Building Capacity for Patient-Centered Clinical Comparative Effectiveness Research (CER) in Rare Lung Disease

Children's Interstitial and Diffuse Lung Disease (chILD) includes rare pulmonary disorders that cause significant morbidity and mortality. These heterogeneous conditions vary in severity, age of onset, and prognosis, with some children requiring minimal support while others face life-threatening challenges or need lung transplantation. Families often struggle with accessing specialized care and managing their child's developmental and emotional needs, alongside finding effective treatments. Additionally, there is limited data on long-term outcomes, including mental health and quality of life. A collaborative, patient-centered approach is vital to address the diverse needs of this population.

chILD disorders represent a significant unmet need in research and treatment. This project aims to establish a collaborative patient-centered clinical effectiveness research (CER) program focused on chILD. By engaging patients, caregivers, and clinicians, the goal is to empower stakeholders, enhance patient care, and create sustainable research addressing the unique challenges faced by those affected by chILD.

Objective: To establish a sustainable, patient-centered CER program for chILD.

• Patient-Centered Research Community – Establish a collaborative network of patients, caregivers, clinicians, and researchers from multiple chILD centers. All members will complete the PCORI Research Fundamentals curriculum and participate in chILD-specific stakeholder webinars.
• Stakeholder Engagement – Two cycles of virtual engagement studios, focus groups, and in-person meetings to generate conceptual models, prioritize research questions, and ensure patient and caregiver perspectives guide the process.
• Annual Hybrid Meeting – Brings stakeholders together each year to prioritize research questions and develop research infrastructure and long-term sustainability plans.
• Dissemination – The research priorities and interests and engagement strategies will be shared via virtual meetings, publications, and our website, reaching both the chILD and broader rare disease communities.

Short-Term (Project Period)

• A diverse, engaged research community trained to participate in CER.
• Completion of the stakeholder engagement process and identification of prioritized CER questions.
• A comprehensive CER research priorities and interests for chILD disorders.

Medium-Term (0–2 Years Post-Project)

• Ongoing dissemination of results through virtual meetings, publications, and social media.
• Continued engagement of the CER community and growing investment in chILD research.
• Raised awareness of CER-trained stakeholders available to collaborate on future projects.

Long-Term (3+ Years Post-Project)

• A sustained program of patient-centered CER focused on improving outcomes for all forms of chILD.
• Empowerment of patients and families to actively participate in research and decision-making.
• Improved outcomes and quality of life for chILD patients across all stages of life.

This project will involve patients, caregivers, clinicians, and researchers from the outset. A Project Oversight Team, including physicians, a parent outreach coordinator, and stakeholder leaders, will guide the project's direction. Stakeholders will be recruited through our Parent/Patient Advisory Committee, online communities, and social media. Physicians and allied health professionals will be drawn from our existing research network. This inclusive, collaborative approach ensures that the voices of patients and families are central to the research process.