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.
-
List item
Workshop report - Demonstrating significant benefit of orphan medicines: concepts, methodology and impact on access (PDF/157.79 KB)
First published: 25/07/2016
Last updated: 25/07/2016
EMA/6690/2016 -
List item
Agenda - Demonstrating significant benefit of orphan medicines: concepts, methodology, and impact on access (PDF/94.65 KB)
First published: 04/09/2015
Last updated: 04/12/2015
EMA/588712/2015 Rev. 2 -
List item
Presentation - Significant benefit: origins and experience up to date (Kerstin Westermark) (PDF/579.83 KB)
First published: 15/12/2015
Last updated: 15/12/2015 -
List item
Presentation - Clinically relevant advantage and major contribution to patient care (Laura Fregonese) (PDF/479.39 KB)
First published: 15/12/2015
Last updated: 15/12/2015 -
List item
Presentation - Significant benefit of oncology products at MA: the COMP experience (Frauke Naumann-Winter) (PDF/422.39 KB)
First published: 15/12/2015
Last updated: 15/12/2015 -
List item
Presentation - HTA/payers’ perspective on significant benefit (Niklas Hedberg) (PDF/696.35 KB)
First published: 15/12/2015
Last updated: 15/12/2015 -
List item
Presentation - ESMO magnitude of clinical benefit scale (ESMO-MCBS) for new anticancer therapies (Richard Sullivan) (PDF/625.51 KB)
First published: 15/12/2015
Last updated: 15/12/2015 -
List item
Presentation - Significant benefit of orphan medicinal products: industry views (Adam Heathfield) (PDF/1.19 MB)
First published: 15/12/2015
Last updated: 15/12/2015 -
List item
Presentation - The patient’s perspectives on significant benefit (Yann Le Cam) (PDF/825 KB)
First published: 15/12/2015
Last updated: 15/12/2015