Adaptive Approaches in Clinical Drug Development: Opportunities and Challenges in Design and Implementation

The crisis in the overall productivity of pharmaceutical research and development (R&D) has attracted attention to innovative ways to improve drug development. This article describes such an initiative that resulted in the creation of a unique cross-functional group of experienced drug developers with backgrounds in clinical pharmacology, medicine and statistics. The objective of the initiative was to test whether a small task force would be able to dramatically improve clinical development planning on a strategic level by (i) ‘walking the talk’ of the ‘learn and confirm’ philosophy, through hands-on interactions with project teams across multiple therapeutic areas; (ii) enabling real-time learning in clinical drug development, through a culture open to innovative and adaptive approaches in the design, implementation and execution of clinical trials; (iii) serving as an integrator across functions and stakeholders, from discovery to commercial; and (iv) ensuring consistent quality in development planning, across much of the R&D portfolio. This adaptive approach has become a platform to integrate cross-functional expertise and to increase the information value per resource unit invested during drug development, through rigorous application of simulation guided planning of development strategies and clinical trials. We also describe opportunities and challenges to successfully designing and conducting adaptive trials in a pharmaceutical R&D environment, in light of recent regulatory developments and guidelines.