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MEDIDATA: Rare Disease Clinical Development: Novel Approaches to Overcoming Operational Challenges

As the age of the blockbuster drug gives way to precision medicine and more targeted patient populations, industry is applying innovative methods and technologies that reduce the cost, complexity, and risk of researching and developing treatments for rare diseases. 
Solutions range from analytical platforms that can identify tools and trial designs that reduce the burden on patients... to methods for increasing patient engagement and sophisticated approaches for managing and analyzing data, Building a strategy that applies all of these these solutions to rare disease clinical drug development enables underserved populations to benefit from these investments and to find the cures they need sooner.

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