Survey of patients' and families' experiences of rare diseases reinforces calls for a rare disease strategy

Abstract

Purpose: This paper seeks to report the key findings of two studies which were undertaken by Rare Disease UK to: understand patients' and their families' experiences of living with a rare disease; identify issues preventing research and access to good quality information, care, treatment and support; identify examples of good practice; and develop recommendations to improve service provision for patients with rare diseases and encourage research. Design/methodology/approach: Across the two reports discussed, a range of methods were used including: a survey of patients/family members; five multi-stakeholder working groups; conference workshops; a consultation paper; interviews; and desk resea..