Module 1: What Is Patient-Centered Research — and Why Does It Matter for SDS?

About this Module If you live with Shwachman-Diamond Syndrome — or care for someone who does — you already know what matters most. You know what it feels like to wait for a bone marrow result, to navigate feeding difficulties, to explain to someone why your child looks fine but is not, or to wonder whether a clinical trial is the right choice. Researchers need to know what you are experiencing in real life. Not as a formality, but to align their research to what actually helps people like you. This module introduces you to patient-centered research: what it is, how it works, and what your role in it can look like. It starts with a presentation given to the SDS community at our PFDD meeting in June 2025 — grounded in SDS from the start, with real examples drawn from our community's experience. It then points you to PCORI's own training resources for those who want to go deeper. You do not need any scientific background to complete this module or engage in research. If you have been living with SDS or are managing it, you already have knowledge that researchers need. What you will be able to do after this module: - Explain what patient-centered comparative effectiveness research (CER) is and how it differs from other kinds of research - Describe the role patients and caregivers can play at different stages of the research process - Connect the community priorities identified in our survey to real research questions in SDS - Understand what stakeholder engagement means — and what it can look like in practice - Feel more confident discussing research with clinicians, researchers, and other stakeholders