Budget impact analysis of darolutamide for treatment of nonmetastatic castration-resistant prostate cancer.

BACKGROUND: Darolutamide, a structurally distinct androgen receptor inhibitor approved for the treatment of men with nonmetastatic castration-resistant prostate cancer (nmCRPC), has been shown to increase metastasis-free survival among men with nmCRPC compared with placebo. This treatment has a novel chemical structure that may also have safety, tolerability, and efficacy advantages for men with nmCRPC. OBJECTIVE: To estimate the projected budget impact of including darolutamide on a U.S. payer formulary as a treatment option for men with nmCRPC. METHODS: A budget impact model was developed to evaluate darolutamide for nmCRPC for a hypothetical 1-million-member plan over a 5-year period. Costs (drug acquisition, drug administration, and treatment-related adverse events [AEs]) were estimated for 2 scenarios: with and without darolutamide treatment for nmCRPC. The budget impact of darolutamide was calculated as the difference in costs for these 2 scenarios. An analysis for high-risk nmCRPC also was conducted. The model included treatments recommended by the National Comprehensive Cancer Network (e.g., apalutamide and enzalutamide) and potential comparators that are used but are not specifically indicated for nmCRPC. All treatments were assumed to be administered in combination with a weighted average androgen deprivation therapy comparator (consisting of luteinizing hormone-releasing hormone [LHRH] agonists, LHRH antagonists, and first-generation antiandrogens). Market share estimates were derived from interviews with physicians treating men with nmCRPC. The model includes grade 3-4 AEs, and the rates were obtained from clinical trial data. Costs were taken from publicly available sources and varied in a one-way sensitivity analysis. RESULTS: For a plan with 1 million lives, there were approximately 90 incident cases of nmCRPC (46 high risk) each year, with 332 (109 high risk) treatment-eligible cases by year 5. Darolutamide's market share increased from 3.6% in year 1 to 18% in year 5. Given the utilization of other agents, introducing darolutamide along with other targeted therapies was predicted to increase the total budget by $158,640 ($0.0132 per member per month [PMPM]) in year 1, which decreased over time to a cost savings of $149,240 ($0.0124 PMPM) by year 5. The scenario with darolutamide showed reduced AE costs each year. Similar results were observed for the high-risk nmCRPC population. CONCLUSIONS: Adding darolutamide to a U.S. payer formulary for the treatment of nmCRPC can result in a manageable increase in the budget that is partly offset by AE costs in the first 4 years, followed by a cost savings by year 5. DISCLOSURES: This study was conducted by RTI Health Solutions under the direction of Bayer U.S. and was funded by Bayer U.S., which was involved in the design of the study; collection, analysis, and interpretation of the data; writing of the report; and the decision to submit the report for publication. Miles and Purser (and/or their institutions) are employees of RTI Health Solutions and received research funding from Bayer U.S. to develop the budget impact model. Appukkuttan and Farej are employees of Bayer U.S. Wen was an employee of Bayer U.S. at the time of the study. This study was presented as a poster at the AMCP Virtual Learning Event, April 20-24, 2020.

Integrating E-Prescribing and Pharmacy Claims Data for Predictive Modeling: Comparing Costs and Utilization of Health Plan Members Who Fill Their Initial Medications with Those Who Do Not.

BACKGROUND: Nonfilling of prescribed medications is a worldwide problem of serious concern. Studies of health care costs and utilization associated with medication nonadherence frequently rely on claims data and usually focus on patients with specific conditions. Past studies also have little agreement on whether higher medication costs associated with higher adherence can reduce downstream health care consumption. OBJECTIVES: To (a) compare the characteristics between people with and without complete medication initiations from a general population and (b) quantify the effect of medication initiation on health care utilization and expenditures with propensity score weighting. METHODS: We conducted a retrospective cohort study using 2012 and 2013 electronic health records (EHR) and insurance claims data from an integrated health care delivery network. We included 43,097 eligible primary care patients in the study. Annual medication fill rates of initial prescriptions in 2012 were defined as the number of filled prescriptions from claims divided by the number of e-prescriptions from EHRs, while excluding all refills. A claim was considered filled if (a) EHR and claims records were from the same drug class; (b) claims occurred between the date of a current EHR order and that of the next EHR order of the same class; and (c) the maximum fill rate was 100%. The 6 annual outcomes included total costs, medical costs, pharmacy costs, being a high-cost "outlier" (in top 5%), having 1 or more hospitalizations, and having 1 or more emergency department (ED) visits. Individuals were classified as either having completed all medication initiations (100% annual filling rate for initiations) or not. We used propensity score weighting to control for baseline differences between complete and incomplete initial fillers. We adopted linear and logistic regressions to model costs and binary utilization indicators for the same year (concurrently) and next year (prospectively). RESULTS: Approximately 42% of the study sample had complete medication initiations (100% filling rate), while the remaining 58% had incomplete initiations. Individuals who fully filled initial prescriptions had lower comorbidity burden and consumed fewer health care resources. After applying propensity score weighting and controlling for variables such as the number of prescription orders, patients with complete medication initiations had lower overall and medical costs, concurrently and prospectively (e.g., $751 and $252 less for annual total costs). Complete medication initiation fillers were also less likely to have concurrent health care utilization (OR = 0.78, 95% CI = 0.68-0.90 for hospitalization; OR = 0.77, 95% CI = 0.72-0.82 for ED admissions) but no difference in prospective utilization other than for ED visits (OR = 0.93, 95% CI = 0.87-0.99). CONCLUSIONS: Identifying the subpopulation of patients with incomplete medication initiations (i.e., filling less than 100% of initial prescriptions) is a pragmatic approach for population health management programs to align resources and potentially contain cost and utilization. DISCLOSURES: No outside funding supported this study. This study applied the Adjusted Clinical Group (ACG) case-mix/risk adjustment methodology, developed at Johns Hopkins Bloomberg School of Public Health. Although ACGs are an important aspect of this study, the goal of the study was not to directly assess or evaluate the methodology. The Johns Hopkins University receives royalties for nonacademic use of software based on the ACG methodology. Chang, Kharrazi, and Weiner receive a portion of their salary support from this revenue. Chang is also a part-time consultant for Monument Analytics, a health care consultancy whose clients include the life sciences industry, as well as plaintiffs in opioid litigation. Alexander is past Chair of FDA's Peripheral and Central Nervous System Advisory Committee; has served as a paid advisor to IQVIA; is a co-founding Principal and equity holder in Monument Analytics; and is a member of OptumRx's National P&T Committee. These arrangements have been reviewed and approved by Johns Hopkins University in accordance with its conflict of interest policies. The other authors have nothing to disclose.

Cost-effectiveness of first-line vs third-line ibrutinib in patients with untreated chronic lymphocytic leukemia.

The ALLIANCE A041202 trial found that continuously administered ibrutinib in the first-line setting significantly prolonged progression-free survival compared with a fixed-duration treatment of rituximab and bendamustine in older adults with chronic lymphocytic leukemia (CLL). In this study, we created a Markov model to assess the cost-effectiveness of ibrutinib in the first-line setting, compared with a strategy of using ibrutinib in the third-line after failure of time-limited bendamustine and venetoclax-based regimens. We estimated transition probabilities from randomized trials using parametric survival modeling. Lifetime direct health care costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) were calculated from a US payer perspective. First-line ibrutinib was associated with an improvement of 0.26 QALYs and 0.40 life-years compared with using ibrutinib in the third-line setting. However, using ibrutinib in the first-line led to significantly higher health care costs (incremental cost of $612 700), resulting in an ICER of $2 350 041 per QALY. The monthly cost of ibrutinib would need to be decreased by 72% for first-line ibrutinib therapy to be cost-effective at a willingness-to-pay threshold of $150 000 per QALY. In a scenario analysis where ibrutinib was used in the second-line in the delayed ibrutinib arm, first-line ibrutinib had an incremental cost of $478 823, an incremental effectiveness of 0.05 QALYs, and an ICER of $9 810 360 per QALY when compared with second-line use. These data suggest that first-line ibrutinib for unselected older adults with CLL is unlikely to be cost-effective under current pricing. Delaying ibrutinib for most patients with CLL until later lines of therapy may be a reasonable strategy to limit health care costs without compromising clinical outcomes.

Medicines' private costs among elderly and the impairment of family income in a medium-sized municipality in the state of São Paulo. / Gasto privado com medicamentos entre idosos e o comprometimento da renda familiar em município de médio porte no estado de São Paulo.

INTRODUCTION: The acquisition of medicines accounts for a significant proportion of private health expenditures. The objective of this study was to analyse the private spending with the purchase of medicines and the commitment of the family income, by the elderly. METHODS: Population survey conducted in Praia Grande, São Paulo, Brazil. The monthly expenditure and the per capita family income commitment with the purchase of medicines were calculated from the information obtained in the interviews. The variables were described in absolute and relative frequencies and the hypothesis test was Pearson's χ2, Student's t and Anova, with a significance level of 5%. RESULTS: The prevalence of drug use was 61.2%. The average monthly expenditure per capita was R$ 34.59, with significantly higher income impairment for individuals with higher levels of education, without chronic diseases and health plan beneficiaries. CONCLUSION: The prevalence of drug use was low. The cost generated by the purchase of medicines is one of the ways in which inequality can manifest in society. The expansion of free drug provision would be necessary to expand access and avoid spending, especially those who have private health plans but cannot afford drug treatment.

Benchmarking biopharmaceutical process development and manufacturing cost contributions to R&D.

This study aims to benchmark and analyze the process development and manufacturing costs across the biopharmaceutical drug development cycle and their contribution to overall research and development (R&D) costs. This was achieved with a biopharmaceutical drug development lifecycle cost model that captured the costs, durations, risks and interdependencies of the clinical, process development and manufacturing activities. The budgets needed for process development and manufacturing at each phase of development to ensure a market success each year were estimated. The impact of different clinical success rate profiles on the process development and manufacturing costs at each stage was investigated, with a particular focus on monoclonal antibodies. To ensure a market success each year with an overall clinical success rate (Phase I to approval) of ~12%, the model predicted that a biopharmaceutical company needs to allocate process development and manufacturing budgets in the order of ~$60 M for pre-clinical to Phase II material preparation and ~$70 M for Phase III to regulatory review material preparation. For lower overall clinical success rates of ~4%, which are more indicative of diseases such as Alzheimer's, these values increase to ~$190 M for early-phase and ~$140 Mfor late-phase material preparation; hence, the costs increase 2.5 fold. The costs for process development and manufacturing per market success were predicted to represent 13-17% of the R&D budget from pre-clinical trials to approval. The results of this quantitative structured cost study can be used to aid decision-making during portfolio management and budget planning procedures in biopharmaceutical development.

Add-On Therapies in Cardiovascular Disease: Reviewing ICER's Report and the Potential Effect on Payers.

DISCLOSURES: No outside funding supported this commentary. The authors have nothing to disclose.

Cost-Effectiveness of Rifaximin Treatment in Patients with Hepatic Encephalopathy.

BACKGROUND: Hepatic encephalopathy (HE) is a complication of cirrhosis of the liver causing neuropsychiatric abnormalities. Clinical manifestations of overt HE result in increased health care resource utilization and effects on patient quality of life. While lactulose has historically been the mainstay of treatment for acute HE and maintenance of remission, there is an unmet need for additional therapeutic options with a favorable adverse event profile. Compared with lactulose alone, rifaximin has demonstrated proven efficacy in complete reversal of HE and reduction in the incidence of HE recurrence, mortality, and hospitalizations. Evidence suggests the benefit of long-term prophylactic therapy with rifaximin; however, there is a need to assess the economic impact of rifaximin treatment in patients with HE. OBJECTIVE: To assess the incremental cost-effectiveness of rifaximin ± lactulose versus lactulose monotherapy in patients with overt HE. METHODS: A Markov model was developed in Excel with 4 health states (remission, overt HE, liver transplantation, and death) to predict costs and outcomes of patients with HE after initiation of maintenance therapy with rifaximin ± lactulose to avoid recurrent HE episodes. Cost-effectiveness of rifaximin was evaluated through estimation of incremental cost per quality-adjusted life-year (QALY) or life-year (LY) gained. Analyses were conducted over a lifetime horizon. One-way deterministic and probabilistic sensitivity analyses were conducted to assess uncertainty in results. RESULTS: The rifaximin ± lactulose regimen provided added health benefits despite an additional cost versus lactulose monotherapy. Model results showed an incremental benefit of $29,161 per QALY gained and $27,762 per LY gained with rifaximin ± lactulose versus lactulose monotherapy. Probabilistic sensitivity analyses demonstrated that the rifaximin ± lactulose regimen was cost-effective ~99% of the time at a threshold of $50,000 per QALY/LY gained, which falls within the commonly accepted threshold for incremental cost-effectiveness. CONCLUSIONS: The clinical benefit of rifaximin, combined with an acceptable economic profile, demonstrates the advantages of rifaximin maintenance therapy as an important option to consider for patients at risk of recurrent HE. DISCLOSURES: This analysis was funded by Salix Pharmaceuticals, a division of Bausch Health US. Salix and Xcenda collaborated on the methods, and Salix, Xcenda, Jesudian, and Ahmad collaborated on the writing of the manuscript and interpretation of results. Bozkaya and Migliaccio-Walle are employees of Xcenda. Ahmad reports speaker fees from Salix Pharmaceuticals, unrelated to this study. Jesudian reports consulting and speaker fees from Salix Pharmaceuticals, unrelated to this study. The results from this model were presented at AASLD: The Liver Meeting 2014; November 7-11; Boston, MA.

Medical Costs and Health Care Utilization Among Self-Insured Members with Carve-In Versus Carve-Out Pharmacy Benefits.

BACKGROUND: Pharmacy benefit can be purchased as part of an integrated medical and pharmacy health package-a carve-in model-or purchased separately and administered by an external pharmacy benefit manager-a carve-out model. Limited peer-reviewed information is available assessing differences in use and medical costs among carve-in versus carve-out populations. OBJECTIVE: To compare total medical costs per member per year (PMPY) and utilization between commercially self-insured members receiving carve-in to those receiving carve-out pharmacy benefits overall and by 7 chronic condition subgroups. METHODS: This study used deidentified data of members continuously enrolled in Cambia Health Solutions self-insured Blue plans without benefit changes from 2017 through 2018. Cambia covers 1.6 million members in Oregon, Washington, Idaho, and Utah. The medical cost PMPY comparison was performed using multivariable general linear regression with gamma distribution adjusting for age, gender, state, insured group size, case or disease management enrollment, 7 chronic diseases, risk score (illness severity proxy), and plan paid to total paid ratio (benefit richness proxy). Medical event objectives were assessed using multivariable logistic regression comparing odds of hospitalization and emergency department (ED) visit adjusting for the same covariates. Sensitivity analyses repeated the medical cost PMPY comparison excluding high-cost members, greater than $250,000 annually. Chronic condition subgroup analyses were performed using the same methods separately for members having asthma, coronary artery disease, chronic obstructive pulmonary disease, heart failure, diabetes mellitus, depression, and rheumatoid arthritis. RESULTS: There were 205,835 carve-in and 125,555 carve-out members meeting study criteria. Average age (SD) was 34.2 years (18.6) and risk score (SD) 1.1 (2.3) for carve-in versus 35.2 years (19.3) and 1.1 (2.4), respectively, for carve-out. Members with carve-in benefits had lower medical costs after adjustment (4%, P < 0.001), translating into an average $148 lower medical cost PMPY ($3,749 carve-out vs. $3,601 carve-in annualized). After adjustment, the carve-in group had an estimated 15% (P < 0.001) lower hospitalization odds and 7% (P < 0.001) lower ED visit odds. Of 7 chronic conditions, significantly lower costs (12%-17% lower), odds of hospitalization (22%-36% lower), and odds of ED visit (16%-20% lower) were found among members with carve-in benefits for 5 conditions (all P < 0.05). CONCLUSIONS: These findings suggest that integrated, carve-in pharmacy and medical benefits are associated with lower medical costs, fewer hospitalizations, and fewer ED visits. This study focused on associations, and defining causation was not in scope. Possible reasons for these findings include plan access to both medical and pharmacy data and data-informed care management and coordination. Future research should include investigation of integrated data use and its effect across the spectrum of integrated health plan offerings, provider partnerships, and analytic strategies, as well as inclusion of analyzing pharmacy costs to encompass total cost of care. DISCLOSURES: This study received no external funding. The study was jointly conducted by employees of Cambia Health Solutions and Prime Therapeutics, a pharmacy benefit manager servicing Cambia Health Solutions. Smith, Lam, Lockwood, and Pegus are employees of Cambia Health Solutions. Qiu and Gleason are employees of Prime Therapeutics.

Cost-effectiveness of Anti-VEGF treatments for age-related macular degeneration: a Brazilian perspective.

PURPOSE: To study the cost-effectiveness of ranibizumab and bevacizumab for the treatment of age-related macular degeneration. METHODS: We used a decision tree model to analyze the cost-effectiveness of ranibizumab and bevacizumab for the treatment of age-related macular degeneration, from the Brazilian Public Health System (SUS) perspective. Ranibizumab and bevacizumab were administered to patients with the same treatment procedure, and the difference in treatment costs was calculated based on the cost of the drugs. Direct costs were estimated using the information provided by the Brazilian SUS. Effectiveness in terms of quality-adjusted life years (QALYs) was calculated based on the utility values for visual impairment. Incremental cost-effectiveness ratio was calculated by comparing both treatments. The analytical horizon was one year. RESULTS: The decision tree analysis showed that the difference in treatment effectiveness was 0.01 QALY. Incremental cost-effectiveness ratio showed that ranibizumab treatment required an incremental annual cost of more than R$ 2 million to generate 1 additional QALY, as compared to bevacizumab. CONCLUSIONS: From the Brazilian SUS perspective, bevacizumab is more cost-effective than ranibizumab for the treatment of neovascular age-related macular degeneration. Its use could allow potential annual savings in health budget.

Cost-effectiveness of Anti-VEGF treatments for age-related macular degeneration: a Brazilian perspective / Estudo de custo-efetividade de tratamentos para a degeneração macular relacionada à idade: uma perspectiva brasileira

ABSTRACT Purpose: To study the cost-effectiveness of ranibizumab and bevacizumab for the treatment of age-related macular degeneration. Methods: We used a decision tree model to analyze the cost-effectiveness of ranibizumab and bevacizumab for the treatment of age-related macular degeneration, from the Brazilian Public Health System (SUS) perspective. Ranibizumab and bevacizumab were administered to patients with the same treatment procedure, and the difference in treatment costs was calculated based on the cost of the drugs. Direct costs were estimated using the information provided by the Brazilian SUS. Effectiveness in terms of quality-adjusted life years (QALYs) was calculated based on the utility values for visual impairment. Incremental cost-effectiveness ratio was calculated by comparing both treatments. The analytical horizon was one year. Results: The decision tree analysis showed that the difference in treatment effectiveness was 0.01 QALY. Incremental cost-effectiveness ratio showed that ranibizumab treatment required an incremental annual cost of more than R$ 2 million to generate 1 additional QALY, as compared to bevacizumab. Conclusions: From the Brazilian SUS perspective, bevacizumab is more cost-effective than ranibizumab for the treatment of neovascular age-related macular degeneration. Its use could allow potential annual savings in health budget.

RESUMO Objetivo: Estudar o custo-efetividade do ranibizumabe e bevacizumabe no tratamento da degeneração macular relacionada à idade neovascular. Métodos: Utilizamos um modelo de árvore de decisão para analisar a relação custo-efetividade do ranibizumabe e bevacizumabe no tratamento da degeneração macular relacionada à idade, sob a perspectiva do Sistema Único de Saúde. O ranibizumabe e bevacizumabe foram administrados a pacientes com o mesmo procedimento de tratamento, e a diferença nos custos do tratamernto foi calculada com base no custo dos medicamentos. Os custos diretos foram estimados utilizando as informações fornecidas pelo SUS. A efetividade foi determinada em anos de vida ajustados pela qualidade (QALY) baseados em valores de utilidade em deficiênciavisual. A razãoincremental custo-efetividadefoicalculada comparando os dois tratamentos. O horizonte analítico foi de um ano. Resultados: A análise da árvore de decisão mostrou que a diferença na efetividade do tratamento foi de 0,01 QALY. A razão incremental de custo-efetividade mostrou que o tratamento com ranibizumabe exigiu um custo anual incremental de R$ 2 milhões para gerar um QALY adicional, em comparação ao bevacizumabe. Conclusões: Do ponto de vista do SUS, o bevacizumabe é mais custo-efetivo que o ranibizumabe no tratamento da degeneração macular relacionada à idade neovascular. O seu uso poderia gerar uma grande economia anual para o orçamento em saúde.

A comparative cost-utility analysis of postoperative calcium supplementation strategies used in the current management of hypocalcemia.

BACKGROUND: Symptomatic hypocalcemia is a common complication of total thyroidectomy. Management strategies include responsive treatment initiation for symptoms or prevention by routine or parathyroid hormone-directed calcium supplementation. The comparative cost-effectiveness of even the most often utilized strategies is unclear. METHODS: A Markov cohort model was created to compare routine supplementation with calcium alone (RS), postoperative parathyroid hormone-based selective supplementation with calcium and calcitriol (SS), and no supplementation (NS) in asymptomatic patients. Patients could remain asymptomatic or develop symptomatic hypocalcemia, managed with outpatient oral supplementation or intravenous calcium infusion and administered either inpatient or outpatient. Effectiveness was measured in quality-adjusted life years. Sensitivity analyses were performed to test model parameter assumptions. RESULTS: RS was the preferred strategy, costing $329/patient and resulting in 0.497 quality-adjusted life years, which was only marginally better compared to SS ($373 for 0.495 quality-adjusted life years). NS was most costly at $4,955 for 0.491 quality-adjusted life years. Preference for RS over SS was sensitive to the probability of developing symptoms and the probability of symptom treatment with intravenous supplementation. On probabilistic sensitivity analysis, RS was preferred in 75.4% of scenarios. CONCLUSION: After total thyroidectomy, a preventative calcium supplementation strategy should be strongly considered. In this data-driven theoretical model, RS was the least costly option and resulted in an incremental gain in quality-adjusted life years.

Understanding profit margins of medical providers from prescription drugs: evidence from Taiwan.

BACKGROUND: This study empirically estimates the magnitude and associated determinants of profit margins that medical providers earn from prescription drugs based on Taiwan's pharmaceutical market. METHODS: Our main data set is from the population-based claims data compiled by the National Health Insurance Research Database covering three waves of price adjustment: July-December 2004, October 2007-September 2008 and October 2009-September 2010. Only drugs whose reimbursement prices were adjusted using the R-zone formula were used as samples for this study. By calculating the difference between retail and wholesale prices for 796 pharmaceutical products, we can estimate the profit margin determinants using the regression model. RESULTS: We found evidence that suppliers of generic drugs tend to offer larger discounts to medical providers than suppliers of brand-name drugs. In addition, the countervailing power of wholesale pharmaceuticals, as measured by the discount rate offered by pharmaceutical manufacturers, is positively associated with the degree of competition within the pharmaceutical market and the size of the market itself. CONCLUSIONS: Our findings imply that the profit-seeking behaviour exhibited by medical providers is the engine of competitive forces in Taiwan's prescription drug market. This creates financial incentives for them, which in turn influences their choices of prescription drugs.

Progress on drug pricing negotiations in China.

On November 28th, 2019, the National Healthcare Security Administration (NHSA) and the Ministry of Human Resources and Social Security (MOHRSS) of China announced the results of drug pricing negotiations. Seventy first-negotiated drugs with 60.7% average price decrease and twenty-seven re-negotiated medicines with 26.4% average price fall, involving 11 disease categories, were successfully incorporated into National Reimbursement Drug List (NRDL). Medicines that successfully get accessed to NRDL are mostly new listings with high clinical value, and more than half of them are manufactured by Chinese enterprises. Compared to the negotiated drug list of 2017, the biggest increase in western medicines is the digestive system medications (10 drugs added), and the traditional Chinese medicine is internal medicine (17 drugs added). The negotiation follows the process including preparation, examination, negotiation, and announcement. There are several innovations in the procedure, such as the parallel calculation of the floor price, the introduction to competitive negotiations, allowing companies to apply for price confidentiality, and increasing government-enterprise communication before negotiations. Incorporating patent drugs into NRDL by negotiation not only helps patients reduce the economic burden, but also encourages pharmaceutical companies to innovate.

Gasto privado com medicamentos entre idosos e o comprometimento da renda familiar em município de médio porte no estado de São Paulo / Medicines' private costs among elderly and the impairment of family income in a medium-sized municipality in the state of São Paulo

RESUMO: Introdução: A aquisição de medicamentos responde por proporção importante dos gastos privados em saúde. O objetivo deste trabalho foi analisar o gasto privado com a compra de medicamentos e o comprometimento da renda familiar por idosos. Métodos: Inquérito populacional realizado em Praia Grande, São Paulo, 2013. O gasto mensal e o comprometimento da renda familiar per capita com a compra de medicamentos foram calculados com base nas informações obtidas nas entrevistas. As variáveis foram descritas em frequências absolutas e relativas, e os testes de hipótese utilizados foram o χ2 de Pearson, o t de Student e a análise de variância (Anova), com nível de significância de 5%. Resultados: A prevalência de utilização de medicamentos foi de 61,2%, e o gasto médio mensal per capita, de R$ 34,59, sendo significativamente maior o comprometimento da renda para os indivíduos com maior escolaridade, sem doenças crônicas e beneficiários de planos de saúde. Conclusão: A prevalência de utilização de medicamentos foi baixa. O custo gerado pela aquisição de medicamentos é uma das formas pelas quais pode se manifestar a desigualdade na sociedade. A ampliação da provisão gratuita de medicamentos seria necessária para expandir o acesso e evitar gastos, sobretudo àqueles que possuem planos de saúde privados, mas que não conseguem arcar com as despesas de tratamento medicamentoso.

ABSTRACT: Introduction: The acquisition of medicines accounts for a significant proportion of private health expenditures. The objective of this study was to analyse the private spending with the purchase of medicines and the commitment of the family income, by the elderly. Methods: Population survey conducted in Praia Grande, São Paulo, Brazil. The monthly expenditure and the per capita family income commitment with the purchase of medicines were calculated from the information obtained in the interviews. The variables were described in absolute and relative frequencies and the hypothesis test was Pearson's χ2, Student's t and Anova, with a significance level of 5%. Results: The prevalence of drug use was 61.2%. The average monthly expenditure per capita was R$ 34.59, with significantly higher income impairment for individuals with higher levels of education, without chronic diseases and health plan beneficiaries. Conclusion: The prevalence of drug use was low. The cost generated by the purchase of medicines is one of the ways in which inequality can manifest in society. The expansion of free drug provision would be necessary to expand access and avoid spending, especially those who have private health plans but cannot afford drug treatment.

Budget Impact of Dabrafenib and Trametinib in Combination as Adjuvant Treatment of BRAF V600E/K Mutation-Positive Melanoma from a U.S. Commercial Payer Perspective.

BACKGROUND: Before the approval of dabrafenib and trametinib in combination, there were no approved therapies in the adjuvant setting that target the RAS/RAF/MEK/ERK pathway. OBJECTIVE: To evaluate the budget impact of dabrafenib and trametinib in combination for adjuvant treatment of patients with BRAF V600 mutation-positive resected Stage IIIA, IIIB, or IIIC melanoma from a U.S. commercial payer perspective using data from the COMBI-AD trial, as well as other sources. METHODS: The budget impact of dabrafenib and trametinib in combination for patients with BRAF V600E/K mutation-positive, resected Stage IIIA, IIIB, or IIIC melanoma was evaluated from the perspective of a hypothetical population of 1 million members with demographic characteristics consistent with those of a commercially insured U.S. insurance plan (i.e., adults aged less than 65 years) using an economic model developed in Microsoft Excel. The model compared melanoma-related health care costs over a 3-year projection period under 2 scenarios: (1) a reference scenario in which dabrafenib and trametinib are assumed to be unavailable for adjuvant therapy and (2) a new scenario in which the combination is assumed to be available. Treatments potentially displaced by dabrafenib and trametinib were assumed to include observation, high-dose interferon alpha-2b, ipilimumab, and nivolumab. Costs considered in the model include those of adjuvant therapies and treatment of locoregional and distant recurrences. The numbers of patients eligible for treatment with dabrafenib and trametinib were based on data from cancer registries, published sources, and assumptions. Treatment mixes under the reference and new scenarios were based on market research data, clinical expert opinion, and assumptions. Probabilities of recurrence and death were based on data from the COMBI-AD trial and an indirect treatment comparison. Medication costs were based on wholesale acquisition cost prices. Costs of distant recurrence were from a health insurance claims study. RESULTS: In a hypothetical population of 1 million commercially insured members, 48 patients were estimated to become eligible for treatment with dabrafenib and trametinib in combination over the 3-year projection period; in the new scenario, 10 patients were projected to receive such treatment. Cumulative costs of melanoma-related care were estimated to be $6.3 million in the reference scenario and $6.9 million in the new scenario. The budget impact of dabrafenib and trametinib in combination was an increase of $549 thousand overall and 1.5 cents per member per month. CONCLUSIONS: For a hypothetical U.S. commercial health plan of 1 million members, the budget impact of dabrafenib and trametinib in combination as adjuvant treatment for melanoma is likely to be relatively modest and within the range of published estimates for oncology therapies. These results may assist payers in making coverage decisions regarding the use of adjuvant dabrafenib and trametinib in melanoma. DISCLOSURES: Funding for this research was provided to Policy Analysis Inc. (PAI) by Novartis Pharmaceuticals. Stellato, Moynahan, and Delea are employed by PAI. Ndife, Koruth, Mishra, and Gunda are employed by Novartis. Ghate was employed by Novartis at the time of this study and is shareholder in Novartis, Provectus Biopharmaceuticals, and Mannkind Corporation. Gerbasi was employed by PAI at the time of this study and is currently an employee, and stockholder, of Sage Therapeutics. Delea reports grant funding from Merck and research funding from Amgen, Novartis, Sanofi, Seattle Genetics, Takeda, Jazz, EMD Serono, and 21st Century Oncology, unrelated to this work.

Impact of China's healthcare price reforms on traditional Chinese medicine public hospitals in Beijing: an interrupted time-series study.

OBJECTIVE: To evaluate the 2017 implementation of China's 2009 healthcare price reforms on Beijing's secondary and tertiary traditional Chinese medicine (TCM) hospitals. DESIGN: We employed a panel-interrupted time-series model with hospital fixed effects to estimate the impact of the price reforms. SETTING: Beijing, April 2014 to April 2018. PARTICIPANTS: All TCM hospitals in Beijing. OUTCOME MEASURES: Our dependent variables comprised the monthly outpatient and inpatient revenues, the number of monthly outpatient visits and inpatient admissions, the average total expenditures per outpatient visit and per inpatient admission, the average drug expenditures (except herbal medicines) per outpatient visit and per inpatient admission and the average medical service expenditures per outpatient visit and per inpatient admission. RESULTS: In tertiary hospitals, the price reforms led to significant reductions in the number of outpatient visits (23.1%), inpatients admission (4.6%) and drug expenditures (except herbal medicines) per inpatient admission (14.0%), and an instant raise in average total expenditure per outpatient (22.0%), medical service expenditures per outpatient visit (58.2%) and inpatient admission (19.0%). There was no significant association between the price reforms and the monthly outpatient and inpatient revenues. After the price reforms, the previous upward trend in medical service expenditures per outpatient visit rose more sharply (from 0.5% to 1.6%). In secondary hospitals, the price reforms decreased the level of drug expenditures (except herbal medicines) per outpatient visit (13.0%) and per inpatient admission (20.8%), but increased medical service expenditures per inpatient admission by 19.0%. CONCLUSION: The Beijing price reforms adjusted the cost structures in secondary and tertiary TCM hospitals without negatively impacting the operation of the hospitals, and through the increased hierarchical medical service fee, shifted patient choices away from tertiary to other health facilities, especially for patients with minor illnesses.

"The biggest reform to China's health system": did the zero-markup drug policy achieve its goal at traditional Chinese medicines county hospitals?

The zero-markup drug policy (ZMDP) was heralded as the biggest reform to China's modern health system. However, there have been a very limited number of investigations of the ZMDP at county hospital level, and those limited county hospital studies have several limitations in terms of sample representativeness and study design. We investigated the overall and dynamic effects of ZMDP at traditional Chinese medicine (TCM) county hospitals. We obtained longitudinal data from all TCM county hospitals in 2004-16 and the implementation year of ZMDP for each hospital. We used differences-in-difference methods to identify the overall and dynamic effects of ZMDP. On average, the ZMDP reform was associated with the reduction in the share of revenue from drug sales (3.1%), revenue from western medicines sales (12.7%), revenue from medical care services (3.6%) and gross hospital revenue (3.4%), as well as increased government subsidies (24.4%). The ZMDP reform was not significantly associated with the number of annual outpatient and inpatient visits. In terms of dynamic effects, the share of revenue from drug sales decreased by 2.5% in the implementation year and by about 5% in the subsequent years. Revenue from western medicine sales fell substantially in the short term and continued to drop in the long term. Government subsidies went up strikingly in the short term and long term, and revenue from medical care services and gross revenue decreased only in the implementation year. The ZMDP achieved its stated goal through reducing the share of revenue from drug sales without disrupting the availability of healthcare services at TCM county hospitals. The success of ZMDP was mainly due to the huge growth in the government's financial investment in TCM hospitals.

Inter-brand competition in the convenience store industry, store density and healthcare utilization.

We investigate the impact of access to convenience stores and competition between convenience store chains on the use of medical care in Taiwan. Using insurance claims from 0.85 million individuals and administrative data on store sales, we find that greater store density and more inter-brand competition reduced expenditures on outpatient medical services and prescription drugs. In support of these findings, we demonstrate that convenience store competition was associated with greater consumption of healthy foods and lower obesity rates. Our estimates suggest that the rise in convenience store competition from 2002 to 2012 reduced outpatient expenditures in Taiwan by 0.44 percent and prescription drug expenditures by 0.85 percent.

Economic Evaluation of Anticyclic Citrullinated Peptide Positivity in Rheumatoid Arthritis.

BACKGROUND: Anticyclic citrullinated peptide (anti-CCP) positivity may be a strong predictor of joint erosion and a potential biomarker for guiding treatment decisions for rheumatoid arthritis (RA). However, limited studies are currently available on the effect of anti-CCP positivity on health care utilization and/or medical costs of RA patients. OBJECTIVE: To investigate short-term and long-term direct health care expenditures associated with anti-CCP positivity in newly diagnosed RA patients. METHODS: A retrospective cohort study was conducted in adult RA patients within a U.S. integrated health care delivery system (January 1, 2007-June 30, 2015). Patients were required to have 2 RA diagnoses and treatment with a conventional or biologic disease-modifying antirheumatic drug (DMARD) within 12 months. The first RA diagnosis date was labeled as the index date, and patients were followed until they left the health plan, died, or reached the end of the study period. Patient demographics, anti-CCP results, comorbid conditions, and health care resource utilization during baseline (12 months before the index date) and follow-up periods were collected. Nationally recognized direct medical costs were assigned to health care utilization to calculate health care costs in 2015 U.S. dollars. The baseline differences between anti-CCP positivity and negativity and differences in censoring during follow-up were addressed using propensity scores. The mean differences in costs were estimated using recycled prediction methods. RESULTS: 2,448 newly diagnosed RA patients were identified and followed for a median of 3.7 years (range = 1-8 years). At baseline, 65.8% of patients were anti-CCP positive. Anti-CCP-positive patients had fewer comorbid conditions at baseline. During the first 12 months of follow-up, median (interquartile range) total health care expenditures for anti-CCP-positive and anti-CCP-negative patients were $6,200 ($3,563-$13,260) and $7,022 ($3,885-$12,995), respectively. After adjusting for baseline differences, total incremental mean cost associated with anti-CCP positivity during the first 12 months was estimated to be $2,163 per patient (P = 0.001). The annual incremental costs in anti-CCP-positive patients became progressively larger over time, from $2,163 during the first year to $5,062 during the fourth year. Anti-CCP positivity was associated with higher prescription, laboratory testing, and rheumatologist utilization. A higher percentage of anti-CCP-positive patients received 1 or more biologic DMARDs (11.6% for anti-CCP-positive vs. 5.7% for anti-CCP negative; P < 0.001) compared with anti-CCP-negative patients during the 12-month follow-up, which resulted in $2,499 in incremental prescription costs (P < 0.001). Total additional burden associated with anti-CCP positivity during the first 4 years was estimated to be $14,089 per patient. CONCLUSIONS: In newly diagnosed RA patients, higher economic burden associated with anti-CCP positivity was mainly driven by prescription costs. DISCLOSURES: This research and manuscript were funded by Bristol-Myers Squibb (BMS). Alemao and Connolly are employees and shareholders of BMS and participated in the design of the study, interpretation of the data, review/revision of the manuscript, and approval of the final version of the manuscript. An and Cheetham received a grant from BMS for this research. At the time of this study, An was employed by Western University of Health Sciences, and Cheetham was employed by Kaiser Permanente Southern California. Bider-Canfield, Kang, and Lin have nothing to disclose. Some study results were presented as a poster at the American College of Rheumatology Annual Meeting; November 5, 2017; San Diego, CA, and at the International Society for Pharmacoeconomics and Outcomes Research Meeting; May 19, 2018; Baltimore, MD.

The effect of a change in co-payment on prescription drug demand in a National Health System: The case of 15 drug families by price elasticity of demand.

OBJECTIVES: To test the heterogeneity of the effect of a change in pharmaceutical cost-sharing by therapeutic groups in a Spanish region. METHODS: Data: random sample (provided by the Canary Islands Health Service) of 40,471 people covered by the Spanish National Health System (SNHS) in the Canary Islands. The database includes individualised monthly-dispensed medications (prescribed by the SNHS) from one year before (August 2011) to one year after (June 2013) the Royal Decree Law 16/2012 (RDL 16/2012). Sample: two intervention groups (low-income pensioners and middle-income working population) and one control group (low-income working population). Empirical model: quasi-experimental difference-in-differences design to study the change in consumption (measured in number of monthly Defined Daily Dose (DDDs) per individual) among 13 therapeutic groups. The policy break indicator (three-level categorical variable) tested the existence of stockpiling between the reform's announcement and its implementation. We ran 16 linear regression models (general, by therapeutic groups and by comorbidities) that considered whether the exclusion of some drugs from public provision impacted on consumption more than the co-payment increase. RESULTS: General: Reduction (-13.04) in consumption after the reform's implementation, which was fully compensated by a previous increase (16.60 i.e., stockpiling) among low-income pensioners. The middle-income working population maintained its trend of increasing consumption. Therapeutic groups: Reductions in consumption after the reform's implementation among low-income pensioners in 7 of the 13 groups, which were fully compensated for by a previous increase (i.e., stockpiling) in 4 groups and partially compensated for in the remaining 3. The analysis without the excluded medicines provided fewer negative coefficients. Comorbidities: Reduction in consumption that was only slightly compensated for by a previous increase (i.e., stockpiling). CONCLUSIONS: The negative impact of cost-sharing produced, among low-income pensioners, a risk of loss of adherence to treatments, which could deteriorate the health status of individuals, especially among pensioners within the most inelastic therapeutic groups (associated with chronic diseases) and patients with comorbidities (also, associated with chronic diseases). Notwithstanding the above, this risk was more related to the exclusion of some drugs from provision than to the cost-sharing increase.