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Executive Summary

Legislation introduced by Rep. Campbell (R-Calif.) would require FDA to permit marketing of drugs and biologics for patients with life-threatening diseases and seriously debilitating illnesses when the products meet a series of conditions. Campbell introduced the "Access to Life-saving Therapies Act" July 11 to authorize FDA to approve drugs and biologics based on surrogate markers when there is no "alternative satisfactory therapy" for the intended patient population. The bill (HR 2872) provides that, "at the request of the sponsor," FDA "shall approve on an expedited basis a drug or biologic needed to treat or prevent a life-threatening disease or seriously debilitating illness" under certain conditions. The measure notes that qualifying products would include therapies for AIDS, cancer, Alzheimer's disease, cardiovascular diseases and Parkinson's disease. Similar legislation is being developed by Rep. Bliley (R-Va.) and Sen. Hatch (R-Utah) ("The Pink Sheet" June 17, T&G-1). Key Democrats in the House and Senate have generally believed that FDA can establish accelerated approval process without statutory authorization. However, the legislators have indicated they will aid the process by showing support through legislative activity. Under the Campbell bill, conditions for expedited approval include "sufficient information," such as "surrogate markers, from preclinical and early clinical studies" to show "promising evidence" of efficacy and "to indicate that the drug is reasonably safe, taking into account the intended use and the patient population for which the drug is intended." In addition there must be "a lack of alternative satisfactory therapy" for the intended patient population, "sufficient pharmacokinetic and dose-response information to recommend a reasonable dosing regimen." Furthermore, the sponsor must have "begun one or more controlled clinical trials of the drug," and there must be "sufficient quantities of the drug to conduct the trial or trials and satisfy the needs of the market." Sponsors also must "establish and maintain a system for collecting data and information on the use of and experience with the drug or biologic and for monitoring patients for adverse effects," and they must "continue and complete adequate and well controlled investigations for the drug or biologic to provide additional data to confirm the initial conclusions on safety and efficacy which formed the basis for approval unless such investigations are not feasible." Expedited approvals under the legislation would be terminated if ongoing clinical trials fail to demonstrate safety and efficacy. "FDA shall suspend the approval of an application . . . on the basis of new information from at least two post-approval studies that fail to confirm the initial conclusions on the safety and efficacy of the drug that formed the basis for approval," the bill states. The introduction to the bill maintains that "the current mechanisms for expanded access to experimental therapies through parallel track, Group C designation and Treatment INDs do not adequately meet the needs of patients with life-threatening or seriously debilitating diseases." The legislation also mandates that therapies approved under the expedited procedure be covered by government and private health care insurance plans. In a House floor statement, Campbell estimated that 8,000- 15,000 persons died from gastric ulcers during the time misoprostol (Searle's Cytotec) was reviewed by FDA. "By the time the drug was approved in the U.S., it was already available in 43 countries," Campbell said, adding: "in some of them three years earlier." He also suggested that the bill would help provide W-L's Cognex to patients who would benefit from the drug. Cognex "has helped many" Alzheimers patients in clinical trials, yet "because FDA is not completely satisfied with the efficacy of THA, many who might benefit from it are prevented from doing so." Similarly, he continued, "two drugs for AIDS, ddI and ddC, have shown great promise, and AIDS victims are desperately seeking access to them."

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