Costs and outcomes over 36 years of patients with phenylketonuria who do and do not remain on a phenylalanine-restricted diet.

BACKGROUND: To quantify the costs and consequences of managing phenylketonuria (PKU) in the UK and to estimate the potential implications to the UK's National Health Service (NHS) of keeping patients on a phenylalanine-restricted diet for life. METHOD: A computer-based model was constructed depicting the management of PKU patients over the first 36 years of their life, derived from patients suffering from this metabolic disorder in The Health Improvement Network database (a nationally representative database of patients registered with general practitioners in the UK). The model was used to estimate the incidence of co-morbidities and the levels of healthcare resource use and corresponding costs over the 36 years. RESULTS: Patients who remained on a phenylalanine-restricted diet accounted for 38% of the cohort. Forty-seven per cent of patients discontinued their phenylalanine-restricted diet between 15 and 25 years of age. Of these, 73% remained off diet and 27% restarted a restricted diet at a mean 30 years of age. Fifteen per cent of the cohort had untreated PKU. Eleven per cent of patients who remained on a phenylalanine-restricted diet for 36 years received the optimum amount of prescribed amino acid supplements. Patients had a mean 12 general practitioner visits per year and one hospital outpatient visit annually, but phenylalanine levels were only measured once every 18 to 24 months. The mean NHS cost (at 2007/08 prices) of managing a PKU sufferer over the first 36 years of their life was estimated to range between £21 000 and £149 000, depending on the amount of prescribed nutrition they received. CONCLUSION: The findings suggest that the majority of patients with PKU were under-treated. The NHS cost of patient management should not be an obstacle to encouraging patients to remain on a restricted diet until further information becomes available about the long-term clinical impact of stopping such a diet. Nevertheless, patients require counselling and managed follow up regardless of the choices they make about their diet.

Modelling the cost-effectiveness of electric stimulation therapy in non-healing venous leg ulcers.

OBJECTIVE: To estimate the cost-effectiveness of using electric stimulation (ES) therapy (Accel-Heal) plus dressings and compression bandaging compared with dressings and compression bandaging alone in treating chronic, non-healing venous leg ulcers (VLUs) of >6 months' duration from the perspective of the National Health Service (NHS) in the UK. METHOD: A 5-month Markov model was constructed, depicting the management of a chronic, non-healing VLU of >6 months' duration. The model considers the decision by a clinician to continue with a patient's previous care plan (comprising dressings and compression bandaging) or treating with ES therapy plus dressings and compression bandaging. The model was used to estimate the relative cost-effectiveness of ES therapy at 2008-2009 prices. RESULTS: According to the model, 38% of VLUs are expected to heal within 5 months after starting ES therapy, with a further 57% expected to improve. This improvement in clinical outcome is expected to lead to a 6% health gain of 0.017 QALYs (from 0.299 to 0.316 QALYs) over 5 months. The model also showed that using ES therapy instead of continuing with a patient's previous care plan is expected to reduce the NHS cost of managing them by 15%, from £880 to £749, due in part to a 27% reduction in the requirement for nurse visits (from mean 49.0 to 35.9 visits per patient) over the first 5 months after the start of treatment. Hence, use of ES therapy was found to be a dominant treatment (improved outcome for less cost). CONCLUSION: Within the model's limitations, use of ES therapy potentially affords the NHS a cost-effective treatment, compared with patients remaining on their previous care plan in managing chronic, non-healing VLUs of >6 months' duration. However, this is dependant on the number of ES therapy units per treatment, the unit cost of the device, and the number of nurse visits required to manage patients in clinical practice. DECLARATION OF INTEREST: This study was sponsored by Synapse Microcurrent Ltd., manufacturers of Accel-Heal. The authors have no other conflicts of interest that are directly relevant to the content of this manuscript. In particular, Synapse Microcurrent Ltd. had no role in the study design, in the collection, analysis and interpretation of data, or in writing the manuscript.

Resource implications and budget impact of managing cow milk allergy in the UK.

AIM: To determine current treatment patterns for infants with cow milk allergy (CMA) and the associated resource implications and budget impact, from the perspective of the UK's National Health Service (NHS). METHODS: A computer-based model was constructed depicting current management of newly-diagnosed infants with CMA derived from patients suffering from this allergy in The Health Improvement Network (THIN) Database. The model spanned a period of 12 months following initial presentation to a general practitioner (GP) and was used to estimate the 12-monthly healthcare cost (at 2006/07 prices) of treating an annual cohort of 18,350 infants from when they initially present to their GP. RESULTS: Patients presenting with a combination of gastrointestinal and atopic symptoms accounted for 59% of all patients. From the initial GP visit for CMA it took a mean 2.2 months to be put on diet, although treatment varied according to presenting symptoms. A total of 60% of all infants were initially treated with soy, 18% with an extensively hydrolysed formula and 3% with an amino acid formula. A mean 9% of patients remained symptomatic on soy and 29% on an extensively hydrolysed formula. The total cost of managing CMA over the first 12 months following initial presentation to a GP was estimated to be £1,381 per patient and £25.6 million for an annual cohort of 18,350 infants. LIMITATIONS: Patients were not randomised to treatment and resource use was not collected prospectively. Nevertheless, 1,000 eligible patients have been included in the analysis, which should be a sufficiently large sample to accurately assess treatment patterns and healthcare resource use in actual clinical practice. The diagnosis of CMA may not be secure in all cases. Nevertheless, patients were diagnosed as having CMA by a clinician and have been managed by their GP as if they had CMA. CONCLUSION: CMA imposes a substantial burden on the NHS. Any strategy that improves healthcare delivery and thereby shortens time to treatment, time to diagnosis and time to symptom resolution should potentially decrease the burden this allergy imposes on the health service and release resources for alternative use.

Modelling the relative cost-effectiveness of amelogenin in non-healing venous leg ulcers.

OBJECTIVE: To assess the cost-effectiveness of using amelogenin plus compression bandaging versus compression bandaging alone in treating non-healing venous leg ulcers (VLUs) of over six months duration, from the perspective of the national health service in England. METHOD: A 12-month Markov model was constructed that depicted the management of a chronic, non-healing VLU of over six months duration. The model considers the decision by a clinician to treat a recalcitrant VLU with amelogenin plus compression bandaging or compression bandaging alone, and was used to estimate the relative cost-effectiveness of amelogenin plus compression bandaging at 20062007 prices. RESULTS: According to the model, 60% of all wounds treated with amelogenin plus compression bandaging are expected to heal within 12 months of the start of treatment compared with 41% of wounds treated with compression bandaging alone (p<0.01). Additionally, 23% of all amelogenin-treated wounds are expected to improve compared with 18% of wounds in the compression bandaging alone group. This difference in effectiveness between the two groups is expected to lead to a 7% improvement in health gain among amelogenin-treated patients when compared with those treated with compression bandaging alone (0.800 versus 0.746 QALYs; p<0.01) at 12 months after the start of treatment. Use of amelogenin is expected to lead a 10% reduction in NHS cost over 12 months from pound4,261 (95% CI: pound3,409; pound5,114) to pound3,816 (95% CI: pound3,227; pound4,405), due in part to a reduction in the requirement for nurse visits. Hence, amelogenin plus compression bandaging was found to be a dominant treatment. Moreover, use of amelogenin is expected to free-up NHS resources for alternative use within the system. CONCLUSION: Within the models limitations, amelogenin plus compression bandaging is expected to afford the NHS a cost-effective dressing compared with compression bandaging alone in the management of chronic non-healing VLUs of more than six months duration. DECLARATION OF INTEREST: This study was sponsored by Mölnlycke Heath Care. The authors have no other conflicts of interest that are directly relevant to the content of this manuscript.