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- PMLiVE

genetic diseases

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

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

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