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- PMLiVE

orphen drugs

Orphan indication? No easy access

Health technology assessment for orphan drugs continues to present challenges for pharmaceutical companies

Sanofi reception

Sanofi strikes $700m rare diseases deal

Company's Genzyme division will obtain significant rights to Alnylam’s pipeline of orphan drugs

- PMLiVE

EMA: more fee reductions for orphan drugs

Greater incentives introduced for larger companies

The logistics of orphan drugs

Managing a rare disease global access programme, the goal is to minimise risk and get the drug to the patient on time

- PMLiVE

Rare disease strategy for the UK launched

Aims to improve understanding and boost research

Shire Basingstoke

Shire snaps up Viropharma to build $2bn rare disease unit

Gains access to Cinryze in expensive but 'strategically sound' deal

Shire Basingstoke

Shire mulls UK job cuts

Enters “employee consultation process” for research site in Basingstoke

- PMLiVE

Orphan status for Stem Cell’s leukaemia drug

Tigecycline wins FDA rare disease designation

- PMLiVE

FDA sets up rare disease research fund

Will invest $14m across 15 orphan drug projects

Orphan drugs: the early access regulatory environment

Despite considerable variation to the rules for unlicensed orphan drugs, there are some clear first steps for a global access programme

- PMLiVE

NICE backs Roche’s MabThera in rare autoimmune disease

Overturns previous negative draft guidance from England's cost-effectiveness body

- PMLiVE

Intermune supports awareness efforts for IPF

Last week's IPF World Week saw the global patient community unite

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