Rare Disease: Beginning With The Patient In Mind

Patient centricity is top of mind in drug development today – and rightly so. The rapid evolution of the industry over the past year alone has brightened that spotlight more than ever, making patient centricity imperative. The question becomes: How do we expand perspectives from a myopic scientific lens to apply holistic real-world considerations to drug development strategy and trial design that truly have the patient in mind? Specifically, how do we accommodate the true real-world patient journey in a population that is unlike any other – the rare disease patient?

Involving patients in the drug development process provides context for benefit-risk assessment as well as aids in the development of new assessment tools, study endpoints, and risk communications.¹ Creating genuinely patient-centric drug development programs takes careful consideration and intention; in some cases, it requires an additional financial investment. But this effort can result in generating better outcomes for all stakeholders and reflect the ultimate goal of research and development – an improvement in the lives of more patients.