Advertising and Promotion Regulatory Affairs Conference

FDA recognizes that the patient perspective on new drug benefits and risks, and whether the disease burdens that matter most are addressed, is critically informative to regulatory decision-making. Drug development programs have often failed to explicitly account for the disease burdens and treatment burdens that would matter most to patients and fail to reliably measure these effects in clinical trials. FDA expects drug sponsors to incorporate the patient’s perspective starting early in development to ensure that the investigational product profile and development program incorporate clinical outcomes and endpoints that reflect what matters to patients. This session describes the background and content of a new FDA guidance series to foster patient focused drug development and sponsor use of such outcomes and endpoints. Reliable measures of what matters to patients and evidence of meaningful improvement will not only inform FDA decision making but may also better inform the decisions of other health authorities (e.g., HTA and payors).