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.