Patient Reported Outcomes Burdens and Experiences - Phase 2 (PROBE)
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ClinicalTrials.gov Identifier: NCT02439710
Recruitment Status : Unknown
Verified October 2015 by McMaster University. Recruitment status was: Recruiting
The Patient Reported Outcomes, Burdens, and Experiences (PROBE) Study 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. With the support of the National Hemophilia Foundation, a global team of investigators will lead a patient focused research project to investigate and directly probe patient perspectives on outcomes they deem relevant to their care. Through PROBE, the investigators will develop and 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. The investigators 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).
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Layout table for eligibility information
Ages Eligible for Study:
Child, Adult, Older Adult
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Moderate / Severe PWH (Age bands or limits may be utilized to narrow the study population). Patients will be recruited according to the final inclusion criteria. No randomization will be required. Two moderate to large test runs of the inventory will be conducted in each country three months apart to demonstrate reproducibility. PWH participating in each of the two test runs may, but would not necessarily be the same.
PWH will be recruited through national hemophilia patient organizations utilizing their existing membership rosters, social media outlets and meetings / events. The investigators are not proposing a pre-determined method of PWH recruitment. They will utilize the information acquired in the workshop and take-home project to inform best practice in recruitment methodology for the study. The investigators may consider requesting different countries test different PWH recruitment strategies to test reproducibility.
Disease severity and Age bands or age limits (e.g. ≥ Age 18) may be utilized to narrow the study population. During the January 2015 meeting of the investigators the inclusion / exclusion criteria will be finalized based on feedback learned during the workshop. Disease severity will be based on patient self-reported severity level.