Multi-Disciplinary Approach to Develop an Ultra-Rare Disease Decision Aid

OBJECTIVES: Despite multiple treatment options for paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare and potentially life-threatening hemolytic disease, no comprehensive patient decision aid (DA) existed to support treatment selection.

METHODS: A multi-disciplinary team designed, tested, and validated a web-based DA using primary and secondary research (targeted literature reviews, cognitive interviews (n=4 physicians; n=10 patients), and decisional needs analysis (n=12 physicians; n=60 patients)).

RESULTS: Development of the DA relied on a team comprised of qualitative researchers to capture decisional needs and facilitate cognitive interviews; market research experts to identify individualized, optimal treatment plans based on conjoint analysis; and a Steering Committee to provide clinical input and represent the patient voice. Development of the DA closely followed the International Patient Decision Aids Standards (IPDAS) and the Standards for UNiversal reporting of patient Decision Aid Evaluation (SUNDAE) Checklist. Orientation towards the Steering Committee charter and bi-directional communication across all parties was essential for data collection, analysis, and iterative updates to the web-based DA.

CONCLUSIONS: Qualitative and quantitative data from feasibility and usability testing indicated that the tool was appropriate for the target population and accepted by end-users. This multi-disciplinary approach can be applied as a model for DA development for other health conditions to ensure tool validity and reliability.