Orphan Drugs and Rare Diseases Conference

17 – 18 October 2018

Holiday Inn Kensington Forum, London, UK



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


Also, in the programme – Post-conference workshop (19th October) overviewing end to end innovation: Successful patient engagement in the generation of innovative therapies for rare diseases that meet genuine unmet medical needs.


View detailed agenda, find out who is speaking and book to attend at http://www.orphandrugs.co.uk/jcsWL


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