Orphan Drugs & Rare Diseases

The call to treat rare diseases has become louder around the world in recent years than ever before. Supportive organisations including patient groups, national health authorities, health trusts, academia, HTAs, pharmaceutical and biotechnical companies are now progressively growing in number and sophistication in their approaches to medicinal care for those with rare and ultra-rare diseases.

Orphan Durgs and Rare Diseases will present briefings from key opinion leaders, those with hands-on experience of regulating new drug discoveries, companies who have already developed advanced orphan drugs & successfully been granted orphan medicine status and selected experts in the field.

Speakers will offer unique insights into cutting-edge international developments taking place in: expanding the reach of medicine to previously untreatable and unreachable patients with rare diseases; different regulatory and policy environments; new drug discoveries; innovative business strategies & funding & financing models, and the importance of partnerships with patient groups and those at the point-of-care. View the Program


Why should you attend this event?

Learn about ultra-rare diseases and the very latest orphan drug developments

Network with regulators and policy makers of orphan medicines

Discover how orphan drugs can be made more accessible to patients

Analyse how R&D and market access costs can be reduced

Discuss successful funding, financial and economic models for orphan drug development and translational research

Consider the ethical and social factors versus drugs-to-make money

Register now

Hotel Accomodations and Event Contact:

Phone: +44 (0) 870 9090 713

Fax: +44 (0) 207 827 6001

E-mail: [email protected]