The U.S. Food and Drug Administration (FDA) plays a vital role in the process of advancing breakthrough cancer therapies to the public. A strong scientific infrastructure at FDA is necessary to ensure that products progress quickly and provide meaningful benefit for patients within acceptable levels of risk. The agency, however, has struggled to keep pace with the enormous scientific and technological advances made in recent years because of previous years of budgetary cutbacks and underinvestment in scientific infrastructure.
On this page you will find information about some of the key policy issues that affect the FDA's ability to help translate cutting-edge cancer research into new treatments for patients.
In 2004, the U.S. Food and Drug Administration (FDA) launched the Critical Path Initiative, a national strategy to help bridge the gap between basic scientific discovery and medical product development and evaluation, in an effort to accelerate the number of innovative drugs and therapies reaching patients.
The initiative emerged after the release of Introduction or Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products, a white paper that called on the FDA to work in collaboration with federal agencies, patient groups, academic researchers, industry and other stakeholders to harness emerging breakthroughs in science and technology to modernize the agency's ability to evaluate the safety and effectiveness of medical products.
The Critical Path Initiative remains one of the agency's top priorities. Since its inception, the number of projects has grown from 40 in 2006 to 95 in 2009.
This report briefly describes more than 50 scientific projects undertaken with participation of FDA centers and offices that received Critical Path support from congressionally appropriated funding during fiscal year (FY) 2008.
This white paper called on the FDA to work in collaboration with a broad range of stakeholders to help modernize the agency's ability to evaluate the safety and effectiveness of medical products.
In 2007, the FDA Science Board's Subcommittee on Science and Technology issued the report, FDA Science and Mission at Risk. The Subcommittee concluded that the agency's scientific base had eroded, its scientific workforce lacked sufficient capacity and capability, and the information technology infrastructure was inadequate.
In 2009, a report released by the Government Accountability Office (GAO), the investigative arm of Congress, placed the FDA on its list of high-risk areas. It questioned the agency's ability to ensure the safety and efficacy of drugs, biologics and medical devices.
This report, released by the Government Accountability Office (GAO), the investigative arm of Congress, placed the FDA on its list of high-risk areas, questioning the FDA's ability to ensure the safety and efficacy of drugs, biologics and medical devices in addition to highlighting specific weaknesses.
This report, issued by the FDA Science Board's Subcommittee on Science and Technology, concluded that the agency's scientific base had eroded, its scientific workforce lacked sufficient capacity and capability, and the information technology infrastructure was inadequate.
This report represents the view of a committee of expert academic scientists and clinicians, research advocates, and representatives of the patient community convened in order to recommend ways in which policy makers in the United States Congress and the United States Food and Drug Administration (FDA) can strengthen product evaluation.
In 2004, the FDA made a historical shift in its approach to regulating cancer treatments by opening an Office of Oncology Drug Products. This was part of a larger effort to streamline product review, and better balance safety and risk considerations specific to oncology drugs. The goal was to develop a stronger, more consistent approach to the review process for cancer therapies to speed up the review of drugs and ensure their safety.
Off-label drug use refers to the practice of prescribing a therapy for any purpose, or in any manner, other than what is described in a product's FDA-approved label. Off-label drug use is particularly common in the treatment of cancer because a drug approved for one cancer is often also effective against other kinds of tumors, and chemotherapy often involves the use of multiple drugs. The treatment of cancer is constantly evolving as researchers discover new uses for existing cancer drugs and find effective new combinations.
In early 2009, the FDA released guidelines permitting drug manufacturers to provide information to doctors about off-label drug uses not approved by the FDA. The distribution of such literature has been a topic of controversy over recent years between the drug industry and its critics in Congress. Proponents argue that the more scientific and medical information that health care professionals have at their disposal, the better they can improve the health of the public. Opponents argue that such policies jeopardize public health by promoting drug uses that have not received adequate testing.
The Reagan-Udall Foundation is a public-private partnership authorized by law in 2007 to award grants to advance regulatory science in an effort to improve FDA evaluation, monitoring and regulation of existing and new technologies. Due to a perception among some members of Congress that the foundation lacked a framework to minimize industry influence, however, funds to the foundation have been blocked.