Observational Registries: Applications in Safety Evaluation. 8 th FDA/Industry Statistics Workshop Washington, D.C. September 14-16, 2005 Wei Dong MD PhD, Katie Miller MSc, Pavel Napalkov MD MPh Epidemiology Genentech, Inc. . Acknowledgements.
8th FDA/Industry Statistics Workshop
Washington, D.C. September 14-16, 2005
Wei Dong MD PhD, Katie Miller MSc, Pavel Napalkov MD MPh
healthyRegistries vs. RCT: Generalizability
Target Population: All Patients with Disease X
(few or no exclusion
Phase III Sampling
Amount of data collected
# of patients enrolled
Duration of follow-up
% of patients
Leukemia risk among patients with no risk factors
by “person-time at risk” vs. “person-time on therapy”
1. Kozloff M. et al. Safety of bevacizumab among patients receiving first-line chemotherapy for metastatic colorectal cancer- preliminary results from a large registry in the US (BRiTE). ASCO 2005.
2. Hurwtz H et al. Bevacizumab plus Irinotecan, Fluorouracil, and Leucovorin for Metastatic Colorectal Cancer. NEJM. 2004;350:2335-2342.
*Bevacizumab labeling  reports 2% GI perforation
Overall ICH: 4.7% (3.8-5.9)
1. Dong W et al.Safety Outcomes of Alteplase in Ischemic Stroke Patients with Special Characteristics. International Stroke Conference 2005.
*. UT reported in-hospital mortality. **. CASES reported outcomes at 90-day follow up. ***. Sample size and results as August.2001.