Using Probability of Program Success and Expected Net Present Value to Compare Alternative Development Strategies: A Case Study from the PhRMA Adaptive Dose-Ranging Studies WG
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*Jose Carlos Pinheiro, Johnson & Johnson PRD 

Keywords: Probability of success, NPV, dose-finding, adaptive design

The pipeline problem experienced by the pharmaceutical industry, coupled with critical imminent patent expirations and high Phase III attrition rates, have led to great interest in innovative designs and analysis methods to improve the efficiency of drug development. Evaluation of such methods often involves extensive simulations and tends to focus on a single study. This talk considers the evaluation of alternative approaches under a program development level perspective, focusing on the impact of alternative dose-ranging approaches and alternative Phase III designs on the outcome of the confirmatory programs. The probability of program success and the expected net present value of the program are used as evaluation metrics. Data from an extensive simulation study conducted by the PhRMA working group on Adaptive Dose-Ranging Studies will be used to motivate and illustrate the methods.