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Rare Diseases and Orphan Drugs Global Forum 2017

November 1, 2017 @ 08:00 - November 2, 2017 @ 17:00 UTC+0

Event Venue:

Crowne Plaza Hotel Belgrade

Vladimira Popovica 10 Beograd 11070 Serbia

Event Description:

The Rare Diseases and Orphan Drugs Global Forum 2017 that will take place in the vibrant city of Belgrade, on the banks of the Danube River. Aimed at bringing together individuals, organisations, industry and investors from across the globe, the Rare Diseases and Orphan Drugs Global Forum 2017 will help the experts in the field to work together in order to improve treatment options for rare disease patients.

The two-day meeting will cover all the aspects of rare diseases and orphan drugs development: from epidemiology, prevalence and incidence, awareness, diagnosis, mis-diagnosis, development of novel therapies and relevant endpoints, investment options, the clinical and regulatory processes, up to discussions of reimbursement and policy. The interactive format of this event will enable attendees to find potential matches for collaboration, investmtent and/or strategic partnerships.

Learn more at https://www.rarebase.co.uk/rare-diseases-and-orphan-drugs-global-forum-2017

Mapi's Event Role:


Featured Mapi Speaker & Presentation:


  • Benoit Arnould, PhD - Senior Director, Patient-Centered Outcomes, Patient-Centered Services, Mapi Group

Presentation Date & Time:

  • Day 2 - 2nd November 2017
  • 10:15-10:45 am

Presentation Title:

  • From patient testimonials to mixed methods research: a scientific approach to place the subject at the center of the clinical development

Presentation Description:

  • The patient's perspective is at the heart of Orphan Drug development. However, researchers struggle to apply the acknowledged principles of scientifically sound outcomes research in clinical trials. As a consequence, decision makers (regulators, payors, prescribers, and ultimately patients) frequently rely on testimonials communicated by the patients, their representatives, or their caregivers, to make group-level or individual-level decisions. Due to the specific features of rare diseases, standardized measures do not appropriate solutions. Mixed Methods research combine the strength of qualitative and quantitative methods to address the research question. Embedding qualitative interviews in the Phase II/III clinical trials is a rapidly developing, promising approach to capture the patient perspective within the scientific research framework.

Register Now:



November 1, 2017 @ 08:00
November 2, 2017 @ 17:00
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