Incorporating the patient voice and patient engagement in GOAL-Hēm: Advancing patient-centric hemophilia care

Background: Goal Attainment Scaling for Hemophilia (GOAL-Hēm) is a novel, hemophilia-specific, validated patient engagement tool and patient-reported outcome instrument. Objective: We evaluated the degree to which the language of GOAL-Hēm was patient-centric and the content valuable and relevant for people with hemophilia (PWH) and/or their caregivers. Patients/Methods: Patients and caregivers participated in one of three investigations: an online survey, one-on-one patient interviews, or a focus group. The survey and interviews assessed the clarity and relevance of the GOAL-Hēm menu items. Interviews were semistructured, audio recorded, and transcribed verbatim. Feedback from interviews was coded as “clear,” “unclear,” “remove,” or “add.” The focus group explored participants’ experience of GOAL-Hēm and elicited recommendations for implementation. Quotations from focus group and interview transcripts were indexed and charted to emergent themes for analysis. Results: Participants comprised 19 adults with hemophilia and 19 caregivers of children with hemophilia (survey, n = 20; interview, n = 12; focus group, n = 6). After their feedback, 32% (15/48) of goals were retained unchanged. Further feedback resulted in the removal of 45% (286/635) of the goal descriptors, and 30% (193/635) of the retained descriptors were modified. Three new (total = 38) goals and 42 descriptors (total = 368) were added to the menu. Thematic analysis indicated that participants were enthusiastic about patient-centric language, empowered through the goal-setting process, and recognized GOAL-Hēm could measure clinically meaningful change. Conclusion: By listening closely to patients and caregivers, we refined GOAL-Hēm to better capture the experiences of PWH, enhance content validity, and augment implementation strategies. Incorporating the patient voice is integral to developing patient-centered outcome measures.