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

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

Key benefits of attending:

• Learn about health technology assessment protocols and market access in the EU and globally
• Network with key regulators of orphan medicines and policy makers for national rare disease treatment
• Discover successful funding, financing and economic models for orphan drug development and translational research
• Analyse industry case studies showing how R&D and market access costs can be reduced
• Understand the latest developments in ultra-rare diseases studies and new medicines for them
• Explore how organisations are merging research, resources and expertise in the race to cure rare diseases

Hear keynote addresses from:
• Dr. Edmund Jessop, Medical Advisor, National Commissioning Group for Highly Specialised Services, NHS
• Josie Godfrey, Head of Policy and Coordination, AGNSS

• Stephen Nutt, Executive Officer, Rare Disease UK

• Carlos R. Camozzi, MD, PhD, MBA, Vice President, Chief Medical Officer, uniQure B.V.

• Marisa Jaconi, Vice-Director and Founder, Swiss Institute of Cell Therapies (SICT)

• Samantha Parker, Director of External Affairs and Rare Disease Partnerships, Orphan Europe