Report card for accelerated FDA approval oncology drugs (1995- 2003): Is it time for a make-up test? S. M. Belknap, E. A. Lyons, J. M. McKoy, C. L. Bennett; Northwestern University, Chicago, IL Abstract: Background: The FDA adopted accelerated approval in 1992 in order to improve access to new drugs based on studies that evaluated markers likely to predict clinical benefit. Methods: The performance of this program was assessed by reviewing new oncology licensing applications and supplements for new uses approved by the FDA from 1995 to September 2003. Data included: the application filing date for each agent, the date of approval, the types of studies that were assessed during FDA review, and the endpoints used in these studies. Results: Of the 21 oncologic indications which received accelerated FDA approval, subsequent studies that were required for conversion to regular approval were submitted and accepted (n=5) [capecitabine, dexrazoxane, docetaxiel, imitinab mesylate, irinotecan], submitted for regular approval but rejected by the FDA (n=3), or have not been submitted (n=14). Surrogate outcomes included response rate (n=16), symptom improvement (n=1) and reduced time to recurrence (n=1). Fourteen approvals were based on results of phase II clinical trials, while only 5 were based on phase III studies. Fewer than 200 patients were evaluated for efficacy for about 2/3 of the indications. Post-marketing studies identified potentially fatal adverse drug reactions for four of the drugs, three of which were identified within 7 months of initial FDA approval (capecitabine-associated interaction with warfarin, imatinib-associated hemorrhage in GIST, and gemtuzumab-associated VOD). Conclusions: To date, drugs which have received accelerated FDA approval have been frequently evaluated in small, short-term phase II clinical trials, have rarely been converted to regular FDA approval, and can have potentially fatal adverse drug reactions identified in the first year of marketing. Abstract No: 6002 Remember we are NOT Doctors and have NO medical training. This site is like an Encylopedia - there are many pages, many links on many topics. Support our work with any size DONATION - see left side of any page - for how to donate. You can help raise awareness of CAM.