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Rare Disease Goalposts: A Conundrum For Pharma, FDA

This article was originally published in Scrip

Executive Summary

Having clinical trial endpoints that are pragmatic and doable and a clear understanding of what the goalposts are for winning the FDA's approval to get a drug on the market will help decrease the uncertainty and drive investment, said Rakesh Marwah of Palo Alto Investors LLC, a Silicon Valley health care investment firm.

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