N/A
N=656
Patient Reported Outcomes Burdens and Experiences - Phase 2
Hemophilia
Bottom Line
View on ClinicalTrials.gov: NCT02439710 ↗Enrolled (actual)
656
Serious AEs
0.0%
Results posted
Oct 2019
Primary outcome: Primary: Response Rate — 656 Questionnaires
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Quality of life measure (survey) (Other)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- McMaster University
- Primary completion
- Dec 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Response Rate |
656 | — |
| PRIMARY Number of Participant Completing the Questionnaire Within 15 Minutes |
474 | — |
| PRIMARY Cost Per Completed Survey |
8.50 | — |
Summary
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).
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT02439710). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.