Whitepapers
Rare Diseases: A New Purpose
How drug repurposing could help drive orphan drug access if pharma can accept the risks By Nader Murad, Senior Analyst Email: [email protected] The US National
November 14, 2018
How drug repurposing could help drive orphan drug access if pharma can accept the risks By Nader Murad, Senior Analyst Email: [email protected] The US National
By Christina Poschen, Consultant Email: [email protected] Background Since 2004, the European Union’s centralised drug approval is in place, allowing manufacturers to submit just one application to
A US and EU perspective By Daniele Severi Bruni & Sophie Schmitz What’s the idea? Payer concerns over increasing cost of therapies and uncertainty of
Chimeric Antigen Receptor T-cell (CAR-T) therapies, which use a patient’s own immune cells to fight cancer, are one of the most exciting breakthroughs in cancer
U.S. Food and Drug Administration (FDA) commissioner Scott Gottlieb has announced that the drug agency is developing a system by which innovative gene therapies could
For the rapidly developing orphan drug market, change is a constant. From new technologies to discussions over pricing, the estimated $137 billion market will witness
Roche’s new haemophilia treatment, Hemlibra, is has a high estimated price tag of $482,000 for the first year of treatment and $448,000 for every subsequent
French pharmaceutical giant Sanofi have agreed to acquire Bioverativ, a U.S. hemophilia specialist for $11.6 billion. This deal is the biggest for Sanofi in seven
The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) announced that there would be no sudden changes to the UK regulatory framework in the event
