Workshop on haemophilia registries

• Date:
  Wednesday, 1 July 2015, All day
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

Independent, disease-based registries are a potential source of valuable safety and efficacy data on products. The European Medicines Agency (EMA) is currently embarking on a strategy for patient registries. This initiative provides an appropriate starting point to consider what regulators need from haemophilia registries. The aim is to get the best capture of data in the European Union (EU) and build on existing expertise in this area. It is proposed to work with stakeholders on this topic, commencing with this EMA workshop. Places limited. Registration open until 01/06/2015.

Documents

Agenda - Workshop on haemophilia registries

Reference Number: EMA/CHMP/BPWP/190322/2015 Summary:

Independent, disease-based registries are a potential source of valuable safety and efficacy data on products. Ideally, when a significant number of patients are entered, registries are able to minimise selection bias. Disease-based registries enable data to be collected over a long period and allow comparison of products. Data on both previously treated patients (PTPs) and previously untreated patients (PUPs) can be collected, provided the initially different needs for monitoring are reflected.

Consensus points - Workshop on haemophilia registries

Reference Number: EMA/CHMP/BPWP/380896/2015

Presentation - Key points from introductory session: Setting the scene (Karri Penttila)

Presentation - Key points from session 2: registries (Christine Keipert)

Presentation - Key points from session 3: industry perspective (Karri Penttila)

Presentation - Safety of new products: do we have enough tools in place? (Flora Peyvandi)

Presentation - Focus on immunogenicity of FVIII and FIX products and general reflections on registries (H. Marijke van den Berg)

Summary:

Workshop on haemophilia registries

Presentation - Factor VIII and factor IX development plans at the Paediatric Committee: overview (Thorsten Olski)

Presentation - The UK national haemophilia database: a thumbnail sketch!(Charles RM Hay)

Presentation - Haemophilia registries quantity versus quality: the current situation in Europe (Christine Keipert)

Presentation - European haemophilia safety surveillance (EUHASS) (Mike Makris)

Presentation - Community counts! CDC Public Health Surveillance for Bleeding Disorders (Mike Soucie)

Presentation - PedNet registry (Rolf Ljung)

Presentation - FranceCoag network (Roseline d'Oiron)

Presentation - IPFA’s perspective on haemophilia registries usage (Franà§oise Rossi)

Presentation - Medical and Regulatory Policy Plasma Protein Therapeutics Association: Industry perspectives (Mary Gustafson)

Presentation - ISTH and EAHAD perspective on haemophilia registries (Flora Peyvandi)

Presentation - World Federation of Hemophilia (Mark Brooker)

Presentation - Haemophilia registries: patients’ perspective - European Haemophilia Consortium (EHC) (Radoslaw Kaczmarek)