (IOM). Several high-profile drug withdrawals and safety alerts in recent years have undermined public confidence in the Food and Drug Administration and the pharmaceutical industry and raised concerns about the agency's ability to assure the safety of critical medicines, according to a panel of health experts assessing "The Future of Drug Safety." The study says that FDA needs more funding, stronger regulatory authority, and many internal reforms to enhance its ability to monitor and implement changes in drug use after a product comes to market.

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