A question- first approach to Rare Disease Studies
Overcoming the unique challenges of rare disease drug development starts with asking the right questions. This paper is a guide to critical questions to ask and best practices to employ when developing and outsourcing rare disease clinical trials. Based on ARG's decade of consulting around the world, this paper is designed to surface and mitigate the unique challenges rare disease studies present.
In this whitepaper, we address:
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Is your study protocol optimized for success in the intended population - and for the planned study sites?
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Is your recruitment plan realistic? Could it be accelerated?
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Are investigator sites adequately supported to ensure trial quality?