The economic burden of tuberous sclerosis complex in the UK: A retrospective cohort study in the Clinical Practice Research Datalink

Abstract

Background: Tuberous sclerosis complex (TSC) is a multi-system genetic disorder characterized by the development of diverse clinical manifestations. The complexity of this disease is likely to result in substantial challenges and costs in disease management throughout the patient's lifetime. This retrospective database study aims to quantify healthcare resources utilized by TSC patients. Methods: TSC patients in the Clinical Practice Research Datalink linked to the Hospital Episodes Statistics database were identified between January 1987 and June 2013. Analyses were conducted over the most recent 3-year period of data and stratified by pediatric (< 18) and adult patients. Prescriptions, procedures, diagnostic tests, and healthcare encounters were reported in comparison with a matched comparator cohort. Costs and key economic drivers by primary organ system manifestations were also examined. Results: A total of 286 patients with TSC were identified and consistently reported 2-fold greater resource use than the matched presumably healthy controls. Despite this comparatively greater resource use, half of TSC patients did not record any procedures, and 20% of patients did not record any diagnostic tests; however, inpatient hospitalizations were greater for the TSC cohort (3.1 vs 1.3), but length of stay was comparable. TSC patients had costs totaling £12,681 per patient over the 3-year period, a figure 2.7-fold greater than the total costs in the comparator cohort (£4,777). Costs for patients with specific primary manifestations were even greater, with brain manifestations incurring £22,139 per affected patient. Kidney and nervous system manifestations were the main cost drivers. Conclusions: The economic burden of TSC and its impact on NHS healthcare resources is mostly attributable to the broad spectrum of manifestations that develop within multiple organ systems. TSC patients may benefit from co-ordinated care based on their requirement for high numbers of healthcare visits across specialties.

Renal manifestation of tuberous sclerosis complex

“…Exhaustive search of commercial and Medicaid databases in the United States indicates that TSC‐renal disease patients utilized more healthcare services than their matched controls, with an annual average total cost that is 10–20 times higher than that of control patients ( p < .05) (Song et al, ). Work from the United Kingdom confirms the high economic burden of TSC (Kingswood, Crawford, et al, ) and that renal complications are a major contributor to this cost (Kingswood, Nasuti, et al, ). The National Institute of Diabetes and Digestive and Kidney Diseases even convened a panel of experts working in the field to identify key unknowns and define possible “next steps” in advancing understanding of TSC‐dependent and mTORC1‐dependent renal disease (Henske, Rasooly, Siroky, & Bissler, ).…”

A systematic review on the burden of illness in individuals with tuberous sclerosis complex (TSC)

“…Vigabatrine is used significantly more often by children (43.2%) than adults (24.4%) as reported from UK data [23]. Several studies showed that individuals with TSC require between four and eight times more prescriptions than the general population [23,26,110]. In a UK study, hypnotics or antipsychotic drugs (not differentiated) were prescribed twice as often in individuals with TSC than in the general population.…”

“…In a UK study, hypnotics or antipsychotic drugs (not differentiated) were prescribed twice as often in individuals with TSC than in the general population. In particular, the difference was sevenfold in children with TSC as compared with in the general population [110]. However, in a French study in which 80% had intellectual disabilities and 70% had psychiatric disorders, only 20% received pharmacotherapy [53].…”

“…Of these, 21 papers report health care resource use [23, 24, 27, 35, 53, 56, 86, 104-108, 110-112, 114, 116, 118, 119, 120, 125]. Direct costs are reported by 10 studies [23, 35, 104, 106, 107, 110-112, 114, 119] and four studies assessed drivers of direct cost [23,35,110,111]. The measures of DALY and QALY were only used by one study that calculated projected costs of different treatment strategies for epilepsy in TSC [109].…”

“…Inhibitors of mTOR were used in 15.3% in one study, without differentiation of application or indication [27]. Anxiolytic therapy may be overstated, as benzodiazepines are also used as ASD and several studies do not precisely differentiate between related drug classes or indications [23,27,56,110]. In TOSCA, 98.1% of patients with focal seizures received ASD treatment, most commonly with GABAergic anticonvulsants (66%) [26].…”