The second in a series of supplements to look at orphan drugs and rare disease, this issue covers early access and regulation.
It features expert opinion on enabling access for patients with rare diseases, how to navigate the regulatory environment and the logistics of running a global access programme.
There’s a look at how to manage projects to ensure that patients needs are met.
View: Orphan drugs and rare diseases: Early Access and Regulation
Further online orphan drug and rare diseases resources are available from PMLiVE in its dedicated online section for the area.
This showcases our orphan drug and rare disease content, splitting it up for easy navigation into sections on CROs and trial design, early access and regulation, strategic communications and value demonstration.