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The development of disease-modifying therapies (DMTs) for Alzheimer's disease (AD) has progressed over the last decade, and the first-ever therapies with potential to slow the progression of disease are approved in the United States. AD DMTs could provide life-changing opportunities for people living with this disease, as well as for their caregivers. They could also ease some of the immense societal and economic burden of dementia. However, AD DMTs also come with major challenges due to the large unmet medical need, high prevalence of AD, new costs related to diagnosis, treatment and monitoring, and uncertainty in the therapies' actual clinical value. This perspective article discusses, from the broad perspective of various health systems and stakeholders, how we can overcome these challenges and improve society's readiness for AD DMTs. We propose that innovative payment models such as performance-based payments, in combination with learning healthcare systems, could be the way forward to enable timely patient access to treatments, improve accuracy of cost-effectiveness evaluations and overcome budgetary barriers. Other important considerations include the need for identification of key drivers of patient value, the relevance of different economic perspectives (i.e. healthcare vs. societal) and ethical questions in terms of treatment eligibility criteria.
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Doença de Alzheimer , Humanos , Estados Unidos , Doença de Alzheimer/diagnóstico , Doença de Alzheimer/tratamento farmacológico , Análise Custo-Benefício , Atenção à SaúdeRESUMO
In the context of pharmaceutical care, policy-makers repeatedly facethe challenge of balancing patient access to effective medicines withaffordability and rising costs. With the aim of guiding the health policydiscourse towards questions that are important to actual and potential patients,this study investigates a broad range of regulatory measures, spanningmarketing authorization to generic substitution and resulting price levels in asample of 16 European health systems (Austria, Belgium, Denmark, England,Finland, France, Germany, Greece, Ireland, Italy, the Netherlands, Poland,Portugal, Scotland, Spain and Sweden).All countries employ a mix of regulatory mechanisms to containpharmaceutical expenditure and ensure quality and efficiency in pharmaceuticalcare, albeit with varying configurations and rigour. This variation alsoinfluences the extent of publicly financed pharmaceutical costs. Overall,observed differences in pharmaceutical expenditure should be interpreted inconjunction with the differing volume and composition of consumption andprice levels, as well as dispensation practices and their impact on measurementof pharmaceutical costs.No definitive evidence has yet been produced on the effects of differentcost-containment measures on patient outcomes. Depending on the foremostpolicy concerns in each country, different levers will have to be used to enablethe delivery of appropriate care at affordable prices.
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Atenção à Saúde , Estudo de Avaliação , Financiamento da Assistência à Saúde , Reforma dos Serviços de Saúde , Planos de Sistemas de Saúde , Assistência FarmacêuticaRESUMO
BACKGROUND: Offering patients in oncology trials the opportunity to cross over to active treatment at disease progression is a common strategy to address ethical issues associated with placebo controls but may lead to statistical challenges in the analysis of overall survival and cost-effectiveness because crossover leads to information loss and dilution of comparative clinical efficacy. OBJECTIVES: We provide an overview of how to address crossover, implications for risk-effect estimates of survival (hazard ratios) and cost-effectiveness, and how this influences decisions of reimbursement agencies. Two case studies using data from two phase III sunitinib oncology trials are used as illustration. METHODS: We reviewed the literature on statistical methods for adjusting for crossover and recent health technology assessment decisions in oncology. RESULTS: We show that for a trial with a high proportion of crossover from the control arm to the investigational arm, the choice of the statistical method greatly affects treatment-effect estimates and cost-effectiveness because the range of relative mortality risk for active treatment versus control is broad. With relatively frequent crossover, one should consider either the inverse probability of censoring weighting or the rank-preserving structural failure time model to minimize potential bias, with choice dependent on crossover characteristics, trial size, and available data. A large proportion of crossover favors the rank-preserving structural failure time model, while large sample size and abundant information about confounding factors favors the inverse probability of censoring weighting model. When crossover is very infrequent, methods yield similar results. CONCLUSIONS: Failure to correct for crossover may lead to suboptimal decisions by pricing and reimbursement authorities, thereby limiting an effective drug's potential.
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Ensaios Clínicos Fase III como Assunto/economia , Ensaios Clínicos Fase III como Assunto/mortalidade , Ensaios Clínicos Controlados Aleatórios como Assunto/economia , Ensaios Clínicos Controlados Aleatórios como Assunto/mortalidade , Antineoplásicos/economia , Antineoplásicos/uso terapêutico , Análise Custo-Benefício , Estudos Cross-Over , Humanos , Neoplasias/tratamento farmacológico , Neoplasias/economia , Neoplasias/mortalidade , Taxa de Sobrevida/tendênciasRESUMO
OBJECTIVE: To determine effectiveness and cost-effectiveness over a one-year time horizon of pharmacological first line treatment in primary care for patients with moderate to severe depression. DESIGN: A multiple treatment comparison meta-analysis was employed to determine the relative efficacy in terms of remission of 10 antidepressants (citalopram, duloxetine escitalopram, fluoxetine, fluvoxamine mirtazapine, paroxetine, reboxetine, sertraline and venlafaxine). The estimated remission rates were then applied in a decision-analytic model in order to estimate costs and quality of life with different treatments at one year. DATA SOURCES: Meta-analyses of remission rates from randomised controlled trials, and cost and quality-of-life data from published sources. RESULTS: The most favourable pharmacological treatment in terms of remission was escitalopram with an 8- to 12-week probability of remission of 0.47. Despite a high acquisition cost, this clinical effectiveness translated into escitalopram being both more effective and having a lower total cost than all other comparators from a societal perspective. From a healthcare perspective, the cost per QALY of escitalopram was 3732 compared with venlafaxine. CONCLUSION: Of the investigated antidepressants, escitalopram has the highest probability of remission and is the most effective and cost-effective pharmacological treatment in a primary care setting, when evaluated over a one year time-horizon. Small differences in remission rates may be important when assessing costs and cost-effectiveness of antidepressants.
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Antidepressivos/economia , Antidepressivos/uso terapêutico , Transtorno Depressivo/tratamento farmacológico , Transtorno Depressivo/economia , Modelos Econômicos , Atenção Primária à Saúde/economia , Análise Custo-Benefício , Humanos , Resultado do TratamentoRESUMO
Objective A randomised controlled study performed from 2007 to 2008 showed beneficial effects of a composite clinical pharmacist service as regards a simple health status instrument. The present study aimed to evaluate if the intervention was cost-effective when evaluated in a decision-theoretic model. Design A piggyback cost-effectiveness analysis from the healthcare perspective. Setting Two internal medicine wards at Sahlgrenska University Hospital, Göteborg, Sweden. Participants Of 345 patients (61% women; median age: 82 years; 181 control and 164 intervention patients), 240 patients (62% women, 82 years; 124 control and 116 intervention patients) had EuroQol-5 dimensions (EQ-5D) utility scores at baseline and at 6-month follow-up. Outcome measures Costs during a 6-month follow-up period in all patients and incremental cost-effectiveness ratio per quality-adjusted life-year (QALY) in patients with EQ-5D utility scores. Inpatient and outpatient care was extracted from the VEGA database. Drug costs were extracted from the Swedish Prescribed Drug Register. A probabilistic analysis was performed to characterise uncertainty in the cost-effectiveness model. Results No significant difference in costs between the randomisation groups was found; the mean total costs per individual±SD, intervention costs included, were 10â748±13â799 (intervention patients) and 10â344±14â728 (control patients) (p=0.79). For patients in the cost-effectiveness analysis, the corresponding costs were 10â912±13â999 and 9290±12â885. Intervention patients gained an additional 0.0051 QALYs (unadjusted) and 0.0035 QALYs (adjusted for baseline EQ-5D utility score). These figures result in an incremental cost-effectiveness ratio of 316â243 per unadjusted QALY and 463â371 per adjusted QALY. The probabilistic uncertainty analysis revealed that, at a willingness-to-pay of 50â000/QALY, the probability that the intervention was cost-effective was approximately 0.2. Conclusions The present study reveals that an intervention designed like this one is probably not cost-effective. The study thus illustrates that the complexity of healthcare requires thorough health economics evaluations rather than simplistic interpretation of data.
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This study assessed the quality of health economic documentation submitted to the Pharmaceutical Benefits Board (PBB) in Sweden. Two different instruments were used in the evaluation: the PBB checklist, which was constructed by the authors from the PBB guidelines for health economic evaluations, and the QHES, a validated quality assessment instrument. Some areas that seem especially problematic, or where the quality was particularly low are identified and discussed. Also, we present the cost per quality-adjusted life-year that the companies have presented and how this related to the PBBs decisions.
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Análise Custo-Benefício , Farmacoeconomia , Humanos , Anos de Vida Ajustados por Qualidade de Vida , SuéciaRESUMO
BACKGROUND: Periodontal scaling procedures commonly require some kind of anesthesia. From the patient's perspective, the choice of anesthetic method is a trade-off between the degree of anesthesia and accepting the side effects. The present study evaluates the preferences for a novel non-injection anesthetic product (a gel, containing lidocaine 25 mg/g plus prilocaine 25 mg/g and thermosetting agents) versus injection anesthesia (lidocaine 2% adrenaline) in conjunction with scaling and/or root planing (SRP). METHODS: In a multicenter, crossover, randomized, open study patients were asked, after they had experienced both products, if they preferred anesthetic gel or injection anesthesia. In addition, the adequacy of anesthesia and occurrence of post-procedure problems were assessed. The patients were also asked about their willingness to return if they were offered anesthetic gel at their next visit and their maximum willingness to pay (WTP) for this option. RESULTS: One-hundred seventy (170) patients at eight centers in Belgium were included in the study. There were 157 per protocol (PP) patients. A vast majority of the PP patients (70%) preferred the anesthetic gel to injection anesthesia (22%). The most common reason was less post-procedure numbness. Eighty percent (80%) of the patients expressed satisfactory anesthesia with the gel and 96% with injection anesthesia (P <0.001). Post-procedure problems were significantly less with the gel than with injection (P <0.001): numbness 15% versus 66%, unpleasant sensations such as soreness and pain 44% versus 63%, and problems connected with daily activities 19% versus 69%. The majority of patients (60%) who preferred gel were also willing to pay for it. A conservative estimate of the median WTP was $10.00. Furthermore, anesthetic gel would make almost every second patient (45%) more or much more willing to return for the next treatment. CONCLUSIONS: The data suggest that a somewhat less profound anesthesia with gel is clearly preferred by the patients because of the low incidence of post-procedure problems as compared to conventional injection anesthesia. The median WTP is likely in excess of the acquisition cost of the product, which indicates a favorable cost-benefit ratio for the individual patient.