Following yesterday’s international Rare Disease Day, here is a snapshot of four new rare disease treatments currently under review by England’s cost-effectiveness watchdog.
The FDA has marked today’s international Rare Disease Day with a number of measures to promote research and patient participation in policy and research.
Pharma company Shire and tech giant Microsoft are to team up with Eurodis, a not-for-profit group representing 700 rare disease organisations, with the goal of cutting the long wait most patients with rare conditions endure before diagnosis.
Vertex is trying reach a deal with NHS England to give patients access to all its approved cystic fibrosis (CF) drugs - and hopes it could also fund further drugs in the pipeline.
Vanessa dos Reis Ferreira is head of Patient Advocacy Europe at Santhera and a rare disease specialist. Here she describes how her company is focused on understanding patient needs to develop and bring to market the best possible medicines.
