WASHINGTON -- A Food and Drug Administration advisorycommittee on Friday recommended approval of Bristol-MeyersSquibb Co.'s dideoxyinosine (ddI) for treatment of adults andchildren with AIDS, but only for patients who cannot tolerateor fail to respond to Burroughs Wellcome's AZT.
The rapid action on the ddI application, coming in only threeand a half months and before clinical trials were completed,created misgivings among the committee members, who askedfor an opportunity to rescind their decision when more databecomes available.
Agency head David Kessler has set a goal of accelerating thereview of AIDS treatments, since there is currently noalternative to AZT.
The Antiviral Drug Products Advisory Committee voted 5-2 toapprove ddI, but then voted 6-1 to recommend intolerance andefficacy limits on the indication for adults, and voted 5-2 toimpose the same limits on the treatment for children.
Committee Chairman Dr. Henry Masur said the acceleratedreview process, in which interim endpoints were used toevaluate data rather than wait for the completion of clinicaltrials, is "clearly unprecedented and historic."
But committee members expressed considerable queasinessabout giving an unconditional recommendation for approvalafter the FDA rushed through collection and analysis of datafrom preliminary studies and an as-yet unfinished expandedtreatment program.
Masur said the committee sent a unanimous and clear messageto the FDA that it wants the ongoing randomized clinical ddItrials to be brought to their scheduled completion this winter,and at that time the committee wants a chance to review thedata.
The committee wants the option to recommend that the FDAreverse any approval for marketing ddI or rescind and revisethe label indication if the final data leads to negativeconclusions about ddI's efficacy and toxicity, Masur toldBioWorld. "It's still conceivable that we will want to withdraweven the approval for the limited population," he said.
Commissioner Kessler said the FDA is considering anaccelerated approval mechanism that "will allow re-review ofdata on completion of clinical trials with acceleratedwithdrawal of marketing approval" if necessary. Kesslerpromised that a final agency decision on ddI would comerapidly and that the committee would be reconvened to re-review the study data at the end of the trials.
Several committee members expressed concern that Friday'srecommendation would set a precedent for "ratcheting down"FDA's approval standard and will remove drug companies'incentive to conduct carefully controlled studies. Committeemember Dr. Deborah Cotton, who voted against approval, calledit a "rush to judgment."
-- Kris Herbst BioWorld Washington Bureau
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