Patient-oriented research for rare diseases.

  • 2021-05-12
  • 3:00 PM
  • Virtual



Time: 3pm Eastern  (4pm Central time/5pm Mountain Time/6pm Pacific time/4pm Atlantic time/430pm NL time)

Health research can produce valuable knowledge about a particular disease and its treatment, which can help patients and their families, health care providers, and policy-makers better understand how to use, recommend, and pay for treatments. Patient-oriented research involves engaging patients, caregivers, and family members as active partners on a research team throughout the research process. This can help ensure that studies are focused on patient priorities, designed to improve aspects of health that are meaningful for patients, and conducted using approaches that align with patient preferences. A clinical trial is one type of research study where volunteer participants contribute to the generation of evidence about the safety of potential treatments and how they work.

INFORM RARE is a research network of patients, family members, health care providers, policy-makers, and researchers across Canada with an aim to improve health care for children with rare genetic diseases. In partnership with CanPKU, we plan to develop a Canadian PKU disease registry to collect health information from health care providers, patients, and families over a long-term period. The registry will serve as an opportunity to recruit patients for clinical trials to better understand treatments and to make recommendations. The first planned trial for PKU will evaluate the impact of different medical formulas on blood phenylalanine and children’s preferences.

This session will involve an introduction to patient-oriented research and clinical trials, examples of opportunities to participate in health research in the context of PKU, and experiences participating as a patient partner and participant in research. There will be perspectives from an expert on patient engagement and clinical trials (Dr. Dawn Richards), clinicians and researchers from the INFORM RARE network (Drs. Pranesh Chakraborty, Sylvia Stockler, and Beth Potter), and a patient partner investigator with the INFORM RARE network (Mr. John Adams).


TIME ZONE IS SET FOR EASTERN. Please adjust accordingly.

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This webinar is being offered in ENGLISH.

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