Speaker Abstract: S-34
Adverse Event Analysis of Gene Therapy Clinical Trials based on a comprehensive database: What are the risks?
Steven Hirschfeld, M.D., Ph.D., Center for Biologics Evaluation & Research/FDA
Title: Is participation in clinical trials using gene therapy products safe?
Background: Available data indicate the likelihood of receiving some benefit from participation in clinical trials, but few data are available with regard to the risks. This is particularly true for newer types of products such as gene therapy.
Methods: A database maintained in MS Access containing all adverse events obtained from spontaneous reports and annual summaries of clinical trials using gene therapy products is being developed at the Center for Biologics Research and Evaluation at the the Food and Drug Administration. Between 11000 and 12000 events obtained from spontaneous filings and annual reports submitted to the FDA have been entered of which about 7500 occurred in cancer patients. This represents about 1800 patients enrolled in about 172 clinical trials. Database queries were constructed to examine general characteristics of the events, diseases, and patients.
Results: The most common adverse events recorded were disease progression (15% of all events), injection site pain (6%) , pyrexia (4%), injection site reaction (3%) , and other pain (2%). The most common serious adverse events (death, prolonged hospitalization, major intervention) occurred in proportion to the use of a product and tended to be disease related. Of 1003 deaths about 770 were considered due to disease progression. The most common diagnoses were metastatic melanoma, glioblastoma multiforme, squamous cell carcinoma of the lung, metastatic renal cell carcinoma, disseminated neuroblastoma, metastatic breast cancer and prostate cancer. The second most frequent cause of death was cardiorespiratory arrest, which occurred in about 1% of all patients. The most frequent cause of prolonged hospitalization was febrile neutropenia, occurring in about 3% of all patients. Detailed analysis of the patients, events, and products did not reveal other risk factors in addition to primary diagnosis
Conclusions: Participation in gene therapy studies does not appear to carry additional risks beyond those expected of patients with relapsed or refractory life threatening disease.
2004 FDA Science Forum | FDA Chapter, Sigma Xi | CFSAN | FDA
Last updated on 2004-MAY-28 by frf