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Ultra orphan drugs: the NHS model for managing extremely rare diseases

Introduction: This review sets out the English model for managing extremely rare diseases. In the planning, funding and monitoring of healthcare services, the National Health Service (NHS) in England works with a concept of ‘ultra orphan’ rather than orphan. Ultra orphan diseases affect less than 1 person in 100,000 but rarity is not the sole criterion. Areas covered: The history of planning and funding treatment of rare diseases is explained. The roles of NHS England and The National Institute for Health and Care Excellence in the ultra orphan field are set out, and how drug spend is contained through the Pharmaceutical Pricing and Reimbursement Scheme. This review also describes the functioning of expert centres for patient with ultra orphan diseases. Expert opinion: The concept of ultra orphan disease grew out of the English system for planning and organising health services, but is likely to be of increasing interest to planners and funders in other health systems struggling to make decisions about very high cost therapies for very rare disease.