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- PMLiVE

EURORDIS

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EU and Australian regulators collaborate on rare diseases

EMA and Therapeutic Goods Administration to share reports for orphan drugs

- PMLiVE

Final NICE backing for MabThera in rare condition

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

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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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J&J gets EU green light for novel TB drug

Sirturo approved to treat multidrug resistant form of the disease

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NICE: Alexion must justify Soliris’ high cost

UK body unable to recommend ‘ultra-orphan’ drug

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Pharma companies back Rare Disease Day

Genzyme, Shire and more hold global campaigns today

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AZ gets limited approval for metreleptin in US

First FDA-backed treatment for rare disease lipodystrophy

Define the patient burden in rare diseases

Putting patients and carers at the heart of the value demonstration equation in orphan drugs is key

- PMLiVE

FDA backs BioMarin drug for rare enzyme disorder

Vimizim is first drug approved in US under rare paediatric disease priority review

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EMA to expand orphan drug HTA collaboration

Will further develop the concept of parallel scientific advice in 2014

- PMLiVE

Orphan drugs: A ‘responsible’ investment

There's growing sense of responsibility in the orphan drug market, but the market remains under-exploited by companies

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