Orphan Drugs & Rare Diseases: Life Sciences and Drug Discovery

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. This conference 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.

Pharmaceutical companies, biotechnology companies, academic research units and other stakeholders organisations are increasingly merging their research, resources and expertise in the critical process of discovering new biopharmaceutical therapies to improve the lives of rare disease sufferers. Orphan Drugs & Rare Diseases will provide an exclusive opportunity to network with these leading experts on not only rare diseases research but ultra-rare diseases and the medicinal solutions currently being invented to treat them.