Hemophilia Trials in the Twenty-First Century: Defining Patient Important Outcomes

Hemophilia Trials in the Twenty-First Century: Defining Patient Important Outcomes

Research and Practice in Thrombosis and Haemostasis (Spring 2019) Vol. 379, No. 20, P. 184 Konkle, Barbara A.; Skinner, Mark; Iorio, Alfonso

The last five decades have been marked by significant progress in hemophilia care, which has expanded to include non-replacement therapies and the approaching availability of gene therapy. These advances, however, warrant taking a new look at the outcome measures used to evaluate factor replacement therapies in clinical studies, namely factor activity level and annualized bleeding rate. These endpoints should be reconsidered not only in the context of new hemophilia treatment strategies but also from the standpoint of a recent new emphasis on patient perspectives related to survival, functional status, and quality of life. Selecting and measuring patient-important outcomes, which often are patient-reported, is becoming a critical step in the clinical trial process. The research community recognizes that patients see issues through a different lens than clinicians, manufacturers, and other stakeholders; it places value on their insight into research design as well as their actual participation. What matters to patients are outcomes that encompass the whole cycle of care: survival, functional status, and quality of life.

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