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    • 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.

    Petauri Evidence

    - PMLiVE

    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',...

    Mednet

    - PMLiVE

    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.

    Petauri Evidence

    - PMLiVE

    Horizon looks bright as FDA panel backs eye drug teprotumumab

    First alternative to surgery for proptosis

    - PMLiVE

    Orphan drugs market to soar to $224bn by 2024

    Cell and gene therapies to fuel growth

    - PMLiVE

    Horizon sunny as thyroid eye disease drug hits the mark

    New treatment helps lessen eye bulging

    - PMLiVE

    ‘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...

    Inizio

    Malthus’ orphans

    Firms focusing on small, high-profit niches risk falling into a 200-year-old trap

    - PMLiVE

    EMA hands UniQure orphan status first in Huntington’s

    The Dutch biotech expects to file the treatment for approval later this year

    Roche Basel Switzerland

    Roche claims its own CAR-T first with orphan drug Actemra

    Comes as FDA approves the first CAR-T therapy from Novartis

    - PMLiVE

    FDA moves to tackle orphan designation backlog

    Some 200 requests are awaiting review by the US regulator

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