This public meeting discusses approaches and opportunities for engaging patients, patient groups, rare disease or condition experts, and experts on small population studies during the drug development process for rare diseases. The meeting focused on how to best understand patients’ experiences living with a rare disease and how to incorporate those experiences and priorities throughout the drug development process. This includes understanding patient perspectives on the burden of their condition and any existing treatment options, as well as how their current health status and risk of disease progression may impact willingness to accept risks from treatment side effects.

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