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Executive Summary

FDA PLAN FOR EXPEDITING APPROVALS "SHOULD BE BROADENED" in scope to cover not only life-threatening diseases but also drugs to enhance survival of patients with critical conditions and to treat "major irreversible morbidity," the Pharmaceutical Manufacturers Association recommended in a Sept. 12 letter to HHS Secretary Bowen. In comments on FDA's proposal for expedited approvals of drugs for life threatening diseases, the association suggested that the preliminary proposal should apply to drugs for "conditions where the end point of clinical analysis is survival," such as increased survival after heart attack, and drugs "to treat major irreversible morbidity (e.g., Alzheimer's disease, illnesses leading to blindness, and prevention of recurrences of breast cancer after surgery)." PMA supports FDA's current plan to implement the proposal through administrative channels rather than through legislation. A draft of the proposal is currently being reviewed by the Office of Management & Budget ("The Pink Sheet" Sept. 12, p. 3) In its comments, signed by PMA President Mossinghoff, the association stated that changes in FDA's operating method "should be done through administrative rather than legislative changes." The agency's approval of Burroughs-Wellcome's Retrovir (AZT) for AIDS provides a model for an expedited drug review under current statutory authority, PMA agreed. "This model is now well understood by scientists, industry, and government health regulators, who could readily react to a formal proposal." The FD&C Act gives the agency "broad flexibility in approving products," the association continued. FDA "has the power to adopt and fine-tune through rule-making procedures the character, quantity, and format of scientific information needed to demonstrate a product's utility and safety." For example, PMA maintained, in considering the risks and benefits of certain drugs, the agency should relax "the rigid demands of the current regulations for more than one adequate and well-controlled study." PMA endorsed the agency's proposed approach to risk-benefit analysis when reviewing drugs for life-threatening illnesses, major morbidity, and survival enhancement. The program should emphasize "the judgment of experts who are trained to make these benefit/risk determinations," and who should be permitted to make them "on the entire database of evidence that is available," the association recommended. "The emphasis should be on the quality, and not the number, of studies." The association also urged the Administration to avoid a plan that would produce conditional approvals. "The end result" of the expedited process "should be a fully approved NDA," PMA contended. "Conditional approval would be a barrier to widespread and effective use of the drug and would create serious legal and product liability problems." PMA further recommended that FDA should limit its involvement in drug research to counseling and review. A proposed provision for FDA's performance of pharmacodynamics and pharmacokinetics studies for small, inexperienced companies should be limited to only "the most extraordinary circumstances." PMA also suggested that FDA's proposed involvement in animal studies should be "at the election of the sponsor" to ensure that companies' research processes are not impeded.

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