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orphan medicines

This page shows the latest orphan medicines news and features for those working in and with pharma, biotech and healthcare.

EMA forges closer ties with HTA umbrella group

EMA forges closer ties with HTA umbrella group

The EMA has extended a partnership with the organisation representing Europe’s health technology assessment (HTA) bodies, aimed at improving access to medicines, for another three years. ... Other projects will look at how the regulator and HTAs define

Latest news

  • The future of biotech The future of biotech

    IMS data for 2015 highlighted that six out of ten of the world's best-selling medicines were biologics: $18bn for Harvoni, a new medicine; $15bn for Humira and $6bn for ... In the US there may be federal developments. In the UK it has led to fragmented

  • Orphan drugs: Leading the way in patient-centricity Orphan drugs: Leading the way in patient-centricity

    Orphan drugs: Leading the way in patient-centricity. What lessons about optimal patient engagement can big pharma learn from those operating in rare disease? ... A recent paper concluded that: 'In practice, this implies a need for more systematic and

  • Surviving Brexit Surviving Brexit

    The product may then be sold throughout the EU. This route is compulsory for most biotechnology products and orphan medicines and is generally followed with other high technology products. ... Similarly, a sponsor of a medicine with orphan drug

  • Horizon swoops on Raptor to add rare disease products Horizon swoops on Raptor to add rare disease products

    Horizon swoops on Raptor to add rare disease products. Gains two new orphan medicines through the $800m deal. ... The deal gives Horizon two new orphan medicines - Procysbi (cysteamine bitartrate) for the treatment of the metabolic disease nephropathic

  • Orphan drugs lead CHMP recommendations Orphan drugs lead CHMP recommendations

    Orphan drugs lead CHMP recommendations. Clinuvel's Scenesse is first drug set for European approval to treat rare intolerance to sunlight. ... Scenesse (afamelanotide), developed by Australian company Clinuvel Pharmaceuticals, was one of two orphan

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Approximately 2 fully matching, plus 40 partially matching documents found.

Latest Intelligence

  • Shoring up the framework Shoring up the framework

    Consequently pharmaceutical companies that hold marketing authorisations for centrally authorised medicines will be required to take certain steps. ... Marketing authorisation holders for medicines, including orphan drugs, must be established in the EU

  • Brexit: Implications for the pharma industry Brexit: Implications for the pharma industry

    Similarly, orphan drug designations currently need to be held by an EU-based entity. ... If the UK ends up outside the EEA, it may, like other non-EEA countries, put in place reduced assessment processes for products categorised as orphan drugs by the

  • A rare talent A rare talent

    A rare talent. Shire’ s Sebastian Stachowiak on dealing with change and working for a fit-for-purpose orphan drug environment. ... The latter, says Sebastian, is “ a very good first step, in terms of recognising that there is a difference when it

  • Market access improvements in Turkey Market access improvements in Turkey

    Almost the entire pharmaceutical market is made up of prescription medicines (93.8%) with 87% reimbursed. ... While welcoming the promise of a slight acceleration of review time with high priority medicines, industry is disappointed that orphan drugs and

  • Tracking comms trends: Europe in the global vanguard on value Tracking comms trends: Europe in the global vanguard on value

    For example, last year the three Benelux countries signed a statement of intent to jointly procure orphan medicines in an effort to lower prices. ... More locally, US HMOs and insurance funds are using levels of HTA and deploying cost containment

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Approximately 1 fully matching, plus 10 partially matching documents found.

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