Patient experience data (PED) has become increasingly important in drug development, and especially so for rare disease, offering critical insight into how people live with their condition, what outcomes matter most to them and how they experience participation in research. Maintaining this focus on patient priorities is particularly important in rare disease, where traditional measures of success may not always reflect what matters most to patients. As rare disease patient advocate and cystic fibrosis survivor, Caleigh Haber observed: “A statistical win is not always a patient win.” Patient communities in rare disease are often small, highly engaged and deeply invested in advancing research. In one recent rare disease programme, we worked alongside both patient communities and the sponsor following the decision to discontinue development.