Healthcare costs and outcomes of managing β‐thalassemia major over 50 years in the United Kingdom

The objective was to estimate the incidence-based costs of treating β-thalassemia major (BTM) to the United Kingdom's National Health Service (NHS) over the first 50 years of a patient's life in terms of healthcare resource use and corresponding costs and the associated health outcomes.This was a modeling study based on information obtained from a systematic review of published literature and clinicians involved in managing BTM in the United Kingdom. A state transition model was constructed depicting the management of BTM over a period of 50 years. The model was used to estimate the incidence-based health economic impact that BTM imposes on the NHS and patients' health status in terms of the number of quality-adjusted life-years (QALYs) over 50 years.The expected probability of survival at 50 years is 0.63. Of patients who survive, 33% are expected to be without any complication and the other 67% are expected to experience at least one complication. Patients' health status over this period was estimated to be a mean of 11.5 discounted QALYs per patient. Total healthcare expenditure attributable to managing BTM was estimated to be £483,454 ($720,201) at 2013/14 prices over 50 years. The cost of managing BTM could be potentially reduced by up to 37% if one in two patients had a bone marrow transplant, with an ensuing improvement in health-related quality of life.This analysis provides the best estimate available of NHS resource use and costs with which to inform policy and budgetary decisions pertaining to this rare disease.