The evolving landscape of pre-approval access to orphan drugs
Journal of Genetic Syndromes & Gene Therapy

Journal of Genetic Syndromes & Gene Therapy
Open Access

ISSN: ISSN: 2157-7412

The evolving landscape of pre-approval access to orphan drugs

8th World Congress on Rare Diseases and Orphan Drugs & Clinical Trials & Regulatory Affairs

November 21-22, 2018 | Paris, France

Stuart Bell

VP Consulting, UK

Scientific Tracks Abstracts: J Genet Syndr Gene Ther

Abstract :

This presentation will cover the evolution over the last 10 years of pre-approval access (expanded access/early access/compassionate use/named patient supply) to drugs, with an emphasis on orphan drugs and rare diseases. ? The presentation will include; ? How has the landscape changed over the last 10 years? ? What are the driving forces behind these changes? ? How have the regulations changed in recent years, and what has the impact been? ? What are the commercial opportunities provided by pre-approval access to orphan drugs? ? What do the next 5-10 years hold for pre-approval access to orphan drugs?

Biography :

Stuart has more than 20 years of healthcare and pharma consulting experience, with a particular focus in unlicensed medicines and pre-approval access. He is one of the senior management team responsible for establishing Inceptua Medicines Access and is responsible for Inceptua’s consulting engagements, covering strategy and policy, real-world evidence, communications and market access. Prior to Inceptua, Stuart set up the Consulting Dept. at Idis/Clinigen, pioneering the development of global corporate strategies on preapproval access and developing the first pre-approval-specific EDC for real-world data collection. He has formerly held roles as: Principal, Real-World Evidence at IQVIA, Director, Informatics Initiative, UK Dept. of Health, Consultant to the European Association of Neuro-Oncology and Director of Communications for the European Cancer Organization.

Email id: [email protected]