Patient Engagement
Patient engagement is embedded in all aspects of the INFORM RARE network. Patients, family members, and patient organization representatives are involved as co-principal investigators, co-investigators, and advisors.
Our Patient Engagement Strategy
As a patient-oriented pediatric research network, a core value of INFORM RARE is that patient partnership is essential to ensure that our research focuses on the priorities of rare disease patients and their families, both in the questions we pursue and the approaches we use. Twenty-six parents, representatives of patient groups, and youth patients and family member partners are involved as co-principal investigators, co-investigators, and advisors in co-designing INFORM RARE research (see below). They are actively involved in the selection and refinement of research questions and priorities, planning and conduct of clinical trials, and dissemination of results.
Patient partner co-investigators were invited to join projects within all INFORM RARE research themes (see Home for a full list) to ensure that patient perspectives are embedded throughout all of our research. Each research theme has at least one patient partner co-investigator.
Guiding Principles
These are the guiding principles to which all INFORM RARE investigators and collaborators will adhere in pursuing the goal of integrating patient engagement into our research:
For a detailed explanation of the guiding principles and the process to develop them, click here.
Showcasing Our Patient Engagement Work
NOV 2023: Oral presentation at the Canadian Organization for Rare Disorders Fall Conference.
Smith M. A whirlwind tour of the wild, wacky, wonderful world of patient partnered research. Presented at the Canadian Organization for Rare Disorders Fall Conference 2023, Calgary, AB, November 29-30, 2023.
APRIL 2023: PPEC Spring Webinar Series: Supporting your Engagement Work through Evaluation (INFORM RARE used as an example)
Webinar recording available here.
DECEMBER 2022: Smith M, Potter B, Vanderhout S, Howie A. Patient partnership in a pediatric rare disease research network: Mutual learning for meaningful research. In: Reszel J, McCutcheon C, Kothari A, Graham ID, editors. How We Work Together: The Integrated Knowledge Translation Casebook. Volume 6. Ottawa, ON: Integrated Knowledge Translation Research Network. 2022; 16-19.
This paper provides an overview of INFORM RARE’s patient engagement strategy. It explains how we initiated and currently manage our partnerships, the impact of the partnerships, and lessons learned.
MAY 2022: Poster presentation at the 2022 Canadian Association for Health Services and Policy Research (CAHSPR) Conference
Smith M, Howie A, Vanderhout S, Malandrino C, Potter BK. Youth and family engagement in a pediatric rare disease research network. Presented at the Canadian Association for Health Services and Policy Research (CAHSPR) Conference (virtual), May 31, 2022.
MAY 2022: Poster presentation at the 2022 Garrod Symposium
Vanderhout S, Howie AH, Malandrino C, Smith M, and Potter BK. Youth and family engagement in clinical trials about phenylketonuria, spinal muscular atrophy, and mucopolysaccharidoses. Presented at the Garrod Symposium, Calgary, AB, May 12-14, 2022.
SEPTEMBER 2021: Vanderhout SM, Smith M, Pallone N, et al. Patient and family engagement in the development of core outcome sets for two rare chronic diseases in children
This article describes the contribution of patient partner co-investigators and family advisors to the design, conduct, and completion of core outcome sets (COSs) for medium-chain acyl-CoA dehydrogenase deficiency and phenylketonuria.
Our approach was feasible and considered valuable by all study team members, including patients and family members, in improving the relevance of the deliverable to patients. The methods outlined herein can be applied to other pediatric disease contexts, allowing patient and family perspectives to influence the direction of future studies to develop COSs.
Images by Freepik