Challenges in Designing Clinical Trials for Rare Diseases

• Activity Number: 277
• Type: Topic Contributed
• Date/Time: Tuesday, August 5, 2014 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Christopher Coffey and Eric Foster*+
• Companies: University of Iowa and University of Iowa
• Keywords: Rare Diseases ; Clinical Trials ; Adaptive Designs

Abstract

Designing clinical trials in the rare disease setting presents unique challenges to the clinical trials community. Summary statistics from small trials are often imprecise, so that the trials have adequate power to detect only large effects of interventions. Furthermore, many standard statistical techniques are based on large sample assumptions adn may not have desirable properties in the small sample setting. As a consequence, the importance of adequate study planning is magnified in small clinical trials. Hence, it is critically important to have a true collaboration between study investigators and statisticians. Based on recent experience with the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT), this presentation will review some specific designs that seem useful in the rare disease setting. In particular, this presentation will address how adaptive designs (which have received substantial recent interest) can be useful in the rare disease setting - and what specific barriers currently impede the use of adaptive designs in current funding environments.