Workshop on demonstrating significant benefit of orphan medicines: concepts, methodology, and impact on access

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TitleWorkshop on demonstrating significant benefit of orphan medicines: concepts, methodology, and impact on access
Date07/12/2015 - 07/12/2015
LocationEuropean Medicines Agency, London, UK
SummaryThis workshop aims to help clarify the concept and demonstrate the significant benefit of new orphan medicines over existing treatments. It will discuss existing methodologies for comparative efficacy and effectiveness and for major contribution to patient care, including patient preferences, and how these could be applied in demonstrating significant benefit at marketing authorisation as well as the impact of significant benefit on health-technology-assessment (HTA) evaluation, pricing decisions and patient access. The workshop is targeted at medicine developers, regulators, healthcare professionals, academia, patients, HTA bodies and healthcare payers.

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Contact point:

For administrative matters:
monica.gomar@ema.europa.eu

For content of the worskhop:
laura.fregonese@ema.europa.eu