Cost-effectiveness of abobotulinumtoxinA plus best supportive care compared with best supportive care alone for early treatment of adult lower limb spasticity following an acute event.

OBJECTIVES: Spasticity is an incurable chronic condition, and patients with spasticity frequently experience symptoms such as muscle stiffness, restricted mobility, fatigue, spasms, and pain. The study objective was to assess the cost-effectiveness of abobotulinumtoxinA plus best supportive care compared with best supportive care alone for the early treatment of adult lower limb spasticity following an acute event (e.g. stroke or traumatic brain injury), from an Australian payer perspective. METHODS: Using clinical data from published pivotal trials, an economic model based on a Markov model was developed to capture changes in treatment costs, healthcare resource use costs, functional outcomes, and health-related quality of life over a lifetime horizon. Scenario analyses and a probabilistic sensitivity analysis were conducted to explore the uncertainty in the model parameters and assumptions used in the base case. RESULTS: AbobotulinumtoxinA plus best supportive care was cost-effective versus best supportive care, yielding an incremental cost-effectiveness ratio of $35,721 per quality-adjusted life year gained. Sensitivity analyses confirm the robustness of the base case, with most results remaining below the commonly acceptable cost-effectiveness willingness-to-pay threshold of $75,000 per quality-adjusted life year for cost-effectiveness in Australia. Inputs and assumptions that produced the top four highest incremental cost-effectiveness ratios include the application of different health resource utilisation source, short time horizon, unweighted regression analyses to determine regression probabilities, and no stopping rule. AbobotulinumtoxinA plus best supportive care has a 74% probability of being cost-effective compared with best supportive care alone at the willingness to pay threshold. CONCLUSION: AbobotulinumtoxinA plus best supportive care treatment is cost-effective in Australia for the management of adult lower limb spasticity in patients treated within 2 years of an acute event.

Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in England.

INTRODUCTION: We sought to evaluate the cost-effectiveness of newborn screening (NBS) versus no NBS for 5q spinal muscular atrophy (SMA) in England. METHODS: A cost-utility analysis using a combination of decision tree and Markov model structures was developed to estimate the lifetime health effects and costs of NBS for SMA, compared with no NBS, from the perspective of the National Health Service (NHS) in England. A decision tree was designed to capture NBS outcomes, and Markov modeling was used to project long-term health outcomes and costs for each patient group following diagnosis. Model inputs were based on existing literature, local data, and expert opinion. Sensitivity and scenario analyses were conducted to assess the robustness of the model and the validity of the results. RESULTS: The introduction of NBS for SMA in England is estimated to identify approximately 56 (96% of cases) infants with SMA per year. Base-case results indicate that NBS is dominant (less costly and more effective) than a scenario without NBS, with a yearly cohort of newborns accruing incremental savings of £62,191,531 and an estimated gain in quality-adjusted life-years of 529 years over their lifetime. Deterministic and probabilistic sensitivity analyses demonstrated the robustness of the base-case results. CONCLUSIONS: NBS improves health outcomes for patients with SMA and is less costly compared with no screening; therefore, it is a cost-effective use of resources from the perspective of the NHS in England.

The cost-effectiveness of isavuconazole compared to the standard of care in the treatment of patients with invasive fungal infection prior to differential pathogen diagnosis in the United Kingdom.

Aims: To estimate the cost-effectiveness of isavuconazole compared with the standard of care, voriconazole, for the treatment of patients with invasive fungal infection disease when differential diagnosis of the causative pathogen has not yet been achieved at treatment initiation.Materials and methods: The economic model was developed from the perspective of the UK National Health Service (NHS) and used a decision-tree approach to reflect real-world treatment of patients with invasive fungal infection (IFI) prior to differential pathogen diagnosis. It was assumed that 7.8% of patients with IFI prior to differential pathogen diagnosis at treatment initiation actually had mucormycosis, and confirmation of pathogen identification was achieved for 50% of all patients during treatment. To extrapolate to a lifetime horizon, the model considered expected survival based on the patients' underlying condition. The model estimated the incremental costs (costs of drugs, laboratory analysis, hospitalization, and management of adverse events) and clinical outcomes (life-years (LYs) and quality-adjusted life-years (QALYs)) of first-line treatment with isavuconazole compared with voriconazole. The robustness of the results was assessed by conducting deterministic and probabilistic sensitivity analyses.Results: Isavuconazole delivered 0.48 more LYs and 0.39 more QALYs per patient at an incremental cost of £3,228, compared with voriconazole in the treatment of patients with IFI prior to differential pathogen diagnosis. This equates to an incremental cost-effectiveness ratio (ICER) of £8,242 per additional QALY gained and £6,759 per LY gained. These results were driven by a lack of efficacy of voriconazole in mucormycosis. Results were most sensitive to the mortality of IA patients and treatment durations.Conclusions: At a willingness to pay (WTP) threshold of £30,000 per additional QALY, the use of isavuconazole for the treatment of patients with IFI prior to differential pathogen diagnosis in the UK can be considered a cost-effective allocation of healthcare resources compared with voriconazole.

Annual direct and indirect costs attributable to nocturia in Germany, Sweden, and the UK.

OBJECTIVE: Our aim was to estimate the prevalence-based cost of illness imposed by nocturia (≥2 nocturnal voids per night) in Germany, Sweden, and the UK in an average year. METHODS: Information obtained from a systematic review of published literature and clinicians was used to construct an algorithm depicting the management of nocturia in these three countries. This enabled an estimation of (1) annual levels of healthcare resource use, (2) annual cost of healthcare resource use, and (3) annual societal cost arising from presenteeism and absenteeism attributable to nocturia in each country. RESULTS: In an average year, there are an estimated 12.5, 1.2, and 8.6 million patients ≥20 years of age with nocturia in Germany, Sweden, and the UK, respectively. In an average year in each country, respectively, these patients were estimated to have 13.8, 1.4, and 10.0 million visits to a family practitioner or specialist, ~91,000, 9000, and 63,000 hospital admissions attributable to nocturia and 216,000, 19,000, and 130,000 subjects were estimated to incur a fracture resulting from nocturia. The annual direct cost of healthcare resource use attributable to managing nocturia was estimated to be approximately €2.32 billion in Germany, 5.11 billion kr (€0.54 billion) in Sweden, and £1.35 billion (€1.77 billion) in the UK. The annual indirect societal cost arising from both presenteeism and absenteeism was estimated to be approximately €20.76 billion in Germany and 19.65 billion kr (€2.10 billion) in Sweden. In addition, in the UK, the annual indirect cost due to absenteeism was an estimated £4.32 billion (€5.64 billion). CONCLUSIONS: Nocturia appears to impose a substantial socioeconomic burden in all three countries. Clinical and economic benefits could accrue from an increased awareness of the impact that nocturia imposes on patients, health services, and society as a whole.

Health economic burden that different wound types impose on the UK's National Health Service.

The aim of this study was to estimate the patterns of care and annual levels of health care resource use attributable to the management of different wound types by the UK's National Health Service (NHS) in 2012/2013 and the annual costs incurred by the NHS in managing them. This was a retrospective cohort analysis of the records of 2000 patients in The Health Improvement Network (THIN) Database. Patients' characteristics, wound-related health outcomes and all health care resource use were quantified, and the total NHS cost of patient management was estimated at 2013/2014 prices. The NHS managed an estimated 2·2 million patients with a wound during 2012/2013. Patients were predominantly managed in the community by general practitioners (GPs) and nurses. The annual NHS cost varied between £1·94 billion for managing 731 000 leg ulcers and £89·6 million for managing 87 000 burns, and associated comorbidities. Sixty-one percent of all wounds were shown to heal in an average year. Resource use associated with managing the unhealed wounds was substantially greater than that of managing the healed wounds (e.g. 20% more practice nurse visits, 104% more community nurse visits). Consequently, the annual cost of managing wounds that healed in the study period was estimated to be £2·1 billion compared with £3·2 billion for the 39% of wounds that did not heal within the study year. Within the study period, the cost per healed wound ranged from £698 to £3998 per patient and that of an unhealed wound ranged from £1719 to £5976 per patient. Hence, the patient care cost of an unhealed wound was a mean 135% more than that of a healed wound. Real-world evidence highlights the substantial burden that wounds impose on the NHS in an average year. Clinical and economic benefits to both patients and the NHS could accrue from strategies that focus on (a) wound prevention, (b) accurate diagnosis and (c) improving wound-healing rates.

Relative cost-effectiveness of using an extensively hydrolyzed casein formula containing the probiotic Lactobacillus rhamnosus GG in managing infants with cow's milk allergy in Poland.

OBJECTIVE: To estimate the cost-effectiveness of using an extensively hydrolyzed casein formula (eHCF) containing the probiotic Lactobacillus rhamnosus GG (eHCF + LGG; Nutramigen LGG) as an initial treatment for cow's milk allergy compared with eHCF alone and amino acid formulas (AAF) in Poland from the perspective of the Polish National Health Fund (Narodowy Fundusz Zdrowia [NFZ]) and parents. METHODS: Decision modeling was used to estimate the probability of cow's milk allergic infants developing tolerance to cow's milk by 18 months. The model also estimated the cost to the NFZ and parents (Polish Zloty [PLN] at 2013-2014 prices) for managing infants over 18 months after starting one of the formulas as well as the relative cost-effectiveness of each of the formulas. RESULTS: The probability of developing tolerance to cow's milk by 18 months was higher among infants who were fed eHCF + LGG (0.82) compared with those fed eHCF alone (0.53) or an AAF (0.22). An infant who is initially managed with eHCF + LGG is expected to consume fewer health care resources than infants managed with the other formulas. Hence, the estimated total health care cost incurred by the NFZ for initially feeding infants with eHCF + LGG (PLN 5,693) was less than that of feeding infants with eHCF alone (PLN 7,749) or an AAF (PLN 24,333). However, the total cost incurred by parents for initially feeding infants with an AAF (PLN 815) was marginally less than that of feeding with eHCF + LGG (PLN 993), which was less than that of feeding with eHCF alone (PLN 1,226). CONCLUSION: Using eHCF + LGG instead of eHCF alone or an AAF for first-line management of newly diagnosed infants with cow's milk allergy affords a cost-effective use of NFZ-funded resources, since it improves outcome for less cost. Whether eHCF + LGG would be viewed as being cost-effective by parents is dependent on their willingness to pay an additional cost for additional tolerance acquisition to cow's milk.

Healthcare costs and outcomes of managing ß-thalassemia major over 50 years in the United Kingdom.

BACKGROUND: The objective was to estimate the incidence-based costs of treating ß-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. STUDY DESIGN AND METHODS: 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. RESULTS: 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. CONCLUSION: 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.

Relative cost-effectiveness of using an extensively hydrolyzed casein formula containing the probiotic Lactobacillus rhamnosus GG in managing infants with cow's milk allergy in Spain.

OBJECTIVE: To estimate the cost-effectiveness of using an extensively hydrolyzed casein formula containing the probiotic Lactobacillus rhamnosus GG (eHCF + LGG; Nutramigen LGG) as a first-line management for cow's milk allergy compared with eHCF alone, and amino acid formulae in Spain, from the perspective of the Spanish National Health Service (SNS). METHODS: Decision modeling was used to estimate the probability of immunoglobulin E (IgE)-mediated and non-IgE-mediated allergic infants developing tolerance to cow's milk by 18 months. The models also estimated the SNS cost (at 2012/2013 prices) of managing infants over 18 months after starting a formula as well as the relative cost-effectiveness of each of the formulae. RESULTS: The probability of developing tolerance to cow's milk by 18 months was higher among infants with either IgE-mediated or non-IgE-mediated allergy who were fed eHCF + LGG compared with those fed one of the other formulae. The total health care cost of initially feeding infants with eHCF + LGG was less than that of feeding infants with one of the other formulae. Hence, eHCF + LGG affords the greatest value for money to the SNS for managing both IgE-mediated and non-IgE-mediated cow's milk allergy. CONCLUSION: Using eHCF + LGG instead of eHCF alone or amino acid formulae for first-line management of newly-diagnosed infants with cow's milk allergy affords a cost-effective use of publicly funded resources because it improves outcome for less cost. A randomized controlled study showing faster tolerance development in children receiving a probiotic-containing formula is required before this conclusion can be confirmed.

Health economic burden that wounds impose on the National Health Service in the UK.

OBJECTIVE: To estimate the prevalence of wounds managed by the UK's National Health Service (NHS) in 2012/2013 and the annual levels of healthcare resource use attributable to their management and corresponding costs. METHODS: This was a retrospective cohort analysis of the records of patients in The Health Improvement Network (THIN) Database. Records of 1000 adult patients who had a wound in 2012/2013 (cases) were randomly selected and matched with 1000 patients with no history of a wound (controls). Patients' characteristics, wound-related health outcomes and all healthcare resource use were quantified and the total NHS cost of patient management was estimated at 2013/2014 prices. RESULTS: Patients' mean age was 69.0 years and 45% were male. 76% of patients presented with a new wound in the study year and 61% of wounds healed during the study year. Nutritional deficiency (OR 0.53; p<0.001) and diabetes (OR 0.65; p<0.001) were independent risk factors for non-healing. There were an estimated 2.2 million wounds managed by the NHS in 2012/2013. Annual levels of resource use attributable to managing these wounds and associated comorbidities included 18.6 million practice nurse visits, 10.9 million community nurse visits, 7.7 million GP visits and 3.4 million hospital outpatient visits. The annual NHS cost of managing these wounds and associated comorbidities was £5.3 billion. This was reduced to between £5.1 and £4.5 billion after adjusting for comorbidities. CONCLUSIONS: Real world evidence highlights wound management is predominantly a nurse-led discipline. Approximately 30% of wounds lacked a differential diagnosis, indicative of practical difficulties experienced by non-specialist clinicians. Wounds impose a substantial health economic burden on the UK's NHS, comparable to that of managing obesity (£5.0 billion). Clinical and economic benefits could accrue from improved systems of care and an increased awareness of the impact that wounds impose on patients and the NHS.