Current Edition

The Case for Response Adaptive Trial Design for Rare Diseases

With 7,000 rare diseases affecting 350 million people worldwide, Holly Jackson at Quanticate identifies how businesses exploring rare diseases can continue their success through developing a comprehensive understanding of the clinical trial design required. She looks at moving from randomized control to response adaptive trials, Bayesian Adaptive designs, personalised medicine and proposed an adaptive trial design, concluding that adoptive designs are a promising option for rare disease trial sponsors.