ArticleOrphanet journal of rare diseases2021
Results of a Patient Reported Experience Measure (PREM) to measure the rare disease patients and caregivers experience: a Spanish cross-sectional study.
Article in Orphanet journal of rare diseases, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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Who cites it
8 citing papers in PubMed.
- 'If You Don't Fight for Yourself, No One Else Will': An In-Depth Interpretative Phenomenological Analysis of Rare Disorder Care Advocacy in Aotearoa New Zealand.Health expectations : an international journal of public participation in health care and health policy · 2026Article
- Implementation of a novel patient reported experience measure (PREM) in rheumatology: a cross-sectional online survey of Australian rheumatology outpatients.Rheumatology international · 2025Article
- Perceptions of Healthcare Quality in Duchenne Muscular Dystrophy: A Patient Experience Exploratory Study.Healthcare (Basel, Switzerland) · 2025Article
- Content validity of patient-reported measures evaluating experiences of the quality of transitions in healthcare settings-a scoping review.BMC health services research · 2024Article
- Integrated Collection of Patient-Reported Outcomes and Experiences in Children with Kidney and Hematological Diseases: A Pilot Study.Children (Basel, Switzerland) · 2023Article
- Article
- Patient-Reported Experience Measures in outpatient rheumatology care: a systematic review.Rheumatology advances in practice · 2021Article
- From care to cure: a patient engagement framework for rare disease and orphan drug research.Therapeutic advances in rare diseaseArticle
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo measure the experience of the person having a rare disease in order to identify objectives for optimal care in the health care received by these patients.
methodsA cross-sectional study was conducted in Spain involving patients associated with the Spanish Rare Diseases Federation [Federación Española de Enfermedades Raras] (FEDER). A modified version of the PREM IEXPAC [Instrumento para evaluar la Experiencia del Paciente Crónico] instrument was used (IEXPAC-rare-diseases). Scores ranged between 0 (worst experience) and 10 (best experience).
resultsA total of 261 caregivers (in the case of paediatric population) and patients with rare diseases (response rate 54.4%) replied. 232 (88.9%) were adult patients and 29 (11.1%) caregivers of minor patients. Most males, 227 (87%), with an average age of 38 (SD 13.6) years. The mean time since confirmation of diagnosis was 7.8 (SD 8.0) years. The score in this PREM was 3.5 points out to 10 (95%CI 3.2-3.8, SD 2.0). Caregivers of paediatric patients scored higher, except for coordination of social and healthcare services.
conclusionsThere are wide and important areas for improvement in the care of patients with rare diseases. This study involves a first assesment of the experience of patients with rare diseases in Spain.
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