Patient Reported Outcomes Burdens and Experiences - Phase 2 - Pilot Feasibility Study

PROBE is the repository of a standardized survey of patients with hemophilia from a group of countries, to be run annually, collecting anonymized information on quality of life measures and treatment modalities. After developing the tool in phase 1 of the project, a pilot feasibility study is hereto described.

Rationale/Goals

Research based on patient-centered data is increasingly valued by government and private payers and utilized in public-policy decisions as part of the cost-benefit justification for high-cost care and treatment. A frequent question of payers is: Will additional investment (to sustain or expand care) improve the lives of persons with hemophilia?

There is a significant need to improve our ability to collect, collate, and interpret relevant patient-centered data to support the implementation of comprehensive care, home treatment and preventative treatment regimens (e.g., prophylaxis). The PROBE study is intended to fill this gap and enhance the direct patient-voice in the delivery of care and move advocacy efforts to sustain and expand care beyond emotion to arguments grounded in evidence and data.

The overall inventory and the specific metrics developed through PROBE will complement the existing global comparative metrics of the World Federation of Hemophilia (WFH) as provided in the WFH Global Survey (e.g., the percent of patients identified vs. expected within a country, amount of treatment products available within a country on a per capita basis, and the ratio of children to adults surviving into adulthood). While the WFH global metrics are valued and useful, they do not provide a complete or direct insight as to how patients are fairing within their health care system. The existing WFH metrics only serve as proxy measures of the impact of care on patient outcomes within a country.