Zahra Nasseri Moghaddam: “Establishing a Core Outcome Set (COS) for CTD & GAMT deficiency through caregiver and health professional collaboration”
SHORT SUMMARY
This poster provides an overview of the Core Outcome Set for GAMT & CTD- its purpose, how the COS was developed, and the final 8 outcomes included.
ABSTRACT
Clinical trials aim to determine the safety and effectiveness of interventions by evaluating their impact on diverse endpoints. However, inconsistencies in defining and measuring these endpoints have posed challenges in applying and comparing trial results. A list of important outcomes, called a core outcome set (COS), is developed to identify a small set of disease-specific outcomes deemed important by stakeholders, and should be reported in every research study and clinical trial. Unfortunately, patient and caregiver perspectives have historically been overlooked in the COS development process, thus limiting their input into the outcome selection. ACD collaborated with caregivers and health professionals to develop a COS of eight outcomes for creatine transporter deficiency (CTD) and guanidinoacetate methyltransferase (GAMT) deficiency. Caregivers were partners throughout the COS development process, increasing community engagement and facilitating caregiver empowerment. Here, we provide an overview of our entire COS development project. Our multifaceted approach included (1) conducting evidence reviews (i.e., focus groups, literature reviews, patient registry data), (2) developing three Delphi surveys for input from the CCDS community at large, and (3) hosting a consensus workshop with caregivers and health professionals to finalize the COS. We expect that this project will (1) ensure a patient centered approach for accelerating drug development, (2) minimize bias, and (3) promote a more efficient use of resources.