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Event Details


Orphan Drugs and Rare Diseases Conference

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This year's SMi's Orphan Drugs and Rare Diseases Conference will bring together regulatory agencies, pharmaceutical companies, non-profit organisations and orphan drugs developers to construct an agenda that addresses the driving economic factors, policies and issues that will affect the development of orphan drugs globally including commercialisation, policies, reimbursement, and pricing. 

Keep up to date with the industry and to learn from expert speaker panel, bringing you important new case studies and reports on this year’s relevant topics.

 Discuss the pricing and reimbursement of orphan drugs
 Understand the challenges of patient recruitment and patient-centric research
 Hear the MHRA's perspective on benefit-risk assessment in rare diseases
 Explore the clinical development of orphan drugs for rare disease
 Gain insight into potential synergies between regulators and assessors

 Carina Schey, Researcher, University of Groningen
 Sheela Upadhyaya, Associate Director, Highly Specialised Technologies, National Institute for Health and Care Excellence (NICE)
 Xavier Ortega, Project Manager, Rare Diseases, Minoryx
 Nigel Nicholls, Director and Country Manager UK/Ireland, BioMarin Europe Ltd
 Yolanda Barbachano, Senior Statistical Assessor, MHRA
 Michale Bouskila-Chubb, Head of Business Development, Healx


Luda Durneva

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