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- PMLiVE

Spectrum Pharmaceuticals

- 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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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

- PMLiVE

Pharma companies back Rare Disease Day

Genzyme, Shire and more hold global campaigns today

- PMLiVE

AZ gets limited approval for metreleptin in US

First FDA-backed treatment for rare disease lipodystrophy

The distressing impact of rare diseases

Much more needs to be done to address the problem of effective communications with stakeholders and adequate information for patients affected

- PMLiVE

FDA approves Chelsea’s rare blood pressure drug

US biopharma company gets green light in US for Northera

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

- PMLiVE

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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