Ahead of eyeforpharma Philadelphia 2019, Sonal Bhatia, Pfizer’s vice president, North America medical lead, rare disease talked to us about the importance of collaboration and RWE to he
Vertex CEO Jeffrey Leiden has offered an olive branch to negotiators in the UK that could see the cystic fibrosis drug Orkambi finally supplied to patients in England.
European regulators have recommended a licence for Akcea and Ionis’ ultra-rare disease drug Waylivra, a therapy that the FDA rejected for the US market last year.
To have any impact, patient engagement needs to start with the drug development pathway, not at the point of licensing. That’s especially true in the rare disease space, where the unme