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: nmurad@partners4access.com The US National
Partners4Access Briefing: November 2018 update on the European Commission regulation proposal on health technology assessment
By Christina Poschen, Consultant Email: cposchen@partners4access.com Background Since 2004, the European Union’s centralised drug approval is in place, allowing manufacturers to submit just one application to
Innovative Access Agreements: A potential payer solution for Cell & Gene Therapies
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
CAR-T Therapies, the future for cancer patients? A market access perspective
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
What does the new FDA accelerated regulatory pathway mean for gene therapies?
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
Biopharma 2018 Success Kit: 11 months to go, 6 rare disease trends to watch!
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
ICER finds Roche’s Hemlibra cost-effective despite high price tag
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
Sanofi to buy Bioverativ in $11.6 billion deal
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
UK regulator attempts to ease fears of impending access abyss
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
