Patient Reported Outcomes, Burdens, and Experiences (PROBE) Study

The mission of the Patient Reported Outcomes, Burdens, and Experiences (PROBE) study is to investigate and directly probe patient perspectives on outcomes they deem relevant to their life and care. PROBE aims to develop a new global tool to enhance the direct patient-voice in health care decision-making. Government and private payers increasingly value data based on patient-centered outcomes research as part of the overall cost-benefit evaluation of high-cost care and treatment of diseases such as hemophilia. This emerging dimension of the healthcare environment presents a significant opportunity and urgent need to improve patient organizations’ ability to collect and interpret relevant outcomes data. More robust patient reported data will improve advocacy efforts to build comprehensive care programs, promote home treatment and implement preventative treatment regimens thus allowing advocacy arguments to move beyond emotion and anecdote to those grounded in real-world patient experiences and evidence. Through PROBE we seek to validate the reliability, reproducibility and responsiveness of a low cost, easily administrable inventory for collecting patient self-reported outcomes, burdens and experiences in living with hemophilia. We anticipate that the metrics established through PROBE will allow for comparison of patient outcomes within a country over time and cross-sectionally between countries (regionally and globally). PROBE is an independent investigator-led research project conducted as a multi-part study.