Impact of Missing Data on the Approvability of Potentially Efficacious Therapies

• Activity Number: 197
• Type: Roundtables
• Date/Time: Monday, August 5, 2013 : 12:30 PM to 1:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Abdul Sankoh and Cynthia DeSouza+
• Companies: Vertex Pharmaceuticals and Vertex Pharmaceuticals
• Keywords: Missing data ; Preventive measures ; Remedial properties

Abstract

There are a number of operationally preventive measures and some statistical approaches with seemingly remedial properties in the clinical and statistical literature that, if appropriately implemented during the design, conduct, and analysis of clinical trial data, should mitigate the occurrence and thus the detrimental impact of missing data on trial outcome and subsequent approval of potentially safe and efficacious new drugs. Nonetheless, many drugs with potential therapeutic benefit have failed to gain approval due to serious missing data issues. This roundtable discussion will probe into the challenges posed by missing data in clinical trials and the utility of current approaches in minimizing the non-approval chances of potentially efficacious new therapies.