2005 Update on Accelerated FDA Approval of Drugs Used To Treat Hematologic Malignancies from the Research on Adverse Drug-Events and Reports (RADAR): Rate of Regular Approval Conversion Remains Low.

Background: At ASH 2004 we reported that prior to 2004, eight drugs had received accelerated approval (AA) for ten hematologic oncology indications, only one had converted to full approval, and three serious adverse events associated with two of these drugs were identified post-approval. Following our presentation, Congressman Ed Markey (D-Massachussets) in June 2005 issued a report (“Conspiracy of Silence”) that raised similar concerns about the low rates of conversion from accelerated to regular approval and the failure of the FDA in ensuring that drug companies complete the required post-marketing studies. We update the experience with accelerated FDA approval for hematologic oncology.

Methods: Information was obtained from the FDA on new drug applications and supplements for new uses, package inserts, and medical literature reviews.

Results: Of 11 drug indications that received AA for hematologic oncology between 1995 and 2005, AA was generally granted for 9 based on clinical trials with <100 patients that identified high response rates. In the past year, none have applied for conversion to regular approval. Bortezomib received regular approval for an indication different than that granted for AA. Three serious adverse drug reactions were identified for two indications (gemtuzumab ozogamicin, imatinib for GIST) within 2 years of AA approval.

Conclusion: Since 2004, one new drug received AA for a hematologic oncology indication, one drug received regular approval for an indication different from that indicated under AA, and although 1 more year has elapsed, conversion has yet to occur for nine AA hematologic oncology indications. It is imperative that the FDA establish clear milestones regarding conversion to regular approval at the time AA is granted.