This toolbox aims to prepare families, healthcare professionals, researchers and other stakeholders to engage in patient-centered and/or comparative clinical effective research as one collective research team, ensuring research priority is given to outcomes of interest for the TANGO2 Deficiency Disorder (TDD) community.
For those who are new to TDD and just getting started on their rare disease journey, please begin by exploring the following website: https://tango2research.org/about-tango2/tango2-deficiency-disorder/
PCOR: Patient-Centered Outcomes Research
Like the Patient-Centered Outcomes Research Institute (PCORI), a patient-centered approach to research is at the heart of the TANGO2 Research Foundation’s mission. We, too, share an unwavering commitment to outcomes that resonate with patients and their families. Our dedication extends to ensuring active participation from patients, families, researchers, and various healthcare and industry stakeholders at every stage of the research process. This collaborative approach involves collectively prioritizing research topics, contributing to study design/implementation, and collaboratively disseminating research results.
Patient-Centered Outcomes Research (PCOR) - PCOR is a type of research that focuses on helping patients and their caregivers make informed healthcare decisions.
Comparative Clinical Effectiveness Research (CER) - CER involves the comparison of different approaches and interventions to determine which ones work best for which patients under specific circumstances.
While the resources in this toolbox are specific to TANGO2 Deficiency Disorder (TDD) research, having a solid foundation in understanding PCOR/CER methodology is vital to preparing you to successfully contribute to research. We encourage all those preparing to engage in PCOR to take some time and explore the available training modules published by PCORI to learn even more.
PCOR in Rare Disease Communities
To download the toolbox as a pdf, click here.
INDEX
Understanding TDD
- TDD Variants
- Families Affected
Engaging in TDD PCOR/CER Research
- Building a Team
- Research Fundamentals
- Stakeholder Engagement
- Research Question Development
- Specimens and Models
Glossary of Terms
TDD Publications
References
This project was funded through a Patient-Centered Outcomes Research Institute® (PCORI®) Eugene Washington PCORI Engagement Award (EASO 30416).
The statements presented in this toolbox are solely the responsibility of TANGO2 Research Foundation and do not necessarily represent the views of PCORI®.