Rare diseases pose a particular challenge for healthcare systems: expertise is concentrated in a few centres, the evidence base evolves quickly, and access to specialist care can vary sharply depending on where a patient lives. For one rare, potentially life-threatening condition that causes recurrent and disabling episodes, guidance in the UK had not kept pace with new treatments and home-therapy options, and specialist provision was felt to differ across the country.
To set a national standard, a multidisciplinary UK steering group, including physicians, specialist nurses and a patient representative, agreed 48 statements across 11 themes. These were tested through a Delphi consensus distributed to UK physicians, specialist nurses, professional societies and patients, using a four-point agreement scale. In total, 91 healthcare workers and 36 patients from all four UK nations responded.
All 48 statements reached consensus in a single round, with more than 90% agreement on almost every statement among healthcare workers and over 97% among patients. The recommendations centred on patient self-treatment, the option of home administration with appropriate training and governance, and better-organised specialist services.
Triducive facilitated this consensus and collated the anonymised responses for the panel. The resulting UK framework complements existing guidelines and makes a strong, evidence-backed case for patient-centred, community-based care.
Longhurst HJ, et al. Clinical and Experimental Immunology, 2015; 180(3): 475–483. DOI: 10.1111/cei.12584
