The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia

M W Skinner, C Chai-Adisaksopha, R Curtis, N Frick, M Nichol, D Noone, B O'Mahony, D Page, J S Stonebraker, A Iorio, M W Skinner, C Chai-Adisaksopha, R Curtis, N Frick, M Nichol, D Noone, B O'Mahony, D Page, J S Stonebraker, A Iorio

Abstract

Background: The interest of health care agencies, private payers and policy makers for patient-reported outcomes (PRO) is continuously increasing. There is a substantial need to improve capacity to collect and interpret relevant PRO data to support implementation of patient-centered research and optimal care in haemophilia. The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project aims to develop a patient-led research network, to develop a standardized questionnaire to gather patient-reported outcomes and to perform a feasibility study of implementing the PROBE questionnaire.

Methods: A pilot questionnaire was developed using focus group methodology. Content and face validity were assessed by a pool of persons living with haemophilia (PWH) and content experts through interactive workshops. The PROBE questionnaire was translated with the forward-backward approach. PROBE recruited national haemophilia patient non-governmental organizations (NGOs) to administer the questionnaire to people with and without haemophilia. PROBE measured the time to complete the questionnaire and gathered feedback on its content and clarity; staff time and cost required to implement the questionnaire were also collected.

Results: The PROBE questionnaire is comprised of four major sections (demographic data, general health problems, haemophilia-related health problems and health-related quality of life using EQ-5D-5L and EQ-VAS). Seventeen NGOs participated in the pilot study of the PROBE Project, recruiting 656 participants. Of these, 71% completed the questionnaire within 15 min, and all participants completed within 30 min. The median total staff and volunteer time required for the NGOs to carry out the study within their country was 9 h (range 2 to 40 h). NGO costs ranged from $22.00 to $543.00 USD per country, with printing and postage being the most commonly reported expenditures.

Conclusions: The PROBE questionnaire assesses patient-important reported outcomes in PWH and control participants, with a demonstrated short completion time. PROBE proved the feasibility to engage diverse patient communities in the structured generation of real-world outcome research at all stages.

Trial registration: Trial registration: NCT02439710.

Keywords: Haemophilia; Patient-centered research; Patient-reported outcomes; Quality of life.

Conflict of interest statement

Approval from the institutional review board or ethics committee was obtained. A participant description and disclaimer was provided with each questionnaire, and then, the participants were invited to proceed if they agreed or have an opportunity to ask questions. No identifiable patient information was disclosed as part of the study.Not applicable.CC has no potential conflict of interest. AI received grants from Baxalta now part of Shire, Bayer, Bioverativ, Sobi, Novo Nordisk and Roche. AI received non-financial support from US National Hemophilia Foundation. RC received grants from Baxalta now part of Shire, Bayer, Bioverativ, Sobi, Novo Nordisk and Roche. RC received non-financial support from US National Hemophilia Foundation. MN received grants from Bayer, Pfizer, Genentech, Biogen, Novo Nordisk, Baxter and CSL Behring. MN received personal fees from Bayer and Biogen. DN received grants from Baxalta now part of Shire, Bayer, Bioverativ, Sobi, Novo Nordisk and Roche. DN also received non-financial support from US National Hemophilia Foundation. BOM received grants from Baxalta now part of Shire, Bayer, Bioverativ, Sobi, Novo Nordisk and Roche. BOM received non-financial support from US National Hemophilia Foundation. MS received grants from Baxalta now part of Shire, Bayer, Bioverativ, Sobi, Novo Nordisk and Roche. MS also received non-financial support from US National Hemophilia Foundation.Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Figures

Fig. 1
Fig. 1
Flow diagram of the PROBE study
Fig. 2
Fig. 2
Participating countries: Argentina (Cordoba Chapter), Australia, Brazil, Canada, France, Germany, Hungary, Ireland, Italy, Japan, Mexico, The Netherlands, New Zealand, Spain, the UK, the USA, Venezuela
Fig. 3
Fig. 3
Number of participants by country
Fig. 4
Fig. 4
Time to completion of the PROBE questionnaire

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