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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.

Building a brighter future for rare disease patients

Building a brighter future for rare disease patients

Historically it was commercially challenging for sponsors to develop medicines to treat these so-called 'orphan' conditions. ... However, the Orphan Drug Act, passed in 1983, ushered in a new era for rare disease development.

Latest news

  • Who's calling the shots in European 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

  • Europe responding to gene therapy challenge, but picture remains fragmented 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 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 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? 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.

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Latest Intelligence

  • Biopharma and orphan drugs Biopharma and orphan drugs

    The success is clear: before the Regulation was introduced, only eight orphan-like medicines were available for patients. ... diseases. “In the area of orphan medicines, as the patient population is very small, the opportunities for the companies

  • 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

    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 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

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Latest from PMHub

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