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Communiqué 53 Cover Image
- PMLiVE

orphen drugs

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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

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

- PMLiVE

Supportive strategies for demonstrating value

Improved planning will support better data generation and improve understanding of rare diseases and the orphan drugs that treat them

- PMLiVE

FDA approves sleep disorder drug for blind people

Vanda Pharmaceuticals wins backing for Hetlioz to improve a person’s body clock

The thing about small numbers

Marketers working in orphan drugs need to take a narrower, deeper approach to relationship building, and look to medical centres of excellence

Small diseases need big picture communications

An early commitment to communications planning in rare diseases is worth the risk

- PMLiVE

Infographic: Orphan drug market

A visual guide to the orphan drug and rare disease market for pharma

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