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:
Patient-reported outcomes, and other patient-relevant measures and endpoints
Presenters: Sergio Diaz, Myeloma UK & Madeline Pe, EORTC
The following presentations held at the WECAN Academy 2019 will help you to understand:
- What is the difference between quality of life / health related quality of life
- What are PROMs, PROs and PREMs?
- What is the difference of PRO tools in clinical trials (e.g. EORTC QLQ30), and PRO tools used in clinical practice (continuous monitoring, e.g. HMPro)?
- Real examples of using PROs in trials vs clinical practice
- What “families” of PRO tools exist to measure PRO and HRQoL – the generic ones (e.g. EQ5D), the symptom specific ones (e.g. fatigue, headache scales), the disease specific ones (e.g. CML, Myeloma, etc)
- Innovative approaches to collecting PRO data (e.g. ePRO) – and their challenges?
- Why are PROs so important to patient advocates, and how they can be involved in PROM development and PRO tool selection
- Example and process to validate a new tool
Links and references
- Osborn et al.2015 BMC Cancer. Improving the assessment of quality of life in the clinical care of myeloma patients: the development and validation of the Myeloma Patient Outcome Scale (MyPOS)
Additional references and relevant links
- Including the patient voice in the development and implementation of patient-reported outcomes in cancer clinical trials. Addario, Geissler, Horn, Krebs, Maskens, Oliver, Plate, Schwartz, Willmarth, in: Health Expectations (2019), doi: 10.1111/hex.12997
- FDA-ASCO Workshop 2019: Clinical Outcome Assessments in Cancer Clinical Trials, 12 July 2019
- EORTC Quality of Life Group (QLG) – free resources on various cancer-specific HRQOL questionnaires developed by the EORTC QLG (including the Item Library and Computer Adaptive Tests). It also provides free manuals related to the development and use of EORTC HRQOL instruments.
- EORTC Quality Of Life Group Item Library – library of questions for the development of new HRQoL instruments. Allows to see how items were formulated in previous questionnaires in order to reuse existing validated items and their translations.