Investigational New Drugs: FDA's Expanded Access Program, Statement of John E. Dicken, Director, Health Care, Testimony Before the Subcommittee on Health, Committee on Energy and Commerce, House of Representatives   [open pdf - 161KB]

"I am pleased to be here today to discuss our recent report on the Food and Drug Administration's (FDA) expanded access program. As you know, through FDA's expanded access program, patients with serious or life-threatening ailments and no other comparable medical options can obtain access to investigational drugs--drugs not yet approved by FDA for marketing in the United States--outside of a clinical trial when appropriate. FDA receives and reviews expanded access requests, and determines whether to allow them to proceed. Other entities also have roles in the process. For example, manufacturers decide whether to give patients access to their investigational drugs; institutional review boards must approve patients' expanded access treatment plans; and physicians treat the patients with the investigational drugs, and monitor their progress. FDA's expanded access program has been criticized by some physician and patient advocacy groups for being too burdensome and confusing to the entities involved, which could pose a barrier to individuals' access to investigational drugs. Additionally, manufacturers have raised questions about how FDA might consider data from expanded access use in its process for approving the drug for marketing in the United States. However, stakeholders--including physicians, patients, and patient advocates--have also highlighted steps FDA and other stakeholders have taken to improve the program."

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Government Accountability Office: http://www.gao.gov/
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