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Orphan Drugs

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FDA approves Janssen drug for rare Castleman’s disease

Sylvant approved to treat lymphoma-like condition

National Institute for Health and Care Excellence NICE logo

NICE gives partial backing to Ferring’s prostate cancer drug

Firmagon on course for use on NHS in England and Wales

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BioAlliance and Topotarget merge to focus on rare cancers

French and Danish firms combine to develop orphan oncology drugs

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EU and Australian regulators collaborate on rare diseases

EMA and Therapeutic Goods Administration to share reports for orphan drugs

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Europe votes for greater clinical trial transparency

European Parliament passes new EU Clinical Trials Regulation

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Interview: Georg Toufar, Mundipharma

Mundipharma’s chief marketing officer Georg Toufar on the benefits of having a smaller, more concentrated focus

National Institute for Health and Care Excellence NICE logo

NICE consults on value-based assessment changes

Aims to create a more flexible approach to drug funding decisions for NHS in England and Wales

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Final NICE backing for MabThera in rare condition

Roche drug available in England and Wales to treat rare form of vasculitis

Lilly launches online training to boost patient voice in HTA

New tool aims to help patient advocates 'better influence decisions'

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Ireland and the impact of reference pricing

Industry remains tight-lipped on the country's pharmaceutical pricing changes

- PMLiVE

No price ceiling for rare disease drugs, say MPs

BIA survey comes ahead of consultation on evaluation framework for ultra-orphans

NICE and ‘ultra orphans’

NICE and its processes for ‘highly specialised therapies’

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