Rare disease trials differ from those in most other therapeutic areas because the indications are more complex and involve smaller patient populations. It is essential to understand the current disease landscape, the prior research, and the competitive clinical trial environment when posi¬tioning a new clinical trial within the rare disease space. This white paper discusses strategies for accelerating rare disease clinical development by lever¬aging data in a more evidence-based approach to guide the design of a trial; to understand treat¬ment trends; and to identify the best investigators, sites, and patients.
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