Development and preliminary validation of a questionnaire assessing caregivers’ knowledge, challenges, and readiness to use mobile application for medical nutritional management of inborn errors of metabolism

Managing inborn errors of metabolism (IEMs) requires complex, lifelong care that places substantial responsibility on caregivers. Despite their critical role, only a limited number of validated instruments exist in English, and to date, no validated tools are available in Arabic. This study aims to develop and validate a preliminary questionnaire that assesses caregivers’ knowledge, challenges, and readiness to use a mobile application for managing IEMs. A cross-sectional design guided tool development through preliminary concept elicitation, literature review and expert input, yielding 28 items. Eight bilingual experts evaluated content validity using Item- and Scale-Level Content Validity Indices (I-CVI and S-CVI). Forward–backward translation followed WHO procedures, with agreement rates measured. Face validity was explored through cognitive interviews with caregivers ( n = 5). Reliability of the tool was assessed using responses from 20 caregivers via Cronbach’s alpha and mean inter-item correlation (MIIC). Content validity was excellent (S-CVI = 0.97); all 28 items met I-CVI ≥ 0.78. Translation accuracy was 89.3% (forward) and 96.4% (backward). Clarity scored 97.8%. Reliability was excellent for items related to knowledge (α = 0.93; MIIC = 0.56) and readiness (α = 0.89; MIIC = 0.58) domains, while acceptable for challenges (α = 0.70; MIIC = 0.29). Overall, caregiver scores indicated high level of self-reported knowledge (87.6%), challenges (81.25%), and readiness (89.5%) to use an mHealth application. The questionnaire demonstrated strong validity, reliability, and cultural suitability, providing the first Arabic tool for assessing caregiver readiness to adopt digital health solutions in IEM management.