Designing trial outcomes that matter
Page last reviewed: 23-Oct-2026
Authors: Refer to contributors below
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About
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Launched in September 2025 within RareKids-CAN, Rare Disease Clinical Trial Outcome Assessment and Design (ROAD) project aims to strengthen the capacity of trialists and patient and family partners to work together in selecting meaningful, feasible outcomes for pediatric rare disease clinical trials.
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The project began with a review of existing research (a scoping review) and a survey of people who design and run clinical trials (an international needs survey). These studies identified gaps in how trial outcomes are currently selected.
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To address these gaps, the team developed two practical templates, one for patient and family partners (ROAD-PF) and one for trialist (ROAD-TT), which are brought together in this toolkit.
Two templates that work together
These templates are designed to be used together when designing a trial
ROAD-PF
Informed by lived experience of patients and families
ROAD TT
Informed by clinical trial design and regulatory guidance
Co-design with patient and family partners
Twelve patient and family partners from rare disease communities in Canada shaped the toolkit through two virtual workshops and a French-language consultation (February to April 2026).
Their lived experience helped ensure the toolkit reflects what matters to people and families affected by a rare disease.
Co-design with international trialists
Seventeen rare disease experts formed the international advisor board and two meetings were held (February to April 2026).
Supporting useful trials
Patient, families, and trialists come together at the trial co-design meeting(s) to bring these perspectives together. The templates help trialists document what outcomes they choose, why they chose them, and who helped make those decisions. This creates a clear record that can be shared with trialists, funders, ethics boards, regulators, and clinical trial registries.
Learn more
Contributors
| Name | Affiliation | Role |
| Veronica Ka Wai Lai | Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, Canada | Researcher; core project team; co-facilitator |
| Maureen Smith | Patient Partner, Ottawa, Canada | Patient partner; co-facilitator; French-language consultation |
| Sara Pot | CanChild Centre for Childhood Disability Research, Hamilton, Canada | Family partner; co-facilitator |
| Alicia Hilderley | Hotchkiss Brain Institute, Cumming School of Medicine, University of Calgary, Canada | Partner; co-facilitator |
| Leah Sarah Peer | Patient Partner, Peer Medical Foundation, Montreal, Canada | Partner; investigation and review |
| Roberta Webster | Patient Partner, Mississauga, Canada | Partner; investigation and review |
| Amanda Doherty-Kirby | Patient Partner, Cornwall, Prince Edward Island, Canada | Partner; investigation and review |
| Cal Robinson | Department of Paediatrics, Division of Nephrology, The Hospital for Sick Children, Toronto, Canada | Investigation, validation, review |
| Thierry Lacaze-Masmonteil | Maternal Infant Child & Youth Research Network (MICYRN), Vancouver, Canada | Investigation, validation, review |
| Ami Baba | Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, Canada | Core project team |
| Martin Offringa | Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, Canada | Core project team; corresponding author |
Have questions?
Contact us with your questions about the ROAD toolkit.
