Orphan Drugs & Rare Diseases 2018 Conference

SMi Group
17/10/2018 - 18/10/2018

Patients with rare diseases cannot continue to be overlooked because of costly therapies and are entitled to the same rights to treatment as any other patient. At this year’s 8th annual Orphan Drugs and Rare Diseases conference, organised by SMi Group and taking place on 17 – 18 October in London, industry experts will address the opportunities and challenges within the rare diseases treatment field.


Keep up to date with the industry and learn from an 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 diseases
  • Gain insight into potential synergies between regulators and assessors




  • Carina Schey, Researcher, University of Groningen
  • 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


Attend the exclusive Interactive Workshop (16 October 2018) run by JG Zebra Consulting, who will be looking into the topic of: “Working together for HTA in rare diseases – a step too far or the way forward?”


This year’s highly anticipated Orphan Drugs and Rare Diseases conference is set to inspire the international rare diseases community by exploring synergies between regulators, health and technology assessors.


Visit the website for more details http://www.orphandrugs.co.uk/pharmaphorumwl


Twitter – @SMIpharm & #smiorphandrugs

LinkedIn – SMi Pharma



+44 (0)20 7827 6000