What are outcomes?
- Outcomes are used to determine if a treatment is effective or not.
- Outcomes may include how someone feels, what they can do, observable symptoms, or results of lab tests.
What is a Core Outcome Set (COS) and why is a COS used?
- A ‘Core Outcome Set’ is a small set of outcomes that are established as important and should be collected in every research study of the same disease.
- An established COS prevents studies from reporting only a selection of the outcomes that were measured. It is expected that they report on all outcomes in the COS.
Why is a COS important?
- If researchers measure different outcomes, it is hard to compare and contrast the effectiveness of interventions or treatments.
- A COS will help us compare CTD and GAMT deficiency studies to identify which treatments work best.
How can I contribute?
In multiple rounds of an online survey, we will show you a list of outcomes related to CTD and GAMT deficiency and ask you to rate how important each outcome is to you. The survey will be available in English, French, and Spanish. As a rare disease community, we need as many patients, caregivers, and clinicians as possible to participate!
Who is eligible?
- Adult patients (18 years or older) diagnosed with CTD or GAMT deficiency
- A parent or caregiver of a person diagnosed with CTD or GAMT deficiency
- A health care professional experienced in caring for patients with CTD or GAMT deficiency (e.g., geneticist, dietician, physical therapist, speech therapist, psychologist, neurologist, etc.)
What is the time commitment?
- You will be asked to complete 2-3 surveys within the next 3-5 months.
- Each survey will take about 1 hour to complete.
- After each survey you complete, you will be entered into a drawing to win:
- One of five Amazon gift cards valued at $50 USD for the first survey round
- One of five Amazon gift cards valued at $75 USD for the second survey round
- If a third survey round is required, additional Amazon gift cards will be awarded