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

Why This Module Exists 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 isn't, or to wonder whether a clinical trial is the right choice. Researchers need to know what you know. Not as a formality, but as a genuine requirement for doing research that 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 draws on training developed by PCORI — the Patient-Centered Outcomes Research Institute, the organization that funds Project PACER — and connects it directly to questions that matter in SDS. You do not need any scientific background to complete this module. If you have been managing SDS, you already have knowledge that researchers do not. 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 Feel more confident discussing research with clinicians, researchers, and other stakeholders