When we think about research, what are two possible things we can compare in TANGO2?

Comparative Study: is a type of study that compares different treatments to see which works best for a specific health problem. It helps doctors, patients, and caregivers make better decisions by showing which treatment is most effective and safest. CER (Comparative Effective Research) looks at real-world outcomes and can include medications, therapies, surgeries, or other interventions. This research is important because it provides evidence on what works best in everyday healthcare settings, helping improve overall patient care and health outcomes.
This is a type of research that the TANGO2 community might one day be a part of.
What questions do you have about CER?

@jmeisner @Ryan_Carrier @seemal @linaghaloul @samuel_mackenzie @HdeCalbiac @Cmiyake @Farah.Ladha @cosmo @EmelYilmazGumus

How will this work when we already have such a range of intensity related to the impact of Tango2?

I am admittedly still confused about how the rare disease community can get involved in CER initiatives (e.g., grants focused on this topic) when there are no treatments or only one treatment available for a condition. As you said, it makes sense to think about as a forward-facing initiative, but I’m not sure how we can or should pursue this now. Would love to hear from PCORI on this topic.

Great comment. PCORI has funded several rare disease CER projects with comparator’s ranging from treatments, decision aids, & programs, to in-patient vs outpatient care.

Check out this list of PCORI funded rare disease CER projects. Be sure to review these project’s professional abstracts to see PICOTS that highlight various comparators.

Are there any comparators we should be thinking about for TDD?
@samuel_mackenzie @Majoarredondo @jmeisner @Cmiyake @seemal @linaghaloul @HdeCalbiac @Ryan_Carrier @cosmo @nicolasabreumd

What about comparing giving b5 supplements in one large daily dose verse multiple doses spread out over the course of the day? Do kids do better one way compared to the other?

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