20 October 2017 - FDA incentives to encourage development of rare disease therapies have paid dividends, with an unprecedented number of novel treatments in the orphan drugs pipeline. 

But expedited regulatory review has also raised concerns about a lowered evidentiary bar when it comes to drug efficacy, cautioned speakers at an October 19 session of the Academy of Managed Care Pharmacy (AMCP) 2017 Nexus in Dallas.

“Rare diseases have come to the forefront and have captured everybody’s attention,” said Alexandra Lin, PharmD, manager, pharmacy clinical programs & customer support at Blue Cross Blue Shield of Michigan, in Detroit.

In 2016, the FDA approved 22 new drugs, of which 41% had an orphan drug designation, she reported.

Read Managed Healthcare Executive article