Keywords: rare disease
Even though prevalence of rare disease is low, roughly 30 million Americans have been affected by one or more of the nearly 7,000 rare diseases. For most rare diseases, it is challenging to conduct clinical trials with enough power to detect the treatment effect. To bring a breakthrough therapy to the market early, it is important to find efficient approaches to utilizing individual patient data (e.g., improved study designs and sound statistical methods).
Many workshops have been held in recent years to accelerate the development of therapies for rare diseases. Researchers from industry, academia, and regulatory agencies are working diligently to develop innovative trial designs and statistical methodologies for this area.
In this round table, we would like to provide a platform for all to share their experience in conducting rare disease clinical trials and to discuss their views and the key points from recently published guidance from FDA and EMA in this area. Questions to Ask: 1. What are the statistical considerations and issues for rare disease clinical trials? 2. How does the challenge of recruitment affect the implementation of clinical trials, and how can different designs, such as enrichment designs and adaptive designs, help assess the treatment effect? What challenges do these designs bring to interpretation? 3. When and how should we utilize historical controls?