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

Date: 07/12/2015
Location: European Medicines Agency, London, UK

This 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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