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Executive Summary

FDA SHOULD EXPEDITE SCLERODERMA STUDY, REP. DINGELL URGES Commissioner Kessler in a March 30 letter on the agency's handling of Therakos' submission for the combination drug-device photopheresis treatment. "FDA must move quickly to help, not hinder, the study of scleroderma," the Michigan Democrat said. "The thousands of Americans who suffer from the severe and fatal form of this disease for which there is no FDA-approved cure deserve some hope." FDA Center for Drug Evaluation & Research Deputy Director for Medical Affairs Bruce Burlington, MD, recently has testified that treatments for severe scleroderma would qualify for accelerated approval procedures ("The Pink Sheet" March 23, T&G-17). However, FDA currently cannot take action on the combo product since the Johnson & Johnson subsidiary Therakos withdrew its marketing application for its UVAR photopheresis device on Jan. 21. ICN, which provided the drug component of the therapy, Oxsoralen (methoxsalen), then withdrew its application ("The Pink Sheet" Jan. 27, p. 3). Two days later, an FDA advisory committee agreed that the data were "either inconclusive or negative." Dingell held a Feb. 6 hearing before his House Energy & Commerce/Oversight subcommittee on the miscommunications between FDA and Therakos ("The Pink Sheet" Feb. 10, p. 6). Summarizing his subcommittee's findings, Dingell told Kessler that "three categories of problems strike at the heart of FDA's ability to discharge its statutory obligations in a fair, objective and efficient manner. First, Dingell said that FDA audits and requests for data on photopheresis for scleroderma did not resolve issues but obfuscated them and appeared to be prejudicial to the application. The congressman cited an apparent bias in FDA auditing of Therakos data which reported in one instance that a patient, who was not in the study protocol but was treated on a compassionate basis and subsequently died, had died "while admitted for photopheresis." Dingell noted that the patient died not at the University of Pennsylvania clinical center but in a New Jersey hospital 197 days after her last treatment. A second problem with FDA's review procedures involves use of outside experts, the letter states. "FDA must improve the manner in which it uses advisory committees and outside consultants," Dingell maintained (his emphasis). Specifically, the letter asserts, "accurate data must be provided to FDA consultants and advisory committees on a timely basis so that these experts can produce studies and give opinions that will provide maximum benefit to the FDA." In addition, it continues, "FDA reviewers would benefit by giving copies of consultant reports to applicants several weeks before any meetings to discuss these reports." Dingell's recommendation addresses a September 1990 meeting between Therakos and FDA Pilot Drug Evaluation Staff Director John Harter, MD, at which Harter announced that further studies were needed to support photopheresis for scleroderma. The firm had asked for but "amazingly had never been shown" a consultant's analysis on which FDA's conclusion was based, Dingell's letter states. Consequently, Therakos was "in no position to provide a thoughtful and detailed response." Further, the letter continues, Harter has admitted "that he had not had the time to perform a detailed review of the Therakos submission himself. Even worse, Dr. Harter had received [the consultant's] written analysis only two days before the meeting." FDA "appeared to accept the views of his consultant without performing a detailed analysis on his own and before giving the applicants the ability to respond to the consultant," Dingell said. "It is little wonder that the meeting is said to have become heated and left feelings of distrust among all its participants." The third problem cited by Dingell is "a serious weakness in FDA's review and handling of protocols for clinical trials -- a matter that goes to the very heart of the approval process." The congressman was referring to instances, discussed during his February hearing, when the agency approved protocols but then changed its mind after initiation of studies. "FDA clearly has bungled" photopheresis applications for T-cell lymphoma (which is approved) and for scleroderma, he said.

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