ABSTRACT
BACKGROUND: Fabry disease is a rare, progressive X-linked lysosomal storage disorder. It is caused by mutations in the GLA gene resulting in deficiency of α-galactosidase A (α-Gal A), leading to peripheral neuropathy, cardiovascular disease, stroke, end-stage renal disease, gastrointestinal disorders and premature death. Given the long-term nature of disease progression, trials in Fabry disease are often not powered to capture these clinical events. Clinical measures such as estimated glomerular filtration rate (eGFR) and left ventricular mass index (LVMI) are often captured instead. eGFR and LVMI are believed to be associated with long-term Fabry disease clinical events of interest, but the precise relationships are unclear. OBJECTIVE: We aimed to identify published literature exploring the link between eGFR/LVMI and long-term clinical events in Fabry disease. METHODS: A comprehensive literature search was conducted in Embase® and MEDLINE® (using Embase.com), and a targeted literature review was conducted. Studies reporting a quantitative relationship between eGFR and/or LVMI and clinical events in Fabry disease were extracted, and narrative synthesis was conducted to understand these predictive relationships. RESULTS: Eight studies, consisting of seven patient-level retrospective analyses plus one prospective cohort study, met the inclusion criteria. Seven of these studies reported eGFR and six reported LVMI, with five reporting both. All studies presented results for either a composite measure including a range of key Fabry disease clinical events, or a composite outcome that included at least one key Fabry disease clinical event. All studies employed Cox proportional hazards survival modelling. The studies consistently reported that eGFR and LVMI are predictors of key clinical events in Fabry disease, with the findings remaining consistent regardless of the therapy received by patients in the studies. CONCLUSIONS: The evidence identified suggests that eGFR and LVMI outcomes may be appropriate indicators for long-term clinical events in Fabry disease, and all identified papers implied the same directional relationship. However, additional research is needed to further understand the specific details of these relationships and to quantify them.
Subject(s)
Fabry Disease , Humans , Fabry Disease/drug therapy , Glomerular Filtration Rate , Retrospective Studies , Prospective Studies , alpha-Galactosidase/genetics , alpha-Galactosidase/therapeutic useABSTRACT
INTRODUCTION: In the UK, biologic interventions for plaque psoriasis can either be administered in a hospital setting or following delivery to a patient's home. To date, limited research has been undertaken on how the administration route affects the overall treatment costs and the implications for this on UK clinical practice. The objective was to explore the cost implications of different administration routes for plaque psoriasis biologic interventions in the UK. METHODS: A simple economic model was developed to estimate and compare the total cost of drug administration over 2 years for all biologic interventions that have been approved by the National Institute of Health and Care Excellence for use in patients with moderate-to-severe plaque psoriasis. Administration costs were estimated for two different scenarios: administration in a hospital setting or following home delivery [paid for by the National Health Service (NHS)]. RESULTS: Costs of home delivery and administration in hospital over a 2-year time horizon varied substantially based on the choice of intervention. For home delivery, the lowest cost of £693 occurred with risankizumab, tildrakizumab and ustekinumab, while the highest cost of £3445 occurred with adalimumab, brodalumab, certolizumab and etanercept. For the scenario in which the interventions were administered in a hospital setting the costs ranged from £4224 for ustekinumab to £7463 for brodalumab. CONCLUSION: These results indicate that drug administration costs are meaningful and should be given greater consideration in the selection process of treatments for plaque psoriasis. Additionally, the NHS could save money by paying for drugs to be delivered to a patient's home, rather than administering them in a hospital setting.
ABSTRACT
INTRODUCTION: In the UK, biologic interventions for plaque psoriasis can either be administered in a hospital setting or following delivery to a patient's home. To date, limited research has been undertaken on how the administration route affects the overall treatment costs and the implications for this on UK clinical practice. The objective was to explore the cost implications of different administration routes for plaque psoriasis biologic interventions in the UK. METHODS: A simple economic model was developed to estimate and compare the total cost of drug administration over 2 years for all biologic interventions that have been approved by the National Institute of Health and Care Excellence for use in patients with moderate-to-severe plaque psoriasis. Administration costs were estimated for two different scenarios: administration in a hospital setting or following home delivery [paid for by the National Health Service (NHS)]. RESULTS: Costs of home delivery and administration in hospital over a 2-year time horizon varied substantially based on the choice of intervention. For home delivery, the lowest cost of £693 occurred with risankizumab, tildrakizumab and ustekinumab, while the highest cost of £3445 occurred with adalimumab, brodalumab, certolizumab and etanercept. For the scenario in which the interventions were administered in a hospital setting the costs ranged from £4224 for ustekinumab to £7463 for brodalumab. CONCLUSION: These results indicate that drug administration costs are meaningful and should be given greater consideration in the selection process of treatments for plaque psoriasis. Additionally, the NHS could save money by paying for drugs to be delivered to a patient's home, rather than administering them in a hospital setting.
