RESUMO
BACKGROUND: Mirabegron, a first-in-class selective oral ß3-adrenoceptor agonist, has similar efficacy to most antimuscarinic agents and a lower incidence of dry mouth in patients with overactive bladder (OAB). OBJECTIVES: To evaluate the cost-effectiveness of mirabegron 50 mg compared with oral antimuscarinic agents in adults with OAB from a UK National Health Service perspective. METHODS: A Markov model including health states for symptom severity, treatment status, and adverse events was developed. Cycle length was 1 month, and the time horizon was 5 years. Antimuscarinic comparators were tolterodine extended release, solifenacin, fesoterodine, oxybutynin extended release and immediate release (IR), darifenacin, and trospium chloride modified release. Transition probabilities for symptom severity levels and adverse events were estimated from a mirabegron trial and a mixed treatment comparison. Estimates for other inputs were obtained from published literature or expert opinion. Quality-adjusted life-years (QALYs) and total health care costs, including costs of drug acquisition, physician visits, incontinence pad use, and botox injections, were modeled. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: Base-case incremental cost-effectiveness ratios ranged from £367 (vs. solifenacin 10 mg) to £15,593 (vs. oxybutynin IR 10 mg) per QALY gained. Probabilistic sensitivity analyses showed that at a willingness-to-pay threshold of £20,000/QALY gained, the probability of mirabegron 50 mg being cost-effective ranged from 70.2% versus oxybutynin IR 10 mg to 97.8% versus darifenacin 15 mg. A limitation of our analysis is the uncertainty due to the lack of direct comparisons of mirabegron with other agents; a mixed treatment comparison using rigorous methodology provided the data for the analysis, but the studies involved showed heterogeneity. CONCLUSIONS: Mirabegron 50 mg appears to be cost-effective compared with standard oral antimuscarinic agents for the treatment of adults with OAB from a UK National Health Service perspective.
Assuntos
Acetanilidas/economia , Acetanilidas/uso terapêutico , Agonistas de Receptores Adrenérgicos beta 3/economia , Agonistas de Receptores Adrenérgicos beta 3/uso terapêutico , Custos de Medicamentos , Recursos em Saúde/economia , Antagonistas Muscarínicos/economia , Antagonistas Muscarínicos/uso terapêutico , Tiazóis/economia , Tiazóis/uso terapêutico , Bexiga Urinária Hiperativa/tratamento farmacológico , Bexiga Urinária Hiperativa/economia , Acetanilidas/efeitos adversos , Agonistas de Receptores Adrenérgicos beta 3/efeitos adversos , Adulto , Teorema de Bayes , Pesquisa Comparativa da Efetividade , Simulação por Computador , Análise Custo-Benefício , Técnicas de Apoio para a Decisão , Recursos em Saúde/estatística & dados numéricos , Humanos , Cadeias de Markov , Modelos Econômicos , Antagonistas Muscarínicos/efeitos adversos , Seleção de Pacientes , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Índice de Gravidade de Doença , Medicina Estatal/economia , Tiazóis/efeitos adversos , Fatores de Tempo , Resultado do Tratamento , Reino Unido , Bexiga Urinária/efeitos dos fármacos , Bexiga Urinária/fisiopatologia , Bexiga Urinária Hiperativa/diagnóstico , Bexiga Urinária Hiperativa/fisiopatologiaRESUMO
BACKGROUND: Overactive bladder (OAB) is highly prevalent and is associated with considerable morbidity and reduced health-related quality of life. ß3-adrenergic receptor (ß3-AR) stimulation is a novel alternative to antimuscarinic therapy for OAB. OBJECTIVE: The objective of this analysis was to assess the cost effectiveness of the ß3-AR agonist mirabegron relative to tolterodine extended release (ER) in patients with OAB from a UK National Health Service (NHS) perspective. METHODS: A Markov model was developed to simulate the management, course of disease, and effect of complications in OAB patients over a period of 5 years. Transition probabilities for symptom severity levels and probabilities of adverse events were estimated from the results of the randomised, double-blind SCORPIO trial in 1,987 patients with OAB. Other model inputs were derived from the literature and on assumptions based on clinical experience. RESULTS: Total 5-year costs per patient were £1,645.62 for mirabegron 50 mg/day and £1,607.75 for tolterodine ER 4 mg/day. Mirabegron was associated with a gain of 0.009 quality-adjusted life-years (QALYs) with an additional cost of £37.88. The resulting incremental cost-effectiveness ratio (ICER) was £4,386/QALY gained. In deterministic sensitivity analyses in the general OAB population and several subgroups, ICERs remained below the generally accepted willingness-to-pay (WTP) threshold of £20,000/QALY gained. The probability of mirabegron 50 mg being cost effective relative to tolterodine ER 4 mg was 89.4 % at the same WTP threshold. CONCLUSIONS: Mirabegron 50 mg/day is likely to be cost effective compared with tolterodine ER 4 mg/day for adult patients with OAB from a UK NHS perspective.
Assuntos
Acetanilidas/economia , Compostos Benzidrílicos/economia , Análise Custo-Benefício , Cresóis/economia , Fenilpropanolamina/economia , Tiazóis/economia , Bexiga Urinária Hiperativa/tratamento farmacológico , Agentes Urológicos/economia , Acetanilidas/administração & dosagem , Acetanilidas/uso terapêutico , Adulto , Compostos Benzidrílicos/administração & dosagem , Compostos Benzidrílicos/uso terapêutico , Cresóis/administração & dosagem , Cresóis/uso terapêutico , Método Duplo-Cego , Humanos , Fenilpropanolamina/administração & dosagem , Fenilpropanolamina/uso terapêutico , Qualidade de Vida , Tiazóis/administração & dosagem , Tiazóis/uso terapêutico , Tartarato de Tolterodina , Reino Unido , Bexiga Urinária Hiperativa/fisiopatologia , Agentes Urológicos/administração & dosagem , Agentes Urológicos/uso terapêuticoRESUMO
OBJECTIVES: Fidaxomicin was non-inferior to vancomycin with respect to clinical cure rates in the treatment of Clostridium difficile infections (CDIs) in two Phase III trials, but was associated with significantly fewer recurrences than vancomycin. This economic analysis investigated the cost-effectiveness of fidaxomicin compared with vancomycin in patients with severe CDI and in patients with their first CDI recurrence. METHODS: A 1 year time horizon Markov model with seven health states was developed from the perspective of Scottish public healthcare providers. Model inputs for effectiveness, resource use, direct costs and utilities were obtained from published sources and a Scottish expert panel. The main model outcome was the incremental cost-effectiveness ratio (ICER), expressed as cost per quality-adjusted life year (QALY), for fidaxomicin versus vancomycin; ICERs were interpreted using willingness-to-pay thresholds of £20,000/QALY and £30,000/QALY. One-way and probabilistic sensitivity analyses were performed. RESULTS: Total costs were similar with fidaxomicin and vancomycin in patients with severe CDI (£14,515 and £14,344, respectively) and in patients with a first recurrence (£16,535 and £16,926, respectively). Improvements in clinical outcomes with fidaxomicin resulted in small QALY gains versus vancomycin (severe CDI, +0.010; patients with first recurrence, +0.019). Fidaxomicin was cost-effective in severe CDI (ICER £16,529/QALY) and dominant (i.e. more effective and less costly) in patients with a first recurrence. The probability that fidaxomicin was cost-effective at a willingness-to-pay threshold of £30,000/QALY was 60% for severe CDI and 68% in a first recurrence. CONCLUSIONS: Fidaxomicin is cost-effective in patients with severe CDI and in patients with a first CDI recurrence versus vancomycin.
Assuntos
Aminoglicosídeos/economia , Antibacterianos/economia , Clostridioides difficile , Infecções por Clostridium/economia , Análise Custo-Benefício/métodos , Vancomicina/economia , Aminoglicosídeos/farmacologia , Aminoglicosídeos/uso terapêutico , Antibacterianos/farmacologia , Antibacterianos/uso terapêutico , Clostridioides difficile/efeitos dos fármacos , Infecções por Clostridium/tratamento farmacológico , Fidaxomicina , Humanos , Cadeias de Markov , Vancomicina/farmacologia , Vancomicina/uso terapêuticoRESUMO
BACKGROUND: Nigeria has included a regulated community-based health insurance (CBHI) model within its National Health Insurance Scheme (NHIS). Uptake to date has been disappointing, however. The aim of this study is to review the present status of CBHI in SSA in general to highlight the issues that affect its successful integration within the NHIS of Nigeria and more widely in developing countries. METHODS: A literature survey using PubMed and EconLit was carried out to identify and review studies that report factors affecting implementation of CBHI in SSA with a focus on Nigeria. RESULTS: CBHI schemes with a variety of designs have been introduced across SSA but with generally disappointing results so far. Two exceptions are Ghana and Rwanda, both of which have introduced schemes with effective government control and support coupled with intensive implementation programmes. Poor support for CBHI is repeatedly linked elsewhere with failure to engage and account for the 'real world' needs of beneficiaries, lack of clear legislative and regulatory frameworks, inadequate financial support, and unrealistic enrolment requirements. Nigeria's CBHI-type schemes for the informal sectors of its NHIS have been set up under an appropriate legislative framework, but work is needed to eliminate regressive financing, to involve scheme members in the setting up and management of programmes, to inform and educate more effectively, to eliminate lack of confidence in the schemes, and to address inequity in provision. Targeted subsidies should also be considered. CONCLUSIONS: Disappointing uptake of CBHI-type NHIS elements in Nigeria can be addressed through closer integration of informal and formal programmes under the NHIS umbrella, with increasing involvement of beneficiaries in scheme design and management, improved communication and education, and targeted financial assistance.
Assuntos
Acessibilidade aos Serviços de Saúde , Seguro Saúde , Programas Nacionais de Saúde , Avaliação de Programas e Projetos de Saúde , Necessidades e Demandas de Serviços de Saúde , Humanos , NigériaRESUMO
BACKGROUND: Systemic Candida infections (SCI) occur predominantly in intensive care unit patients and are a common cause of morbidity and mortality. Recently, changes in Candida epidemiology with an increasing prevalence of SCI caused by Candida non-albicans species have been reported. Resistance to fluconazole and azoles in general is not uncommon for non-albicans species. Despite guidelines recommending initial treatment with broad-spectrum antifungals such as echinocandins with subsequent switch to fluconazole if isolates are sensitive (de-escalation strategy), fluconazole is still the preferred first-line antifungal (escalation) in many clinical practice settings. After diagnosis of the pathogen, the initial therapy with fluconazole is switched to a broad-spectrum antifungal if a non-albicans is identified. METHODS: The cost-effectiveness of initial treatment with micafungin (de-escalation) vs fluconazole (escalation) in patients with SCI was estimated using decision analysis based on clinical and microbiological data from pertinent studies. The model horizon was 42 days, and was extrapolated to cover a lifetime horizon. All costs were analyzed from the UK NHS perspective. Several assumptions were taken to address uncertainties; the limitations of these assumptions are discussed in the article. RESULTS: In patients with fluconazole-resistant isolates, initial treatment with micafungin avoids 30% more deaths and successfully treats 23% more patients than initial treatment with fluconazole, with cost savings of £1621 per treated patient. In the overall SCI population, de-escalation results in 1.2% fewer deaths at a marginal cost of £740 per patient. Over a lifetime horizon, the incremental cost-effectiveness of de-escalation vs escalation was £15,522 per life-year and £25,673 per QALY. CONCLUSIONS: De-escalation from micafungin may improve clinical outcomes and overall survival, particularly among patients with fluconazole-resistant Candida strains. De-escalation from initial treatment with micafungin is a cost-effective alternative to escalation from a UK NHS perspective, with a differential cost per QALY below the 'willingness-to-pay' threshold of £30,000.
Assuntos
Antifúngicos/economia , Antifúngicos/uso terapêutico , Candidíase/tratamento farmacológico , Equinocandinas/economia , Equinocandinas/uso terapêutico , Lipopeptídeos/economia , Lipopeptídeos/uso terapêutico , Antifúngicos/administração & dosagem , Candidíase/economia , Candidíase/mortalidade , Análise Custo-Benefício , Técnicas de Apoio para a Decisão , Equinocandinas/administração & dosagem , Fluconazol/economia , Fluconazol/uso terapêutico , Serviços de Saúde/economia , Serviços de Saúde/estatística & dados numéricos , Humanos , Expectativa de Vida , Lipopeptídeos/administração & dosagem , Micafungina , Testes de Sensibilidade Microbiana , Anos de Vida Ajustados por Qualidade de VidaRESUMO
BACKGROUND: Nigeria and Ghana have recently introduced a National Health Insurance Scheme (NHIS) with the aim of moving towards universal health care using more equitable financing mechanisms. This study compares health and economic indicators, describes the structure of each country's NHIS within the wider healthcare system, and analyses impacts on equity in financing and access to health care. METHODS: The World Bank and other sources were used to provide comparative health and economic data. Pubmed, Embase and EconLit were searched to locate studies providing descriptions of each NHIS and empirical evidence regarding equity in financing and access to health care. A diagrammatical representation of revenue-raising, pooling, purchasing and provision was produced in order to analyse the two countries' systems. RESULTS: Over the period 2000-2010, Ghana maintained a marked advantage in life expectancy, infant mortality, under-5 year mortality, and has a lower burden of major diseases. Health care expenditure is about 5% of GDP in both countries but public expenditure in 2010 was 38% of total expenditure in Nigeria and 60% in Ghana. Financing and access are less equitable in Nigeria as, inter alia, private out-of-pocket expenditure has fallen from 80% to 66% of total spending in Ghana since the introduction of its NHIS but has remained at over 90% in Nigeria; NHIS membership in Nigeria and Ghana is approximately 3.5% and 65%, respectively; Nigeria offers a variable benefits package depending on membership category while Ghana has uniform benefits across all beneficiaries. Both countries exhibit improvements in equity but there is a pro-rich and pro-urban bias in membership. CONCLUSIONS: Major health indicators are more favourable in Ghana and overall equity in financing and access are weaker in Nigeria. Nigeria is taking steps to expand NHIS membership and has potential to expand its public spending to achieve greater equity. However, heavy burdens of poverty, disease and remote settings make this a substantial challenge. Ghana's relative success has to be tempered by the high number of exemptions through taxation and the threat of moral hazard. The results and methods are anticipated to be informative for policy makers and researchers in both countries and other developing countries more widely.
Assuntos
Acessibilidade aos Serviços de Saúde/normas , Disparidades em Assistência à Saúde , Seguro Saúde/organização & administração , Programas Nacionais de Saúde/organização & administração , Gana , Gastos em Saúde , Acessibilidade aos Serviços de Saúde/economia , Acessibilidade aos Serviços de Saúde/organização & administração , Humanos , Expectativa de Vida , Programas Nacionais de Saúde/economia , NigériaRESUMO
Healthcare decision makers who determine funding for new medical technologies depend on manufacturers to provide evidence of the technology's efficacy, safety and cost-effectiveness. Constrained budgets and increasing reliance on formal health technology assessment (HTA) have created an abundance of external hurdles that manufacturers must navigate to ensure successful product commercialization. These demands have pushed pharmaceutical companies to adjust their internal structures to coordinate generation of appropriate evidence. In this article we summarize internal and external opportunities for manufacturers to establish a foundation of evidence for successful market access, starting in Phase I of development and continuing throughout the post-approval product lifecycle.
Assuntos
Biofarmácia/economia , Pesquisa Comparativa da Efetividade , Descoberta de Drogas/economia , Reembolso de Seguro de Saúde , Marketing/economia , Avaliação de Resultados em Cuidados de Saúde/economia , Avaliação da Tecnologia Biomédica/economia , Análise Custo-Benefício , Humanos , Ensaios Clínicos Controlados Aleatórios como AssuntoRESUMO
BACKGROUND: The objective of this study was to model the economic impact of optimal dosing of immune tolerance induction (ITI) in haemophilia patients with inhibitors. Evidence based research suggests that, in the right patient population characterised by ITI risk status, the use of high-dose ITI regimen in 'poor risk' patients, and the low-dose regimen in 'good risk' patients, would be the cost effective strategy. The model also explored the impact of anamnestic response (AR), a phenomenon which worsens patients' ITI risks. METHOD: A cost-minimisation technique was used to compare the cost of managing inhibitor patients whose bleeds were managed pre-ITI with an immunogenic bypassing agent, activated prothrombin complex concentrate (APCC), with patients previously on a non-immunogenic product, recombinant activated factor VII (rFVIIa). Patients were subsequently offered a low-dose or high-dose ITI regime depending on their ITI risk status. The study perspective was that of the United Kingdom NHS, hence, all resources used were based on UK costs. RESULTS: The model estimated the mean cost of managing inhibitor patients from detection of titres through ITI to be 959,250.39 pound sterling and 770,834.17 pound sterling in the APCC and rFVIIa treatment options, respectively. Meanwhile, the costs per effectively tolerised patients were 1,505,279 pound sterling and 1,196,706 pound sterling for APCC and rFVIIa treated patients, respectively. Of the incremental cost in the APCC-treated patients in the model, 129,367 pound sterling (68%) represents additional ITI cost attributable to anamnestic response to earlier treatment with an immunogenic bypassing agent (APCC). CONCLUSION: The study concludes that decreasing factor VIII usage during ITI, through the identification and management of 'good risk' ITI patients with low-dose protocol, while managing 'poor risk' patients with high-dose regimen, will significantly lower the cost of ITI. Furthermore, avoiding AR prior to ITI by using non-immunogenic bypassing agents to manage spontaneous bleeds also has the potential for significant cost savings.
Assuntos
Fator VIIa/uso terapêutico , Custos de Cuidados de Saúde , Hemofilia A/tratamento farmacológico , Tolerância Imunológica , Protrombina/uso terapêutico , Hemofilia A/imunologia , Humanos , Proteínas Recombinantes/uso terapêuticoRESUMO
OBJECTIVE(S): This study aimed to assess the resource utilisation, health benefits and cost-effectiveness of intra-detrusor injections of botulinum neurotoxin-A (BoNT/A) in patients with overactive bladder (OAB). METHODS: 101 patients with urodynamically-proven detrusor overactivity of either neurogenic (NDO; n = 63) or idiopathic (IDO; n = 38) origin received intra-detrusor injections of 200-300 units of BoNT/A in 20-30 ml saline as part of a research protocol. Twenty-nine patients received repeat injections after 7-26 months. Symptom severity and urodynamic parameters were assessed at 0, 4 and 16 weeks. The cost of therapy was quantified based on the NHS resources used by typical patients and was used to calculate the cost-effectiveness of BoNT/A compared with standard care from the perspective of the UK NHS. RESULTS: In an intent-to-treat analysis, 82% of patients showed a 25% or greater improvement in at least two out of five parameters (urinary frequency, urgency, urgency incontinence episodes, maximum cystometric capacity and maximum detrusor pressure) four weeks after treatment, reducing to 65% after 16 weeks. A 50% or greater improvement in the frequency of micturition, urgency or urgency incontinence was seen in 73% of patients at four weeks and 54% at 16 weeks. There were no significant differences between IDO and NDO patients in the proportion meeting these endpoints. Therapy cost pounds 826 per patient, with a cost-effectiveness ratio of pounds 617 per patient-year with > or = 25% clinical improvement. CONCLUSION(S): This study demonstrates that intra-detrusor BoNT/A is an effective treatment for OAB that is highly likely to be cost-effective in both idiopathic and neurogenic disease.
Assuntos
Toxinas Botulínicas Tipo A/uso terapêutico , Fármacos Neuromusculares/uso terapêutico , Incontinência Urinária/tratamento farmacológico , Adulto , Idoso , Idoso de 80 Anos ou mais , Toxinas Botulínicas Tipo A/administração & dosagem , Toxinas Botulínicas Tipo A/economia , Análise Custo-Benefício , Feminino , Humanos , Injeções Intramusculares , Masculino , Pessoa de Meia-Idade , Músculo Liso/efeitos dos fármacos , Reino Unido , Incontinência Urinária/economiaRESUMO
The National Institute for Health and Clinical Excellence (NICE) issues mandatory guidance on health technologies to the UK NHS, based on clinical evidence, cost-effectiveness and other considerations. However, the exact factors considered, their relative importance and tradeoffs between them are not made explicit. Previous research modelled NICE decisions as a binary choice (accept/reject) dependent on cost-effectiveness, amongst other variables. This paper proposes and tests an alternative model of decision-making that may better represent the "yes, but..." nature of many NICE decisions. Decisions were categorised as "recommended for routine use", "recommended for restricted use" or "not recommended". The NICE appraisal process was modelled as a single decision between the three categories. Multinomial logistic regression techniques were used to evaluate the impact of: quantity/quality of clinical evidence; cost-effectiveness; decision date; existence of alternative treatments; budget impact; technology type. Results suggest that interventions supported by more randomised trials are more likely to be recommended and endorsed for routine use. Higher cost-effectiveness ratios increased the likelihood of interventions being rejected rather than recommended for restricted use but did not significantly affect the decision between routine and restricted use. Pharmaceuticals, interventions appraised early in the NICE programme and those with more systematic reviews were also less likely to be rejected, while patient group submissions made a recommendation for routine rather than restricted use more likely. The presence of factors affecting the decision between routine and restricted use but not that between routine use and rejection suggests that modelling these three outcomes reflects NICE decision-making more closely than binary-choice analyses.