Held during the HTAi 2023 Annual Meeting in Adelaide, Australia, the panel session titled “Real World Evidence from Rare Disorders’ Registries: A Problem or Part of the Solution to Inform Decisions?” aimed to shed light on the critical role of real-world evidence (RWE) derived from registries focused on rare disorders.
Registries should ideally be owned by the disease community, subject to strict governance mechanisms, but taking account of the needs of all stakeholders, such as HTA bodies and Payers. For HTA, registries can inform understanding of disease prevalence, burden of illness, the patient journey, progression of disease and for longer term data collection in local clinical practice in an Outcomes-Based Managed Entry Agreement. For rare disorders, registries should be aligned across healthcare jurisdictions and whilst ensuring strict data governance, patient access to their own data and permissions for use in research must be ensured. HTA bodies are developing processes to evaluate the quality of registries to understand if they provide valid, reliable and relevant data for the specific context of an HTA question. This requires transparency about the registry construct, governance, data curation and methods for RWE generation.
The report of the HTAi 2023 Panel on RWE from Rare Disorders Registries is available to view below, or via pdf here (file size 1 MB).