NICE has recommended the NHS should fund Novartis’ Luxturna for a rare eye disorder, after the company agreed a discount from the list price of more than £613,000 per patient.
The FDA has just made it easier for Sweden’s Calliditas to develop Nefecon for a rare kidney disease, accepting a faster readout that should hasten development of the drug.
Alexion’s rare disease drug Soliris has been approved in a new use in Europe, to tackle a disorder of the optic nerves for which it recently got FDA approval.
NHS England has agreed to reimburse Roche’s Hemlibra (emicizumab) in a new group of patients with haemophilia A, widening access to the potentially life-saving drug.
Scotland’s cost-effectiveness body has rejected regular NHS funding for Vertex’s cystic fibrosis drugs Orkambi and Symkevi because of concerns about whether their high costs justify uncerta
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.