Real-World Data Generation and Access to Support Decision-Making About Treatments for Rare Diseases


Project Line:
Methods and Guidelines
Project Number:

Introduction and Objectives

Optimal use of real-world evidence requires a collaborative, multistakeholder approach and timely access to data that is fit for the purpose of informing health care decision-making. Integration of different types of evidence is especially important for decision-making for the care of rare diseases because of unmet need, smaller numbers of patients, and the associated challenges with conducting randomized clinical trials.  CADTH’s goals for this strategy are to:   

  • identify and increase awareness of existing sources of health data
  • enhance coordination and collaboration among different stakeholders to build stronger relationships that will help to increase and enhance access to health data
  • explore and review types of infrastructure, governance, processes, and standards that can facilitate, enhance, and expand Canada’s capacity to collect or generate real-world data (RWD) that is fit for the purpose of informing decision-making about care for rare diseases.

Rare Disease Registry Environmental Scan 

CADTH will carry out an environmental scan on rare disease registries identified through a literature review and stakeholder consultations. Using publicly available information, identified registries will be described according to the REQueST Tool developed by EUnetHTA.

Exploring Rare Disease Registry Appraisal

Representatives of selected rare disease registries will be engaged by CADTH to complete the  REQueST Tool  developed by EUnetHTA. CADTH will work collaboratively with registry representatives to get their perspectives on the value and feasibility of the use of this tool for appraisal of rare disease registries in Canada.

Registry representatives may also be engaged to provide a written overview of their registry in order to publicly share important learnings.

Collaborative Partnerships With pan-Canadian Administrative Data Holders and Data Access Facilitators

CADTH is working with key pan-Canadian organizations such as the Canadian Institute for Health Information (CIHI), Statistics Canada, and Health Data Research Network (HDRN) Canada, to explore the potential for RWD generation at a national level to support decision-making about care for rare diseases and to establish partnerships to streamline access to RWD.