Determine the Primary Endpoint in Infection Disease Studies

• Activity Number: 355
• Type: Contributed
• Date/Time: Tuesday, August 6, 2013 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Chunzhang Wu*+
• Companies: Astellas Pharma Global Development, Inc.
• Keywords: primary endpoint ; non-inferiority margin ; assay sensitivity ; placebo effect ; random effect

Abstract

For fungal infection disease, such as invasive aspergillosis infection, mycological, clinical, and radiological responses are mostly standard assessments to determine the treatment effect for infected patients. However, due to difficulty of early diagnosis for confirmed invasive aspergillosis infection and impossible to have placebo data related to these type of clinical assessments, it is difficult to design an appropriate powered clinical trial with well justified non-inferiority margin if use one response or composite responses as the primary endpoint. This paper in depth to discuss to consider, instead, using all-cause mortality as the primary endpoint to design an appropriate powered study, and how to justify the non-inferiority margin when historical placebo clinical response data were deemed impossible. Some regulatory submission consideration are also presented.