FDA Articles

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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Patient reported outcomes (PROs) are data obtained directly from patient self-reports, and their use in clinical trials is increasingly common.1 The U.S. Food and Drug Administration (FDA) recently published for comment their "Patient Reported Outcome (PRO) Measures: Use in Medical Product Development to Support Labeling Claims,"2 hereafter referred to as the PRO Guidance, to describe how they will evaluate PRO instruments. The PRO Guidance lays out the type and quality of information that clinical researchers will need to provide to justify the use of a particular PRO instrument in a trial and highlights the significance of PRO data in drug development.

By setting ground rules for use of PROs, the FDA has implicitly given credibility to PROs as the basis for evaluating drugs and biologics. Further, such guidance will eventually make for more efficient, effective, and appropriate use of these tools.

As the Food and Drug Administration celebrated its 100th birthday in June, it unveiled a major initiative for overhauling how it regulates clinical trials and protects participants in research studies. In outlining the plan for the Human Subject Protection and Bioresearch Monitoring (HSP/BIMO) initiative at the annual meeting of the Drug Information Association (DIA), Deputy Commissioner for Operations Janet Woodcock noted that it reflects the emergence of an "increasingly large, decentralized, and global" research enterprise. She cited the need to respond to dramatic changes in the nature and conduct of clinical research characterized by:

a steady increase in the number of clinical trial studies and sites in the United States and abroad
larger trials involving many small, individual sites
proliferation of different electronic record-keeping methods and systems for data collection and processing

Like the rest of the pharmaceutical industry, Pfizer must react to FDA guidance issued June 9 that speeds the adoption of track-and-trace technology—ultimately these will be radio-frequency identification (RFID) systems—in the nation's drug-supply chain. RFID tags on medicine packages emit a unique radio signal that, unlike a bar code, can be read in bulk by electronic readers, greatly reducing the time it takes to register the arrival or departure of individual packages within a large shipment of product. Whole lots can be read on pallets without being unpacked. By checking that every package carries a unique number, the system authenticates that each package in the shipment is the same as the one that left the manufacturer's facility on a certain date.

Yes, the US Food and Drug Administration has issued its long-awaited task force report and has stated: "We continue to believe that RFID is the most promising technology for implementing electronic track and trace in the drug supply chain and that the stakeholders should move quickly to implement this technology." No, it's not a mandate, but it's clearly an "almost." The June 8, 2006 Drug Taskforce Report has the makings of becoming a high-level doctrine toward building a more secure and dependable system for safeguarding the drug supply chain.

Baby steps. With more than 24 months of in-depth research, the taskforce has been examining RFID technology and the search for the Holy Grail: an e-pedigree solution that will stem the tide of drug counterfeiting, black marketing, gray market diversion, and tampering.

The concepts for a new FDA framework for regulating manufacturing—the framework that now supports quality-by-design, risk-based regulation, process analytical technology (PAT), and their kin—began to emerge around 2002. The agency was looking for ways to loosen some of the regulatory bonds that, officials felt, constrained manufacturing progress in the United States—while finding a way to cope with the combined pressures of a growing number of products, fewer FDA inspections, diminishing financial resources, advances in technology, an emphasis on risk-based regulation, and international harmonization (1, 2).

The industry press has written a lot about those planned changes in the years since, but generally in the future tense (3, 4). The word "paradigm" was used; for once, it might have been appropriate. But, there were also raised eyebrows and an implied, "if it happens."

It's happening.

April 1 is usually a day to expect the unexpected. However, on April 1, 2005, the U.S. Food and Drug Administration (FDA) released the "Guidance for Industry, Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics."1 The document describes the agency's "current thinking" while providing "recommendations to sponsors on endpoints for cancer clinical trials submitted to the FDA."

The following article explores the impact this guidance is having on the clinical research industry and the increasingly important role that imaging has in the assessment of cancer tumors.

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The Food and Drug Administration unveiled its long-awaited Critical Path Opportunities List in March, a summary of some 76 scientific projects that agency leaders believe can increase the efficiency, predictability, and productivity of medical product research and development. At the top of the list are a number of initiatives to identify and validate new biomarkers for clinical studies and product evaluation, along with proposals to improve clinical research practices and trial design. New statistical and computational approaches may make clinical research more informative, while computer modeling promises to avoid wasted efforts and streamline development programs.

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After months of turmoil over drug safety, vaccine shortages, and pressure to import low-cost drugs from abroad, the Food and Drug Administration faces further internal upheaval. Commissioner Lester Crawford resigned abruptly in September after only two months in the top spot, generating considerable uncertainty about the future leadership of the agency. The White House quickly named Andrew von Eschenbach, director of the National Cancer Institute (NCI) at the National Institutes of Health (NIH), as acting FDA commissioner. Von Eschenbach agreed to drop day-to-day management of NCI after being blasted for proposing to lead both NCI and FDA. He faced clear conflicts of interest in advocating for speedy access to new cancer treatments while overseeing the assessment of safety and effectiveness in those therapies. Leading legislators have urged the White House to nominate a permanent, full-time FDA commissioner.

Q. In 2005, what are the FDA's current and emerging priorities for its bioresearch monitoring program and GCP compliance?

A. In December 2004, the FDA began a major initiative to re-assess its GCP and bioresearch monitoring programs within all of its various program centers, including CDER, CBER, and CDRH. An internal FDA working group comprising representatives from throughout these centers is actively examining the entire BIMO process to re-evaluate how it works currently, what aspects of the program can be improved, how the centers can better coordinate, how the BIMO program can better focus on the highest-risk clinical studies, and how the agency can improve oversight of clinical trials involving special and vulnerable populations, including pediatric

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