Transfer of high cost drugs to NICE risks fragmentation of care of rare diseases

1 Commissioning of services for conditions individually affecting only 10% of the population presents challenges to value, fairness, and equity. Since 2005 the establishment of a UK National Commissioning Group for Rare Disorders (latterly National Specialist Commissioning Team) and Advisory Group for National Specialised Services (AGNSS) has become a model for evidence based evaluation and delivery of both drugs and services to which many countries aspire. 2 This process has been open and transparent using the same expertise to evaluate funding for service provision and treatments, thereby avoiding inefficient and artificial separation of commissioning for drugs and services. Rarity forces a close working relationship between all the clinicians, patient organisations, and multiple industry partners, and many of the authors will have received educational grants, honorariums, and help with service costs from these companies or be involved in clinical trials by them.