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- PMLiVE

Lumena Pharmaceuticals

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

Shire to file Vyvanse for binge-eating in Q3

Wants to mirror drug’s success as ADHD treatment

- PMLiVE

Shire’s ADHD drug fails in depression trial

Company drops Vyvanse for use in major depressive disorder

- 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

Shire elects new chair

Susan Kilsby to replace Matthew Emmens who retires

- PMLiVE

Infographic: Orphan drug market

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

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