The first workstream seeks to untangle the complexities of registries, to find common ground, and to co-construct a collectively endorsed forward direction. It focuses on challenges such as data sharing, multiplicity of registries, ownership, and the overall purpose of registries.

About the workstream

Registries constitute key instruments to increase knowledge on bleeding disorders for patients, healthcare professionals, payers, insurance companies, researchers, and policy makers. Registries can include epidemiology of the disorder, demonstrate efficacy of different management and therapeutic options, and be a significant enabler for clinical research. Some of the central issues with national and pan-national registries to address include principles for data collection, funding sustainability, ownership and access to data, lack of interconnectivity, and real-world evidence integration.

This workstream seeks to better untangle the complexities of this area, to find common ground, and to co-construct a collectively endorsed forward direction.

Composition

The composition of the workstream aims to reflect the diversity of key actors who have hands-on knowledge of challenges and possible solutions in this thematic area. This includes, but is not limited to: patients, healthcare professionals, industry partners, private companies, policymakers, researchers, and academics.

The general membership profile is built around required skill sets and may evolve throughout the co-creation process. The initial skill sets include the ability to ‘problem map’ salient points and their relationships, requiring: 

  • Hands-on knowledge of the topic area,
  • Genuine willingness to learn about, and actively contribute to, a systems change culture,
  • Experienced, analytical, open, hands-on ability to remove stakeholder ‘hats’, and
  • Respect of Chatham House Rule and culture of conduct and engagement.

Each workstream brings together 16-20 individuals, which is broad enough to ensure a comprehensive overview, while small enough to be workable.

Workload

The mandate duration and meeting cadence are due to change based on collective needs and decisions. However, the preliminary anticipated commitment is three to four (3-4) hours per meeting, once a semester, trimester or quarterly, subject to the above collective decision.

The workstream met for the first time in Q4 2021.

Public outcome

As part of sharing the outcome of the work of the EHC Think Tank, the first paper from the workstream on registries in which workstream members identify key challenges concerning registries is now published.

This paper is the first in a series of outcomes aiming to synthesise how we might change registries.

Bok A, Noone D, Skouw-Rasmussen N, EHC Think Tank. Key challenges for patient registries – A report from the 1st workshop of the EHC Think Tank Workstream on Registries. The Journal of Haemophilia Practice. 2022;9(1): 14-19. https://doi.org/10.2478/jhp-2022-0002

Real World Data (RWD) have become an important part of both the development and the evaluation of new and novel therapies. For rare diseases, the challenge is to identify a sufficient number of patients when performing analyses using multiple data sources, and to set up a governance structure for such analyses.

This report presents the results of the mapping of registries for rare bleeding disorders, more specifically, what registries and data are currently available as a first step towards defining such a governance structure. In this report, opportunities for development have also been identified.

Registry Mapping – An overview of rare bleeding disorder registries across Europe by Copenhagen Economics

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