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- PMLiVE

Raptor Pharmaceutical

Small diseases need big picture communications

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

Orphan indication? No easy access

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

- 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

US approval for Novo’s Factor XIII drug Tretten

Becomes first recombinant treatment licensed for a rare clotting disorder

- PMLiVE

Janssen’s Sirturo wins backing for conditional EU licence

CHMP recommends first-in-class multidrug-resistant tuberculosis treatment

- 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

EMA to review safety of Ariad’s Iclusig

Product information will be updated to warn of blood clot risk

- 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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