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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.

Who's calling the shots in European healthcare?

This has been driven by the sector’s shift towards developing more and more orphan medicines – these are often groundbreaking advances for patients, but also carry very high price tags which ... Natsis believes this is a pointer to the future

Latest news

Europe responding to gene therapy challenge, but picture remains fragmented

Among its provisions are plans for compulsory patient registries for all orphan products and medicines approved via conditional approval or exceptional circumstances. ... However Mr Iotti indicated that the administration of this was more complicated
NICE and Canadian counterpart to offer joint advice service

Just last month NICE, CADTH and The US-based Institute for Clinical and Economic Review (ICER) announced they will team up on a medicines cost evaluation project. ... orphan medicines.
EMA sees approval numbers rise in 2018

From these, 21 were orphan medicines (exactly 50%) and three were advanced therapy medicinal products – Novartis’ Kymriah and Gilead/Kite’s Yescarta CAR-T therapies, plus Spark’s Luxturna, co-marketed ... Lastly, the EMA’s key human medicines
Beneluxa: the future of European market access?

However there is one specialist area in which there is unquestionably a growing appetite for European greater collaboration: health technology assessment of innovative medicines. ... and has so far focused on appraising high cost orphan medicines.
Orphan drugs from Biogen and Amicus win UK Prix Galien

There was a much warmer welcome at the awards, hosted by ValueBase, for the pharma and biotech companies with products on the shortlist, which was dominated by orphan medicines. ... All bar one of the finalists were medicines for orphan conditions, and

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Latest Intelligence

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

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 comes to
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

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