Abstract
Background Congenital hyperinsulinism (CHI) is a rare, genetic disease which causes persistent hypoglycaemia, typically in new-borns. Patients with the diffuse disease variant often require near-total surgical removal of the pancreas, causing insulin-dependent diabetes mellitus (IDDM). The CHI economic burden is currently unknown. This study aimed to estimate the annual cost of illness (COI) of CHI patients in the UK from a service provider perspective (National Health Service, NHS and Personal Social Services), and to explore cost distribution within the patient population.
Highlights
The disease is characterised by debilitating comorbidities such as severe and progressive mobility loss, visual and audio impairments, diabetes mellitus and cardiac abnormalities, which often result in premature death
This study aimed to evaluate the cost of illness (COI) from a service provider (National Health Service, NHS) and societal perspective of all patients with Friedreich’s ataxia (FRDA) in the UK
The development and delivery of new treatment options, to address the loss of mobility and cardiac abnormalities experienced by FRDA patients, will substantially reduce the economic burden of this rare and devastating disease
Summary
This study aimed to evaluate the cost of illness (COI) from a service provider (National Health Service, NHS) and societal perspective of all patients with FRDA in the UK. Results: Overall, the total COI of FRDA patients to the NHS was £8,038,645 per year, with a mean annual cost per patient of £3,556.
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