A Rational Approach to Drug Development: The Confirmatory Phase

Abstract

A rational approach to drug development would be model-based, target label and knowledge driven where the objective is to characterize the response surface – the interplay of drug regimen/exposure, and patient factors to elicit response (efficacy/safety) – that would result in the right dose for the right patient at the time of marketing the drug. This implies using population PK/PD, knowledge discovery and creation approaches, clinical trial simulation, appropriate surrogate/clinical endpoints, and appropriate statistical analysis for the characterization of the response surface. It also implies interacting with regulatory authorities prior to the initiation of clinical development and throughout the phases of clinical development to ensure that appropriate data to support a new drug application are being generated. To enable further characterization of the response surface in the later phases of development (i.e. phase IIb and beyond) and registration, the confirm–learn aspect of the learn–confirm–learn paradigm of drug development is advocated as the basis for answering the crucial drug development questions – “Is the NCE tolerated and at what dose range?” and “Does it work within the tolerated dose range?”

Key words: :

• Common Technical Document
• Confirmatory Trials
• Investigational New Drug Application
• Learn-Confirm-Learn
• New Drug Application
• Pharmaco Kinetic/Pharmacodynamic Knowledge Discovery and Creastion
• Rational Drug Development
• Response Surface