Article
The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis.
Orphanet journal of rare diseases - 7 May 2022
Costa Enrico, Girotti Silvia, Pauro Francesca, Leufkens Hubert G M, Cipolli Marco
Abstract excerpt
BACKGROUND: Over the past decade, a new class of drugs called CFTR (cystic fibrosis transmembrane conductance regulator) modulators have shown to be able to improve clinical outcomes in patient with Cystic Fibrosis. In this analysis, we have extensively reviewed the regulatory pathways and decisions adopted by FDA and EMA to speed up the development, the review and the approval of these drugs, with the aim of...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.