Patient participation in the development of a clinical guideline for inherited retinal dystrophies. (2nd July 2016)

• Record Type: Journal Article
• Authors: Serrano-Aguilar, P.; Trujillo-Martin, M. M.; del Pino-Sedeño, T.; Pérez de la Rosa, A.; de Pascual y Medina, A. M.; Perestelo-Pérez, L.; Toledo-Chávarri, A.; Posada de la Paz, M.; Sarría Santamera, A.
• Is Part Of: Expert opinion on orphan drugs. Volume 4:Number 7(2016:Jul.)
• Page Start: 691
• Page End: 697
• Publication Date: 2016-07-02
• Subjects: Inherited retinal dystrophy -- retinitis pigmentosa -- self-reported need -- Delphi method survey -- patient-centred care; Orphan drugs -- Periodicals; Rare diseases -- Periodicals; Chemotherapy -- Periodicals; 615.1
• DOI: 10.1080/21678707.2016.1182907
• Languages: English
• ISSNs: 2167-8707

Abstract

ABSTRACT: Objective : To identify the most relevant self-perceived health problems and unmet needs among adults with inherited retinal dystrophies (IRDs) in order to incorporate patients' perspective from the initial design of a clinical practice guideline (CPG) for IRDs in Spain. Research design and methods : A systematic review (SR) and a survey according to the Delphi method were carried out. Main Outcome Measures : Self-perceived health problems, unmet needs of care and treatment expectations related to IRDs. Results : From the SR, no information was found regarding unmet health care needs or dissatisfaction with health care services. In the survey, the most significant self-reported health problems were progressive decrease in visual acuity, visual field loss, abnormal sensitivity to light or glare, and night blindness. The main unmet needs were scarcity of disease information, lack of protocols for effective disease management and limited professional empathy. Treatment expectations were focused on the availability of protective and aids devices, easier access to rehabilitative services and psychological support. Conclusion : Patient involvement in CPG development helps to reduce the gap between the drawbacks of the routine clinical management and real-life needs of patients, especially in the context of rare diseases as IRDs where evidence-based therapies are scarce.