early access and regulation
Challenges for orphan medicines entering the European market
We explore the various challenges facing orphan medicines entering the European market and offer solutions to help improve patient access.
What are “orphan drugs” and how do we navigate the challenges of writing about them?
The first in our 'Ask Alex' series, where Principal Medical Writer, Dr Alexandra Loveday answers some frequently asked questions about Medcomms. This week, she takes a look at 'orphan drugs',...
Rare disease market access – Launching orphan drugs in secondary markets: the Nordic and MENA regions
Join us live at 1.00pm (GMT)/2.00pm (CET)/8.00am (EST) on Thursday 29th February, to explore rare disease market access challenges and learn more about these high potential markets.
Horizon looks bright as FDA panel backs eye drug teprotumumab
First alternative to surgery for proptosis
‘Inflexible’ NICE blocking access to rare disease drugs
Spinraza most high profile example of access problem
Rare diseases and orphan drugs: Our experience and expertise
We have designed a complimentary infographic outlining the market opportunity as well as our experience and expertise in the growing area of rare diseases and orphan drugs, which includes links to...
Malthus’ orphans
Firms focusing on small, high-profit niches risk falling into a 200-year-old trap
EMA hands UniQure orphan status first in Huntington’s
The Dutch biotech expects to file the treatment for approval later this year
Roche claims its own CAR-T first with orphan drug Actemra
Comes as FDA approves the first CAR-T therapy from Novartis
FDA moves to tackle orphan designation backlog
Some 200 requests are awaiting review by the US regulator