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How to measure outcomes in hemophilia: patient, physician and payer perspectives

Prophylaxis is costly and necessary throughout the patient’s life. The evaluation of treatment outcomes can help secure access to hemophilia therapy and improve its efficiency and cost effectiveness.

During the medical plenary, Kathelijn Fischer, Associate Medical Professor, University Medical Center, Utrecht, defined those involved in treatment and how their expected outcomes differed. “Patients want a normal life, healthcare providers aim to optimize treatment and payers work towards reducing costs.”

There are a variety of outcome parameters for hemophilia: bleeding symptoms, pain, joint health and health-related quality of life (HRQoL). “But how do we measure them?” asked Fischer. While bleeding is hard to diagnose and definitions vary, pain can be evaluated using surveys such as the short-form McGill Pain Questionnaire. Joint health can be quantified using the Haemophilia Joint Health Score, but ultrasound and magnetic resonance imaging techniques are increasingly used to detect changes in the synovium and cartilage in the absence of bleeding.

“In many cases, HRQoL is the most important outcome for the patient,” said Fischer. There are 10 HRQoL examinations available for both children and adults in the field of bleeding disorders. The volume of tools and the variation between each one makes it difficult to compare outcomes across individuals. Fischer provided an example of a wheelchair-bound person with hemophilia with a high reported HRQoL. Although the patient seemed to be satisfied, their treatment outcomes did not align with the healthcare provider’s goal of optimizing treatment. “This highlights the need for a combination of both subjective and objective tools,” concluded Fischer.

“Using dissimilar outcome measures to assess drugs in different trials makes it impossible to judge therapies against a common standard,” said Brian Feldman, MD, Professor of Pediatrics at The Hospital for Sick Children in Toronto, Canada. He further explained a core set of subjective and objective tools for measuring outcomes in arthritis, and how these could be applied to hemophilia. Such tools would allow accurate comparison of clinical trial data and address what is important to treaters, patients and payers.

Mitch Semienchuk, Editor, Hemophilia World Online, wishes to thank Georghia Michael. PhD, for her contributions to this article.