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