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

• Date:
  Monday, 7 December 2015, All day
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

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.

Documents

Workshop report - Demonstrating significant benefit of orphan medicines: concepts, methodology and impact on access

Reference Number: EMA/6690/2016

Agenda - Demonstrating significant benefit of orphan medicines: concepts, methodology, and impact on access

Reference Number: EMA/588712/2015 Rev. 2

Presentation - Significant benefit: origins and experience up to date (Kerstin Westermark)

Presentation - Clinically relevant advantage and major contribution to patient care (Laura Fregonese)

Presentation - Significant benefit of oncology products at MA: the COMP experience (Frauke Naumann-Winter)

Presentation - HTA/payers’ perspective on significant benefit (Niklas Hedberg)

Presentation - ESMO magnitude of clinical benefit scale (ESMO-MCBS) for new anticancer therapies (Richard Sullivan)

Presentation - Significant benefit of orphan medicinal products: industry views (Adam Heathfield)

Presentation - The patient’s perspectives on significant benefit (Yann Le Cam)

Multimedia