HPR150 P&R Policies and Macro-Trends Pertinent to CGTs: Where Are the Main Barriers and Who Leads the Efforts to a Greater Access?

Abstract

Cell and gene therapies (CGTs) is a rapidly growing therapeutic area holding great promise for treating rare diseases with high unmet medical needs, with nearly 60% of the 2,400 ongoing CGT trials at the end of 2021 targeting prevalent diseases. Our paper aims to highlight, among the markets in scope, where recent policy developments suggest the most promising or challenging outlook.