Agenda released for 8th annual Orphan Drugs and Rare Diseases

Patients with rare diseases cannot continue to be overlooked because of costly therapies and have the same rights to treatment as any other patient. At this year’s 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 disease treatment field.


Brief overview of the topics on offer:


17 October (Day 1 – Main Conference) is chaired by Carina Schey, Researcher at University of Groningen who will also deliver a presentation on “Primary data collection on the weighting preferences of a wide range of people for criteria used in multi-criteria decision analysis (MCDA)”; Yolanda Barbachano, Senior Statistical Assessor at MHRA will be focusing her talk on Benefit-Risk assessment in rare diseases; while Nigel Nicholls’s – Director and Country Manager UK/Ireland at BioMarin Europe – case studies will show participants manufacturer’s perspective of the HST process and NICE-lessons


18 October (Day 2 – Main Conference) will see Patrick Mollon, Director Health-Economics, Outcomes Research & Epidemiology at Shire analysing challenges and future strategies of market access in rare diseases; AKU Society’s CEO and Board Chair – Nicolas Sireau will deliver a Keynote Address on patient involvement in orphan drug clinical development; Xavier Ortega, Project Manager, Rare Diseases at Minoryx presenting a Case Study on targeting a peroxisomal disorder (X-ALD)


Gaining more from the event – on 19 October an Interactive Workshop run by JG Zebra Consulting will be looking into question: “Working together for HTA in rare diseases – a step too far or the way forward?”


Orphan Drugs and Rare Diseases conference this year is set to inspire the international rare diseases community by exploring synergies between regulators, health and technology assessors


Further details at


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