Project Summary

Background: Rare diseases are often poorly understood, and patients and clinicians struggle to find evidence-based therapies because of lack of research. Patients often turn to online support groups to navigate the health system. Patient advocacy groups typically become experts in their condition and begin calling for and leading research in partnership with scientists to better understand the disease. Intestinal malrotation is a rare congenital anomaly that is poorly understood, leading to delays in diagnosis and an underrecognition of chronic gastrointestinal symptoms following surgical treatment. These challenges demonstrate the need for engagement across diverse stakeholders and for a robust Patient-Centered Outcomes Research/Comparative Clinical Effectiveness Research (PCOR/CER) Research Agenda that prioritizes research questions relevant to patients.

Proposed Solution to the Problem: This project will use a patient-centered approach to bring caregivers and patients living with intestinal malrotation together with researchers and clinicians across multiple specialty areas to establish the Intestinal Malrotation Patient and Clinician Team (IMPACT) and to develop an actionable PCOR/CER Research Agenda to work toward a better understanding of intestinal malrotation. Additionally, the project team will create tools and resources for other rare disease patients and patient advocacy organizations to build capacity in PCOR/CER.

Objectives: The primary objective of this project is to develop a patient-centered PCOR/CER Research Agenda to outline future research directions for intestinal malrotation. The project team will evaluate its methods and processes and develop community-initiated participatory research tools for the broader rare disease community to engage with researchers and clinicians to drive research of their rare disease.

Activities: The project team will develop and sustain IMPACT and a wider network of caregivers and patients (CAPs). The team will train stakeholders in PCOR/CER and hold listening sessions with CAPs to gather patient-centered information that will be used in virtual annual strategic planning meetings to draft, revise and finalize a PCOR/CER Research Agenda for intestinal malrotation prioritizing research directions important to CAPs. These meetings will also be used to create tools for the broader rare disease community to initiate research.

Projected Outcomes and Outputs:

Short-term outcomes during the project period include: a sustained IMPACT, a PCOR/CER Research Agenda for intestinal malrotation and tools for rare disease patient communities

Medium-term outcomes (0-2 years post project period) include: multiple research studies and a network of partnering institutions

Long-term outcomes (3+ years post project period) include: PCOR/CER studies to improve patient and clinician decision making and patient outcomes

Patient and Stakeholder Engagement Plan: The project will include patients, caregivers and additional stakeholders in each phase of the project. Using the extensive intestinal malrotation online communities and networks, the project team will recruit a large network of CAPs who will be trained in PCOR/CER. They will also establish IMPACTs made up of caregivers, patients and clinicians who will advise on all aspects of the project. The team will engage with CAPs through listening sessions and a virtual annual meeting and use online platforms to disseminate project information throughout the project period and beyond.

Project Collaborators: Project collaborators include the University of Oklahoma Health Sciences Center, West Virginia University, and Boston Children’s Hospital.

Project Information

Sydney Martinez, MPH, PhD
University of Oklahoma Health Sciences Center
$249,970

Key Dates

May 2025
2023

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Last updated: April 3, 2024