Patient-reported outcomes, and other patient-relevant measures and endpoints

Patient-reported outcomes (PROs) can be measured in parallel with clinical outcomes to record the patient’s lived experience and burden of a disease, its treatment and management, and its influence on day-to-day functioning and health-related quality of life. PROs provide a patient perspective on a disease and treatment that is not captured by clinical measurement, but may be as important to the patient as clinical outcomes. The inclusion of PROs as trial endpoints help to guide treatment choice, the development of guidelines, and regulatory decision making on risks and benefit of a new treatment.

However, a meaningful measurement of PROs in clinical trials that truly reflects patient experiences, as well as the measurement of PROs in clinical practice, is still in infancy today – either because appropriate PRO instruments are not available, or are not used appropriately to measure patient reality, disease burden and treatment burden. Even if PROs are measured, the findings are often not published properly. In addition, PRO data is mostly not comparable between clinical studies, which is still an unresolved methodological issue. However efforts are being done by various international groups to address these issues and move the field forward.

This is why patient advocates need to be involved in the development and use of PRO measurements by bringing in their specific knowledge and expertise. To be able to do so, patient advocates need to understand what PROs are, how PROs are being measured, and how they can bring the patient perspective into development, use and reporting of PRO measurements.

Although PROMS have been used previously for patient research, there remain questions about how consistently such tools should be used for myeloma research. There is also still some academic debate regarding the method’s effectiveness for measuring what really matters to myeloma patients. However, Myeloma UK consider PROMS to be a highly effective method to help understand the impact that myeloma has on individuals, so that their needs can be addressed and that the data gathered can drive improvement in outcomes for patients.

Presentation at the WECAN Academy 2019: