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1.
J Med Econ ; : 1-12, 2020 Feb 13.
Artigo em Inglês | MEDLINE | ID: mdl-31971453

RESUMO

Aim: Hemophilia A is a genetic, chronic disorder classified by deficient or defective coagulation factor VIII (FVIII) that puts those affected at risk for spontaneous bleeding episodes, which lead to joint damage and chronic pain over time. Currently, most severe hemophilia A patients are treated with prophylactic FVIII, which requires costly and frequent infusions and life-long adherence to medication. A gene therapy (valoctocogene roxaparvovec) is currently in development for the treatment of severe hemophilia A. This model assessed the potential cost-effectiveness of treating patients with valoctocogene roxaparvovec rather than prophylactic therapy.Materials and methods: We developed an individual-based, state-transition microsimulation model for assessing the likely cost-effectiveness of valoctocogene roxaparvovec compared to prophylactic FVIII. Men aged 30 with severe hemophilia A were modeled over a lifetime horizon, and costs were reported from the perspective of the United States health care system. Through microsimulation, patient-level heterogeneity was captured in starting weight, starting Pettersson score (PS), durability of valoctocogene roxaparvovec, and annual bleed rate (ABR).Results: The model projects that treatment with single-administration valoctocogene roxaparvovec would be cost-saving to people with hemophilia A at a price point comparable to other currently available gene therapy products due to its potential to reduce FVIII utilization, direct medical costs, lifetime bleeds, and accumulated joint damage.Limitations: The model relies upon evidence-based assumptions for clinical inputs due to limited data availability. Such uncertainty was mitigated by modeling heterogeneity across the population, specifically with regards to long-term gene therapy durability, lifetime bleed rates, and joint damage progression.Conclusion: Valoctocogene roxaparvovec was found to be cost-saving-on average by about $6.8 million per patient-and more effective than prophylactic therapy for treatment of hemophilia A. The comparative benefit of gene therapy was observed across a broad range of simulated patients that were representative of the real-world severe hemophilia A population.

2.
Artigo em Inglês | MEDLINE | ID: mdl-31999308

RESUMO

Importance: With the increasing emphasis on economic evaluations, there is a need for additional methods of measuring patient utility in the obstructive sleep apnea population. Objective: To develop and validate a utility scoring algorithm for a sleep apnea-specific quality-of-life instrument. Design, Setting, and Participants: Development and validation were conducted at 2 tertiary referral sleep centers and associated sleep clinics and included patients with newly diagnosed obstructive sleep apnea from a randomized clinical trial and an associated observational cohort study. Baseline participants were randomly divided into a model development group (60%) and a cross-validation group (40%). Main Outcomes and Measures: Utility scoring of the Symptoms of Nocturnal Obstruction and Related Events (SNORE-25) was mapped from the SF-6D utility index through multiple linear regression in the development sample using the Akaike information criterion to determine the best model. Results: A total of 500 participants (development, n = 300; validation, n = 200) were enrolled; the analyzed sample of 500 participants included 295 men (59%), and the mean (SD) age was 48.6 (12.8) years, with a range of 18 to 90 years. The mean (SD) SF-6D utility among participants with untreated sleep apnea was 0.61 (0.08; range, 0.40-0.85) with similar utility across sleep apnea severity groups. The best-fit model (the SNORE Utility Index) was the natural log conversion of the instrument subscales (r2 = 0.32 in the development sample). The SNORE Utility Index retained this association within the validation sample (r2 = 0.33). Conclusions and Relevance: The SNORE Utility Index provides a validated, disease-specific, preference-weighted utility instrument that can be used in future studies of patients with obstructive sleep apnea.

3.
Value Health ; 22(11): 1231-1239, 2019 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-31708059

RESUMO

BACKGROUND: For patients undergoing percutaneous coronary intervention, gene-drug associations exist relevant to first-line treatment options-antiplatelet agent, clopidogrel, and pain medication, tramadol. Knowledge of genotype information may allow for avoidance of adverse drug events during critical clinical windows. OBJECTIVE: This evaluation estimated cost-effectiveness associated with a multi-gene panel pre-emptively testing two genes providing CYP2C19 genotype-guided strategy for antiplatelet therapy, with CYP2D6 genotype-guided pain management, compared to single gene test for CYP2C19 with random assignment for pain treatment, and to no testing (empiric clopidogrel with random assignment for pain treatment). METHODS: Decision analysis modeling was used to project costs from a payer perspective and patient quality-adjusted life years (QALYs) from the three strategies. The model captured composite risks of major adverse cardiovascular events and pain therapy-related adverse drug events and associated utility estimates. We conducted sensitivity analyses to assess influential input parameters. RESULTS: Over 15 months, multi-gene testing was least costly and yielded more QALYs compared to both single gene and no testing; total incremental costs were $1646 lower with incremental gains of 0.04 QALYs for multi-gene compared with single gene and $11 368 lower with 0.17 QALY gains compared to no test. Base case analyses revealed multi gene was dominant compared to both single gene and no test, as it demonstrated cost savings with increased QALYs. CONCLUSIONS: For these patients, a multi-gene-guided strategy yields a favorable incremental cost-effectiveness ratio compared to the other two treatment strategies. Pre-emptively ascertaining additional gene-drug pair information can inform clinical and economic decision-making at the point of care.

4.
J Manag Care Spec Pharm ; 25(11): 1185-1192, 2019 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-31663458

RESUMO

In 2016, The Professional Society for Health Economics and Outcomes Research (ISPOR) formed a special task force (STF) to review approaches and methods to support the definition and use of high-quality U.S. value frameworks. As the leadership group of that initiative, we present our perspective, focusing on implications for the managed care pharmacy community. Our reflections are organized by 9 key observations and conclude with a summary recommendation. We begin by emphasizing the importance of distinguishing among "perspectives" and "decision contexts." Possible perspectives include patient, payer, provider, health care sector, and societal. Decision contexts range from formulary inclusion to guideline development to clinical shared decision making, and multiple perspectives can be taken on each of these decisions. The STF focused on value in the context of including a new medicine in a formulary and, thus, health plan, using a health economics approach that compares marginal benefit (gross value) and marginal (opportunity) cost, yielding the net value. Health care is unique compared with other markets. While economists often use market purchases as indicators of value, they also recognize that this does not work well in health care, since most patent-protected drugs are covered by insurance. To assess the likely health and economic impact, health economists often employ cost-effectiveness analysis, using the quality-adjusted life-year (QALY), a metric that combines mortality and morbidity into a single preference-based index. We strongly endorse the STF's recommendation that payers should use the cost-per-QALY metric as a starting point. However, like the STF, and many of those stakeholders who provided input, we recognize that this metric has some limitations in theory and in practice. Nonetheless, the cost-per-QALY metric is a pragmatic tool that can be augmented to address some of its limitations by integrating other elements of value, particularly those related to uncertainty, such as financial risk protection, health risk protection, the value of hope, real option value, and the value of knowing. The resulting adjusted ratio can be compared with a willingness-to-pay threshold or combined in a measure of net monetary benefit. Alternatively, the array of elements can be valued using multi-criteria decision analysis. We end with the key recommendation that further development and testing of these promising approaches is needed to improve the deliberative process of health technology assessment. DISCLOSURES: No outside funding supported the writing of this article. The authors are leaders of the ISPOR Special Task Force on U.S. Value Frameworks. Willke is employed by ISPOR. Garrison and Neumann have nothing to disclose. The opinions expressed in this article should be considered as belonging only to the authors.

5.
J Manag Care Spec Pharm ; 25(10): 1082-1087, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31556828

RESUMO

This analysis addresses an important policy question: "What are potentially transformative strategies and methods to define and measure value at all levels of decision making that are aligned with personalized medicine/precision medicine (PM/PrM)?" To approach this question, we build on our previous work with PM and recent work as members of ISPOR's Special Task Force on Value Assessment Frameworks. We focused on 3 key challenges: (1) the need for a broader concept of value for PM/PrM; (2) the development of appropriate concepts and methods for measuring and splitting the innovation reward for PM/PrM; and (3) the application of this expanded concept to align value assessment in the hierarchy of related decision contexts, from formulary inclusion and value-based pricing to shared patient-provider decision making. Our working assumption was that the goal of policy affecting PM/PrM technologies should be to promote "dynamic efficiency"-the optimal development, adoption, and use of these technologies. For this purpose, we laid out 6 basic cross-cutting policy principles. First, rewards for innovation should be value-based and flexible over time and across indications as new evidence emerges. Second, the concept of value should be based on the microeconomic concept of willingness to pay and should include, at its core, the value of health gain-in terms of the length and quality of life- but also going beyond this to include other uncertainty-related elements. Third, innovation rewards should be modifiable given new real-world evidence and knowledge. Fourth, the scientific information embedded in PM and PrM technologies is a global public good that should be financed globally through differential pricing. Fifth, reward systems need to recognize that the diagnostic technologies that make PM and PrM possible are technically "economic complements." Sixth, splitting the rewards among complementary inputs is arbitrary in a static sense but matters dynamically. For PM/PrM value assessment, we argue for including the "value of knowing"; the need for indication-specific pricing; the importance of updating the economic evaluation and pricing over the product life cycle; addressing the new challenge posed by next-generation sequencing and high-cost combination therapies; and aligning value metrics across different decision contexts based on health benefit and patient-centered utility. DISCLOSURES: No previous outside funding supported this policy analysis. The authors have nothing to disclose. The authors are grateful to have received first place in the PhRMA Foundation-Personalized Medicine Coalition 2018 Value Assessment Challenge Award for this work.

6.
J Immunother Cancer ; 7(1): 235, 2019 09 03.
Artigo em Inglês | MEDLINE | ID: mdl-31481112

RESUMO

Advances in the immunotherapy of cancer have prolonged survival for cancer patients, but the clinical and financial impact of treatments must be considered in determining the overall clinical utility and economic value of therapeutic agents. Quality-adjusted life years and incremental cost-effectiveness ratios are clinical and economic metrics that can be used to evaluate the value of immune checkpoint inhibitors. This Commentary provides perspective on the limitations, benefits, and potential enhancement of this approach to support value-based medicine.

7.
Value Health ; 22(7): 777-784, 2019 07.
Artigo em Inglês | MEDLINE | ID: mdl-31277824

RESUMO

BACKGROUND: Innovations that extend life can generate option value and cost of experiencing future technologies. OBJECTIVES: To understand how consideration of option value may affect the potential cost-effectiveness of a treatment through a case study of ipilimumab for previously untreated metastatic melanoma. METHODS: We estimated the cost-effectiveness of ipilimumab in 2 scenarios: a conventional scenario, for which we constructed the model using the standard methods that rely on efficacy data directly from the phase III trial of ipilimumab, and an option value scenario, where we incorporated future hypothetical improvements in mortality for metastatic melanoma owing to innovations. We developed 2 approaches to incorporate option value. In the first approach, we forecasted mortality trends based on historical trends from the Surveillance, Epidemiology, and End Results (SEER) Program registry. Alternatively, we identified drugs being studied in clinical trials at the time of ipilimumab's approval on clinicaltrials.gov and estimated their likelihood and timing of approval, potential efficacy, and cost. We accounted for increases in overall cancer treatment cost and unrelated medical cost in the option value scenario. RESULTS: In the option value scenario, using the SEER approach, the incremental quality-adjusted life-years (QALYs) gained and the incremental cost increased by 6.2% and 3.8%, respectively, whereas the incremental cost-effectiveness ratio (ICER) decreased by 2.3% compared with the conventional scenario. Using the clinicaltrials.gov approach, the incremental QALY gained and the incremental cost increased by 7.5% and 7.1%, respectively, whereas the ICER decreased by 0.40%. CONCLUSIONS: We developed generalizable approaches to estimating option value in cost-effectiveness analysis.


Assuntos
Antineoplásicos Imunológicos/economia , Antineoplásicos Imunológicos/uso terapêutico , Custos de Medicamentos , Ipilimumab/economia , Ipilimumab/uso terapêutico , Melanoma/tratamento farmacológico , Melanoma/economia , Neoplasias Cutâneas/tratamento farmacológico , Neoplasias Cutâneas/economia , Antineoplásicos Imunológicos/efeitos adversos , Tomada de Decisão Clínica , Ensaios Clínicos Fase III como Assunto , Análise Custo-Benefício , Progressão da Doença , Feminino , Humanos , Ipilimumab/efeitos adversos , Expectativa de Vida , Masculino , Cadeias de Markov , Melanoma/mortalidade , Melanoma/secundário , Pessoa de Meia-Idade , Modelos Econômicos , Intervalo Livre de Progressão , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Neoplasias Cutâneas/mortalidade , Neoplasias Cutâneas/patologia , Fatores de Tempo
9.
PLoS One ; 14(6): e0217331, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-31170193

RESUMO

OBJECTIVES: To estimate the modified societal costs of cervical cancer treatment in Kenya; and to compare the modified societal costs of treatment for pre-cancerous cervical lesions integrated into same-day HIV care compared to "non-integrated" treatment when the services are not coordinated on the same day. MATERIALS AND METHODS: A micro-costing study was conducted at Coptic Hope Center for Infectious Diseases and Kenyatta National Hospital from July 1-October 31, 2014. Interviews were conducted with 54 patients and 23 staff. Direct medical, non-medical (e.g., overhead), and indirect (e.g., time) costs were calculated for colposcopy, cryotherapy, Loop Electrosurgical Excision Procedure (LEEP), and treatment of cancer. All costs are reported in 2017 US dollars. RESULTS: Patients had a mean age of 41 and daily earnings of $6; travel time to the facility averaged 2.8 hours. From the modified societal perspective, per-procedure costs of colposcopy were $41 (integrated) vs. $91 (non-integrated). Per-procedure costs of cryotherapy were $22 (integrated) vs. $46 (non-integrated), whereas costs of LEEP were $50 (integrated) and $99 (non-integrated). This represents cost savings of $25 for cryotherapy and $50 for colposcopy and LEEP when provided on the same day as an HIV-care visit. Treatment for cervical cancer cost $1,345-$6,514, depending on stage. Facility-based palliative care cost $59/day. CONCLUSIONS: Integrating treatment of pre-cancerous lesions into HIV care is estimated to be cost-saving from a modified societal perspective. These costs can be applied to financial and economic evaluations in Kenya and similar urban settings in other low-income countries.

10.
Health Econ ; 28(7): 855-867, 2019 07.
Artigo em Inglês | MEDLINE | ID: mdl-31237095

RESUMO

A change in the expectations about future treatments may change the option value of a current treatment, thereby affecting its utilization. We conducted an interrupted time series analysis using a large administrative claims database to test whether the utilization of existing cancer treatments changed after the disclosures of the then-investigational drug ipilimumab's Phase II and Phase III results among metastatic melanoma patients from 2008 to 2011. We used a multinomial logistic regression to analyze the temporal probability of receiving antineoplastic systemic therapy, surgical resection of metastasis, or both, relative to no treatment, in the first 3 months following the first metastasis diagnosis. One thousand eight hundred forty-six metastatic melanoma patients were included. After adjusting for clinical and sociodemographic variables and the underlying time trend, the disclosure of ipilimumab's Phase II result was associated with a nearly twofold immediate increase in the probability of receiving surgical resection of metastasis relative to no treatment, which was significant at 5% level. No significant effect was observed for the time trend. No significant effects were found for the announcement of the Phase III result. Our findings in metastatic melanoma provide the first empirical evidence of the impact of option value in cancer treatment decision making.

11.
Value Health ; 22(6): 648-655, 2019 06.
Artigo em Inglês | MEDLINE | ID: mdl-31198181

RESUMO

OBJECTIVES: Recent regulatory approvals of potentially curative but high-cost treatments have made these therapies a focus of health policy discussions. Cures present new challenges to healthcare payers because they have high upfront costs but have life-long health benefits. The objectives of this study are to understand how healthcare payers define and manage cures. We investigated payers' views on key features of curative treatments and the affordability and value challenges they present. METHODS: We conducted semistructured interviews in 2016 with key informants in US payer organizations. Interviewees were directly involved in coverage determination for highly effective and curative therapies. RESULTS: We contacted 24 individuals and 18 participated. When asked what aspects of cures were important for coverage determination, an equal percentage of respondents (61% each) mentioned clinical and economic factors. In defining a cure, half of respondents included an economic element such as no downstream costs associated with the disease. When asked about challenges, 72% of respondents mentioned uncertainty regarding long-term outcomes and 56% mentioned membership churn and competition. CONCLUSIONS: Payers expressed a novel definition of a cure-which we call a "healthcare cost cure"-that captures both the clinical and economic consequences of treatment. This definition may be more pertinent in fragmentary financing systems that unevenly distribute cure costs and benefits across payers. Overall findings indicate that decision makers desire evidence to ensure that the long-term real-world consequences of covering cures match the expected benefits. Future policies need to balance upfront acquisition costs with downstream financial benefits.


Assuntos
Custos de Cuidados de Saúde/normas , Política de Saúde/tendências , Terapêutica/economia , Adulto , Idoso , Tomada de Decisões , Feminino , Custos de Cuidados de Saúde/tendências , Humanos , Entrevistas como Assunto/métodos , Masculino , Pessoa de Meia-Idade , Pesquisa Qualitativa , Terapêutica/métodos , Terapêutica/tendências
12.
J Med Econ ; 22(10): 1006-1013, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31050315

RESUMO

Purpose: The EF-14 trial demonstrated that adding tumor treating fields (TTFields) to maintenance temozolomide (TMZ) significantly extends progression-free survival (PFS) and overall survival (OS) for newly-diagnosed glioblastoma (GBM) patients. This study assessed the cost-effectiveness of TTFields and TMZ for newly-diagnosed GBM from the US healthcare system perspective. Methods and materials: Outcomes for newly-diagnosed GBM patients were estimated over a lifetime horizon using an area under the curve model with three states: stable disease, progressive disease, or death. The survival model integrated the 5-year EF-14 trial results with long-term GBM epidemiology data and US background mortality rates. Adverse event rates were derived from the EF-14 trial data. Utility values to determine quality-adjusted life-years, adverse event costs, and supportive care costs were obtained from published literature. A 3% discount rate was applied to future costs and outcomes. One-way and probabilistic sensitivity analyses were performed to assess result uncertainty due to parameter variability. Results: Treatment with TTFields and TMZ was estimated to result in a mean increase in survival of 1.25 life years (95% credible range [CR] = 0.89-1.67) and 0.96 quality-adjusted life years (QALYs) (95% CR = 0.67-1.30) compared to treatment with TMZ alone. The incremental total cost was $188,637 (95% CR = $145,324-$225,330). The incremental cost-effectiveness ratio (ICER) was $150,452 per life year gained and $197,336 per QALY gained. The model was most sensitive to changes in the cost of TTFields treatment. Conclusions: Adding TTFields to maintenance TMZ resulted in a substantial increase in the estimated mean lifetime survival and quality-adjusted survival for newly-diagnosed GBM patients. Treatment with TTFields can be considered cost-effective within the reported range of willingness-to-pay thresholds in the US.

13.
J Med Econ ; 22(8): 830-839, 2019 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-31081729

RESUMO

Background: Economic theory argues that specialization in medicine improves efficiency. Current literature suggests that access to and utilization of specialist care vary widely based on many determinants. Thus, understanding the determinants of specialist physician ambulatory care utilization is integral to healthcare policy. Objectives: The objective is to investigate the individual and community determinants of specialist ambulatory care utilization-specifically neurologists. The aim was to find predictors of specialist utilization and to identify the particular determinants that can be modified by regulatory or legislative action. Methods: A large claims database, Truven Health Analytics™ Marketscan data, was used from 2007-2010 as the sample. These data are supplemented with data from the American Academy of Neurology (for geographic distribution of neurologists) and the US Census American FactFinder (for community demographic factors). Multivariate regression analysis was run to test the hypotheses. Several robustness tests of our models were included. Results: Most importantly, neurologists per capita has a meaningful impact on utilization. Additionally, the difference in neurologist usage by neurological condition is an important factor. It was also found that union status, age, comorbidities, and diagnosis are significant individual level determinants, and that the percentage of Hispanic residents and median income are significant community level determinants. Conclusions: There are two predictors believed to be the most important. The first is the unique neurologists per 1,000 capita variable, which shows a small increase in the number of neurologists would be correlated with a small increase in the probability of seeing a neurologist. We suggest that this is within policymakers' control, and policymakers should consider this action in the face of the predicted shortage. The second is what appears to be possible sorting by neurologists of patients based on diagnosis - the large difference in the fraction of patients seeing a neurologist by disease.


Assuntos
Assistência Ambulatorial/estatística & dados numéricos , Neurologistas/estatística & dados numéricos , Características de Residência/estatística & dados numéricos , Adulto , Fatores Etários , Idoso , Comorbidade , Feminino , Acesso aos Serviços de Saúde/estatística & dados numéricos , Humanos , Masculino , Pessoa de Meia-Idade , Fatores Sexuais , Fatores Socioeconômicos , Estados Unidos
14.
Value Health ; 22(4): 408-415, 2019 04.
Artigo em Inglês | MEDLINE | ID: mdl-30975391

RESUMO

OBJECTIVE: The APHINITY trial assessed the effectiveness and the safety of adding pertuzumab to trastuzumab and chemotherapy (THP) compared to trastuzumab and chemotherapy (TH) in the adjuvant management of human epidermal growth factor 2-positive (HER2+) breast cancer. We performed a study to project the potential cost-effectiveness of THP vs. TH. STUDY DESIGN: Trial-based cost-utility modeling analysis. METHODS: We performed an economic evaluation from a payer perspective using a Markov model with six health states: invasive disease-free survival, non-metastatic recurrence, remission, first-line metastatic, subsequent line metastatic, and death. We parameterized the model using data from both arms in APHINITY extrapolated to a patient's lifetime horizon. Estimates of health state utilities were based on EQ-5D trial data and the literature, and costs were estimated from government sources and the published literature. The primary outcomes of the model were life-years (LYs), quality-adjusted LYs (QALYs), costs, and incremental cost-effectiveness ratios (ICERs). Uncertainty was addressed via univariate and probabilistic sensitivity analyses. RESULTS: For the intention-to-treat population, the model projected improved outcomes (by 0.50 LYs and 0.45 QALYs) and increased costs (by $74 420) for ICERs of $147 774/LY gained and $167 185/QALY gained for PHT vs. HT patients. In the node-positive patient population, the model projected improved outcomes (by 0.86 LYs and 0.76 QALYs) and increased costs (by $66 647) for ICERs of $77 684/LY gained and $87 929/QALY gained. For the hormone-receptor-negative patient population, the model projected health gains, increased costs, and ICERs of $147 022/LY gained and $166 518/QALY gained. The results were sensitive to changes in the model time horizon. CONCLUSION: The addition of pertuzumab to the available regimens for HER2+ early breast cancer is likely to be cost-effective for patients in the U.S. at high risk of recurrence.


Assuntos
Anticorpos Monoclonais Humanizados/administração & dosagem , Anticorpos Monoclonais Humanizados/economia , Antineoplásicos Imunológicos/administração & dosagem , Antineoplásicos Imunológicos/economia , Protocolos de Quimioterapia Combinada Antineoplásica/economia , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Neoplasias da Mama/tratamento farmacológico , Neoplasias da Mama/economia , Custos de Medicamentos , Receptor ErbB-2/antagonistas & inibidores , Trastuzumab/administração & dosagem , Trastuzumab/economia , Idoso , Idoso de 80 Anos ou mais , Anticorpos Monoclonais Humanizados/efeitos adversos , Antineoplásicos Imunológicos/efeitos adversos , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Neoplasias da Mama/enzimologia , Neoplasias da Mama/mortalidade , Quimioterapia Adjuvante/economia , Redução de Custos , Análise Custo-Benefício , Progressão da Doença , Intervalo Livre de Doença , Feminino , Humanos , Cadeias de Markov , Pessoa de Meia-Idade , Modelos Econômicos , Metástase Neoplásica , Recidiva Local de Neoplasia , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Ensaios Clínicos Controlados Aleatórios como Assunto , Receptor ErbB-2/metabolismo , Fatores de Tempo , Trastuzumab/efeitos adversos , Resultado do Tratamento
15.
J Manag Care Spec Pharm ; 25(7): 793-799, 2019 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-30784347

RESUMO

While one-time gene replacement therapies may offer transformative innovation for the management of ultrarare, health-catastrophic diseases, they also pose challenges to the current U.S. health care system. Historically, the United States and other countries have demonstrated a willingness to support higher prices for health gains in rare diseases. However, payers may be ill-prepared to address reimbursement based on single administrations associated with gene therapies. As yet, there is no consensus on how to appropriately reward gene therapy innovation. The purpose of this article is to characterize challenges for traditional approaches to assessing the value of one-time gene replacement therapies and to provide a health economic rationale for a higher value-based cost-effectiveness threshold (CET). There is a general recognition that ultrarare, health-catastrophic conditions should be judged against a higher CET. The Institute for Clinical and Economic Review in the United States has discussed a range of up to $500K per quality-adjusted life-year (QALY) gained for ultrarare diseases, and the National Institute for Health and Care Excellence in the United Kingdom has described a variable threshold up to £300,000 per QALY depending on the magnitude of the health gains. In practice, health technology assessment decision makers often make comparisons to "benchmarks" to justify both standard and extraordinary CETs. We briefly review and present a list of relevant benchmarks. We also sketch out how a broader concept of value could provide the basis for higher CETs for some ultrarare diseases. This approach is outlined by the recent International Society for Pharmacoeconomics and Outcomes Research Special Task Force on Value Assessment Frameworks. In addition to the QALY gains, other elements of value related to uncertainty may also be important. They include insurance value, severity of disease, real option value, value of hope, and equity. A gene therapy currently in development for the treatment of spinal muscular atrophy (SMA) provides an exemplar for discussing the issues that accompany one-time gene replacement therapies. It is imperative that we find a consensus on how to appropriately reward value created by these gene therapies to incentivize appropriate risk taking and investments by their developers-a higher CET would, by economic logic, support a higher value-based price. If consensus on appropriate rewards cannot be found for safe and effective gene therapies for diseases such as SMA with clear criticality and unmet need, it will be even more difficult to do so for diseases where the value provided is less apparent. DISCLOSURES: Funding for the writing of this article was provided by AveXis Pharmaceuticals, which reviewed the manuscript and contributed feedback during manuscript development. The authors had final editorial control. Jackson and Paul are employees of MME, a biopharmaceutical consulting firm that received funding from AveXis for work on this project. Jackson and Paul also report consulting fees from numerous other biopharmaceutical companies outside of this project. Garrison reports consulting fees from AveXis for work on this project and advisory/consultancy fees from BioMarin, Roche, Novartis, and Pfizer unrelated to this project. Kenston is a former employee of AveXis and reports consulting fees from AveXis for this project and for other projects outside of this work.


Assuntos
Doença Catastrófica/terapia , Assistência à Saúde/economia , Terapia Genética/métodos , Mecanismo de Reembolso/economia , Benchmarking , Doença Catastrófica/economia , Análise Custo-Benefício , Terapia Genética/economia , Humanos , Atrofia Muscular Espinal/economia , Atrofia Muscular Espinal/terapia , Anos de Vida Ajustados por Qualidade de Vida , Estados Unidos
16.
Value Health Reg Issues ; 17: 219-223, 2018 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-30528780

RESUMO

In February and September of 2017, the International Society for Pharmacoeconomics and Outcomes Research Health Technology Assessment Council held roundtables focused on Latin America to discuss health technology assessment best practices, collaboration opportunities, and regional experiences regarding health policies to improve the affordability of and access to healthcare technologies. The access to high-cost technologies, increased social pressure to achieve universal coverage, population aging, and the limits of traditional mechanisms to control costs create political pressure to begin considering other pricing alternatives, including value-based pricing, in Latin America. This article attempts to conceptualize key stakeholders' perceptions of their experiences, opportunities, and barriers to implementing value-based pricing in Latin America.


Assuntos
Custos e Análise de Custo , Farmacoeconomia/tendências , Avaliação da Tecnologia Biomédica/tendências , Cobertura Universal do Seguro de Saúde/tendências , Envelhecimento , Tecnologia Biomédica/economia , Política de Saúde , Humanos , América Latina
17.
Value Health ; 21(10): 1133-1149, 2018 10.
Artigo em Inglês | MEDLINE | ID: mdl-30314613

RESUMO

This report provides recommendations for budget holders and decision makers in high-, middle, and low-income countries requiring economic analyses of new vaccination programs to allocate scarce resources given budget constraints. ISPOR's Economic Evaluation of Vaccines Designed to Prevent Infectious Disease: Good Practices Task Force wrote guidelines for three analytic methods and solicited comments on them from external reviewers. Cost-effectiveness analyses use decision-analytic models to estimate cumulative changes in resource use, costs, and changes in quality- or disability-adjusted life-years attributable to changes in disease outcomes. Constrained optimization modeling uses a mathematical objective function to be optimized (e.g. disease cases avoided) for a target population for a set of interventions including vaccination programs within established constraints. Fiscal health modeling estimates changes in net present value of government revenues and expenditures attributable to changes in disease outcomes. The task force recommends that those designing economic analyses for new vaccination programs take into account the decision maker's policy objectives and country-specific decision context when estimating: uptake rate in the target population; vaccination program's impact on disease cases in the population over time using a dynamic transmission epidemiologic model; vaccination program implementation and operating costs; and the changes in costs and health outcomes of the target disease(s). The three approaches to economic analysis are complementary and can be used alone or together to estimate a vaccination program's economic value for national, regional, or subregional decision makers in high-, middle-, and low-income countries.


Assuntos
Comitês Consultivos/economia , Análise Custo-Benefício/métodos , Programas de Imunização/economia , Programas de Imunização/métodos , Política de Saúde/economia , Humanos , /métodos
18.
Sci Rep ; 8(1): 6066, 2018 04 17.
Artigo em Inglês | MEDLINE | ID: mdl-29666455

RESUMO

Promising multi-dose HIV vaccine regimens are being tested in trials in South Africa. We estimated the potential epidemiological and economic impact of HIV vaccine campaigns compared to continuous vaccination, assuming that vaccine efficacy is transient and dependent on immune response. We used a dynamic economic mathematical model of HIV transmission calibrated to 2012 epidemiological data to simulate vaccination with anticipated antiretroviral treatment scale-up in South Africa. We estimate that biennial vaccination with a 70% efficacious vaccine reaching 20% of the sexually active population could prevent 480,000-650,000 HIV infections (13.8-15.3% of all infections) over 10 years. Assuming a launch price of $15 per dose, vaccination was found to be cost-effective, with an incremental cost-effectiveness ratio of $13,746 per quality-adjusted life-year as compared to no vaccination. Increasing vaccination coverage to 50% will prevent more infections but is less likely to achieve cost-effectiveness. Campaign vaccination is consistently more effective and costs less than continuous vaccination across scenarios. Results suggest that a partially effective HIV vaccine will have substantial impact on the HIV epidemic in South Africa and offer good value if priced less than $105 for a five-dose series. Vaccination campaigns every two years may offer greater value for money than continuous vaccination reaching the same coverage level.


Assuntos
Vacinas contra a AIDS/uso terapêutico , Infecções por HIV/prevenção & controle , Programas de Imunização , Vacinas contra a AIDS/economia , Adolescente , Adulto , Análise Custo-Benefício , Feminino , Infecções por HIV/economia , Infecções por HIV/epidemiologia , Humanos , Programas de Imunização/economia , Programas de Imunização/métodos , Masculino , Pessoa de Meia-Idade , Modelos Econômicos , Anos de Vida Ajustados por Qualidade de Vida , África do Sul/epidemiologia , Resultado do Tratamento , Adulto Jovem
20.
J Med Econ ; 21(7): 712-720, 2018 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-29701508

RESUMO

BACKGROUND: Autonomic nervous system (ANS) testing with heart rate variability (HRV) has been shown in early research to predict 52-week outcomes in rheumatoid arthritis (RA). HRV testing could be combined with putative ANS biologic pathways to improve treatment response for RA patients. This study explored potential costs and health outcomes of introducing HRV testing into RA treatment, without and with ANS optimization. METHODS: A decision tree exploratory economic model compared HRV testing to standard care in moderate-to-severe biologic-eligible patients over a 10-year time horizon. HRV data was derived from an observational study of RA patients (n = 33). Patients were stratified into treatment groups based on HRV test scores indicating "low probability of response" and "moderate to high probability of response". This study explored adding ANS optimization based on HRV score followed by clinically-appropriate treatment. Costs and quality-adjusted life-years (QALYs) for the US population were estimated. RESULTS: HRV testing in biologic-eligible patients decreased non-effective biologic use, reducing US healthcare costs by $34.6 billion over 10 years with QALYs unchanged. When combined with ANS optimization in biologic-eligible patients, HRV testing could increase costs by $3.6 billion over 10 years but save over 350,000 QALYs. Among all RA patients, HRV testing with ANS optimization could save over $8 billion and over 100,000 QALYs over 10 years, depending on the positive predictive value (PPV) of the HRV test. CONCLUSIONS: The potential economic impact of introducing HRV testing and ANS optimization into RA treatment appears substantial and cost-effective based on the exploratory analysis. Additional rigorous studies are warranted in larger patient samples to better inform decision-making.


Assuntos
Antirreumáticos/economia , Antirreumáticos/uso terapêutico , Artrite Reumatoide/tratamento farmacológico , Produtos Biológicos/economia , Produtos Biológicos/uso terapêutico , Frequência Cardíaca/fisiologia , Artrite Reumatoide/economia , Artrite Reumatoide/fisiopatologia , Análise Custo-Benefício , Árvores de Decisões , Gastos em Saúde , Humanos , Modelos Econométricos , Modelos Econômicos , Anos de Vida Ajustados por Qualidade de Vida , Índice de Gravidade de Doença , Estados Unidos
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