Putting Stakeholder Engagement at the Center of Health Economic Modeling for Health Technology Assessment in the United States.

While evidence generated from health economic (HE) models is being used more commonly in health technology assessment (HTA) in the US, it is not consistently adopted by different stakeholder groups or across therapeutic areas. We hypothesize that actively engaging with multiple stakeholder groups throughout the model development process may result in models more widely considered by decision makers. To test this hypothesis, the Innovation and Value Initiative has launched a modeling effort to build an open-source HE model focusing on the disease state 'major depressive disorder'. A 20-member advisory group has been formed with representatives from patients, employers, clinicians, innovators, payers, and researchers to guide the model development process. While this effort is still in the early stages, the ongoing stakeholder engagement effort has yielded valuable insights that inform the model design. We have also identified several challenges to implementing this new approach. Our early findings suggest that the stakeholder engagement approach to HE model development has the potential to improve HTA in the US.

Developing Open-Source Models for the US Health System: Practical Experiences and Challenges to Date with the Open-Source Value Project.

The Innovation and Value Initiative started the Open-Source Value Project with the aim to improve the credibility and relevance of model-based value assessment in the context of the US healthcare environment. As a core activity of the Open-Source Value Project, the Innovation and Value Initiative develops and provides access to flexible open-source economic models that are developed iteratively based on public feedback and input. In this article, we describe our experience to date with the development of two currently released, Open-Source Value Project models, one in rheumatoid arthritis and one in epidermal growth factor receptor-positive non-small-cell lung cancer. We developed both Open-Source Value Project models using the statistical programming language R instead of spreadsheet software (i.e., Excel), which allows the models to capture multiple model structures, model sequential treatment with individual patient simulations, and improve integration with formal evidence synthesis. By developing the models in R, we were also able to use version control systems to manage changes to the source code, which is needed for iterative and collaborative model development. Similarly, Open-Source Value Project models are freely available to the public to provide maximum transparency and facilitate collaboration. Development of the rheumatoid arthritis and non-small-cell lung cancer model platforms has presented multiple challenges. The development of multiple components of the model platform tailored to different audiences, including web interfaces, required more resources than a cost-effectiveness analysis for a publication would. Furthermore, we faced methodological hurdles, in particular related to the incorporation of multiple competing model structures and novel elements of value. The iterative development based on public feedback also posed some challenges during the review phase, where methodological experts did not always understand feedback from clinicians and vice versa. Response to the Open-Source Value Project by the modeling community and patient organizations has been positive, but feedback from US decision makers has been limited to date. As we progress with this project, we hope to learn more about the feasibility, benefits, and challenges of an open-source and collaborative approach to model development for value assessment.

Share of Oncology Versus Nononcology Spending in Episodes Defined by the Centers for Medicare & Medicaid Services Oncology Care Model.

PURPOSE: Performance-based payments to oncology providers participating in the Centers for Medicare & Medicaid Services (CMS) Oncology Care Model (OCM) are based, in part, on overall spending in 6-month episodes of care, including spending unrelated to oncology care. The amount of spending likely to occur outside of oncologists' purview is unknown. METHODS: Following the OCM definition of an episode, we used SEER-Medicare data from 2006 to 2013 to identify episodes of cancer care for the following diagnoses: breast cancer (BC), non-small-cell lung cancer, renal cell carcinoma, multiple myeloma (MM), and chronic myeloid leukemia. Claims were categorized by service type and, separately, whether the content fell within the purview of oncology providers (classified as oncology, with all other claims nononcology). We calculated the shares of episode spending attributable to oncology versus nononcology services. RESULTS: The percentage of oncology spending within OCM episodes ranged from 62.4% in BC to 85.5% in MM. The largest source of oncology spending was antineoplastic drug therapy, ranging from 21.8% of total episode spending in BC to 67.6% in chronic myeloid leukemia. The largest source of nononcology spending was acute hospitalization and inpatient physician costs, ranging from 6.6% of overall spending for MM to 10.4% for non-small-cell lung cancer; inpatient oncology spending contributed roughly similar shares to overall spending. CONCLUSION: Most spending in OCM-defined episodes was attributable to services related to cancer care, especially antineoplastic drug therapy. Inability to control nononcology spending may present challenges for practices participating in the OCM, however.

Value of expanding HCV screening and treatment policies in the United States.

OBJECTIVES: To investigate the value of expanding screening and treatment for hepatitis C virus (HCV) infection in the United States. STUDY DESIGN: Discrete-time Markov model. METHODS: We modeled HCV progression and transmission to analyze the costs and benefits of investment in screening and treatment over a 20-year time horizon. Population-level parameters were estimated using National Health and Nutrition Examination Survey data and published literature. We considered 3 screening scenarios that vary in terms of clinical guidelines and physician awareness of guidelines. For each screening scenario, we modeled 3 approaches to treatment, varying the fibrosis stage of treatment initiation. Net social value was the key model outcome, calculated as the value of benefits from improved quality-adjusted survival and reduced transmission minus screening, treatment, and medical costs. RESULTS: Expanded screening policies generated the largest value to society. However, this value is constrained by the availability of treatment to diagnosed patients. Screening all individuals in the population generates $0.68 billion in social value if diagnosed patients are treated in fibrosis stages F3-F4 compared with $824 billion if all diagnosed patients in stages F0-F4 are treated. Moreover, increased screening generates cumulative net social value by year 8 to 9 under expanded treatment policies compared with 20 years if only patients in stages F3-F4 are treated. CONCLUSIONS: Although increasing screening for HCV may generate some value to society, only when paired with expanded access to treatment at earlier disease stages will it produce considerable value. Such a "test and treat" strategy is likely to entail higher short-term costs but also yield the greatest social benefits.

Costs and spillover effects of private insurers' coverage of hepatitis C treatment.

OBJECTIVES: Hepatitis C virus (HCV) treatment incentives for private payers may be misaligned because payers must bear immediate costs and may not realize long-term benefits. However, these benefits may accrue to future payers, including Medicare. We examined how and to what extent private payers' current HCV treatment coverage decisions impact Medicare's and private payers' future costs. STUDY DESIGN: Discrete-time Markov model. METHODS: We modeled HCV disease progression and transmission to simulate the economic and social effects of different private-payer HCV treatment scenarios on Medicare. The model examined differences between a baseline scenario (current practice guidelines) and 2 alternative scenarios that expand treatment coverage. Spillover effects were measured as reduced HCV treatment costs and medical expenditures in Medicare. We calculated the spillover effects and net social value of each scenario (total value of quality-adjusted life-years accrued over time minus cumulative treatment and medical costs). RESULTS: With expanded HCV treatment coverage, private payers experience reduced medical expenditures in the 3-to-5-year time horizon; however, they still face higher treatment costs. Over a 20-year horizon, private payers experience overall savings of $10 billion to $14 billion after treatment costs. The expansion of coverage by private payers generates positive spillover benefits to Medicare of $0.3 billion to $0.7 billion over a 5-year horizon, and $4 billion to $11 billion over a 20-year horizon. CONCLUSIONS: When private payers increase HCV treatment coverage, they may achieve significant savings while inducing spillover benefits to Medicare. Future savings, however, may not motivate immediate treatment investments among private payers who experience high beneficiary turnover.

Evaluating Expected Costs and Benefits of Granting Access to New Treatments on the Basis of Progression-Free Survival in Non-Small-Cell Lung Cancer.

IMPORTANCE: Surrogate end points may be used as proxy for more robust clinical end points. One prominent example is the use of progression-free survival (PFS) as a surrogate for overall survival (OS) in trials for oncologic treatments. Decisions based on surrogate end points may expedite regulatory approval but may not accurately reflect drug efficacy. Payers and clinicians must balance the potential benefits of earlier treatment access based on surrogate end points against the risks of clinical uncertainty. OBJECTIVE: To present a framework for evaluating the expected net benefit or cost of providing early access to new treatments on the basis of evidence of PFS benefits before OS results are available, using non-small-cell lung cancer (NSCLC) as an example. DESIGN, SETTING, AND PARTICIPANTS: A probabilistic decision model was used to estimate expected incremental social value of the decision to grant access to a new treatment on the basis of PFS evidence. The model analyzed a hypothetical population of patients with NSCLC who could be treated during the period between PFS and OS evidence publication. Estimates for delay in publication of OS evidence following publication of PFS evidence, expected OS benefit given PFS benefit, incremental cost of new treatment, and other parameters were drawn from the literature on treatment of NSCLC. MAIN OUTCOMES AND MEASURES: Incremental social value of early access for each additional patient per month (in 2014 US dollars). RESULTS: For "medium-value" model parameters, early reimbursement of drugs with any PFS benefit yields an incremental social cost of more than $170,000 per newly treated patient per month. In contrast, granting early access on the basis of PFS benefit between 1 and 3.5 months produces more than $73,000 in incremental social value. Across the full range of model parameter values, granting access for drugs with PFS benefit between 3 and 3.5 months is robustly beneficial, generating incremental social value ranging from $38,000 to more than $1 million per newly treated patient per month, whereas access for all drugs with any PFS benefit is usually not beneficial. CONCLUSIONS AND RELEVANCE: The value of providing access to new treatments on the basis of surrogate end points, and PFS in particular, likely varies considerably. Payers and clinicians should carefully consider how to use PFS data in balancing potential benefits against costs in each particular disease.

Clinical evidence inputs to comparative effectiveness research could impact the development of novel treatments.

AIM: This study aims to analyze the impacts of a range of clinical evidence generation scenarios associated with comparative effectiveness research (CER) on pharmaceutical innovation. MATERIALS & METHODS: We used the Global Pharmaceutical Policy Model to project the effect of changes in pharmaceutical producer costs, revenues and timings on drug innovation and health for the age 55+ populations in the USA and Europe through year 2060 using three clinical scenarios. RESULTS: Changes in producer incentives from widespread CER evidence generation and use had varied but often large predicted impacts on simulated outcomes in 2060. Effect on the number of new drug introductions ranged from a 81.1% reduction to a 45.5% increase, and the effect on population-level life expectancy ranged from a 15.6% reduction to a 11.4% increase compared to baseline estimates. CONCLUSION: The uncertainty surrounding the consequences of increased clinical evidence generation and use on innovation calls for a carefully measured approach to CER implementation, balancing near-term benefits to spending and health with long-term implications for innovation.

Cancer mortality reductions were greatest among countries where cancer care spending rose the most, 1995-2007.

Health care spending and health outcomes vary markedly across countries, but the association between spending and outcomes remains unclear. This inevitably raises questions as to whether continuing growth in spending is justified, especially relative to the rising cost of cancer care. We compared cancer care across sixteen countries over time, examining changes in cancer spending and two measures of cancer mortality (amenable and excess mortality). We found that compared to low-spending health systems, high-spending systems had consistently lower cancer mortality in the period 1995-2007. Similarly, we found that the countries that increased spending the most had a 17 percent decrease in amenable mortality, compared to 8 percent in the countries with the lowest growth in cancer spending. For excess mortality, the corresponding decreases were 13 percent and 9 percent. Additionally, the rate of decrease for the countries with the highest spending growth was faster than the all-country trend. These findings are consistent with the existence of a link between higher cancer spending and lower cancer mortality. However, further work is needed to investigate the mechanisms that underlie this correlation.

Economic burden of disease-associated malnutrition in China.

Disease-associated malnutrition (DAM) is a well-recognized problem in many countries, but the extent of its burden on the Chinese population is unclear. This article reports the results of a burden-of-illness study on DAM in 15 diseases in China. Using data from the World Health Organization (WHO), the China Health and Nutrition Survey, and the published literature, mortality and disability-adjusted life years (DALYs) lost because of DAM were calculated; a financial value of this burden was calculated following WHO guidelines. DALYs lost annually to DAM in China varied across diseases, from a low of 2248 in malaria to a high of 1 315 276 in chronic obstructive pulmonary disease. The total burden was 6.1 million DALYs, for an economic burden of US$66 billion (Chinese ¥ 447 billion) annually. This burden is sufficiently large to warrant immediate attention from public health officials and medical providers, especially given that low-cost and effective interventions are available.

Effect of hospital use of oral nutritional supplementation on length of stay, hospital cost, and 30-day readmissions among Medicare patients with COPD.

BACKGROUND: COPD is a leading cause of death and disability in the United States. Patients with COPD are at a high risk of nutritional deficiency, which is associated with declines in respiratory function, lean body mass and strength, and immune function. Although oral nutritional supplementation (ONS) has been associated with improvements in some of these domains, the impact of hospital ONS on readmission risk, length of stay (LOS), and cost among hospitalized patients is unknown. METHODS: Using the Premier Research Database, we first identified Medicare patients aged ≥ 65 years hospitalized with a primary diagnosis of COPD. We then identified hospitalizations in which ONS was provided, and used propensity-score matching to compare LOS, hospitalization cost, and 30-day readmission rates in a one-to-one matched sample of ONS and non-ONS hospitalizations. To further address selection bias among patients prescribed ONS, we also used instrumental variables analysis to study the association of ONS with study outcomes. Model covariates included patient and provider characteristics and a time trend. RESULTS: Out of 10,322 ONS hospitalizations and 368,097 non-ONS hospitalizations, a one-to-one matched sample was created (N = 14,326). In unadjusted comparisons in the matched sample, ONS use was associated with longer LOS (8.7 days vs 6.9 days, P < .0001), higher hospitalization cost ($14,223 vs $9,340, P < .0001), and lower readmission rates (24.8% vs 26.6%, P = .0116). However, in instrumental variables analysis, ONS use was associated with a 1.9-day (21.5%) decrease in LOS, from 8.8 to 6.9 days (P < .01); a hospitalization cost reduction of $1,570 (12.5%), from $12,523 to $10,953 (P < .01); and a 13.1% decrease in probability of 30-day readmission, from 0.34 to 0.29 (P < .01). CONCLUSIONS: ONS may be associated with reduced LOS, hospitalization cost, and readmission risk in hospitalized Medicare patients with COPD.

Economic burden of community-based disease-associated malnutrition in the United States.

BACKGROUND: The burden imposed by disease-associated malnutrition (DAM) on patients and the healthcare system in food-abundant industrialized countries is often underappreciated. This study measured the economic burden of community-based DAM in the United States. METHODS: The burden of DAM was quantified in terms of direct medical costs, quality-adjusted life years lost, and mortality across 8 diseases (breast cancer, chronic obstructive pulmonary disease [COPD], colorectal cancer [CRC], coronary heart disease [CHD], dementia, depression, musculoskeletal disorders, and stroke). To estimate the total economic burden, the morbidity and mortality burden was monetized using a standard value of a life year and combined with direct medical costs of treating DAM. Disease-specific prevalence and malnutrition estimates were taken from the National Health Interview Survey and the National Health and Nutrition Examination Survey. Deaths by disease were taken from the Center for Disease Control and Prevention. Estimates of costs and morbidity were taken from the literature. RESULTS: The annual burden of DAM across the 8 diseases was $156.7 billion, or $508 per U.S. resident. Nearly 80% of this burden was derived from morbidity associated with DAM; around 16% derived from mortality and the remainder from direct medical costs of treating DAM. The total burden was highest in COPD and depression, while the burden per malnourished individual was highest in CRC and CHD. CONCLUSION: DAM exacts a large burden on American society. Therefore, improved diagnosis and management of community-based DAM to alleviate this burden are needed.

Impact of oral nutrition supplements on hospital outcomes in pediatric patients.

BACKGROUND: Nutrition deficiency is common among hospitalized children. Although oral nutrition supplements (ONS) may improve malnutrition in this population, the benefits and healthcare costs associated with their use have not yet been fully explored. The objective of this study was to assess the effect of ONS use on inpatient length of stay (LOS) and episode cost in hospitalized children. MATERIALS AND METHODS: Retrospective analysis of 557,348 hospitalizations of children aged 2-8 years in the Premier Research Database. The effect of ONS use on LOS and episode cost in a propensity score- matched sample was estimated in analyses with and without the use of instrumental variables (IVs) to reduce confounding from unobserved variables. RESULTS: ONS were prescribed in 6066 of 557,348 inpatient episodes (1.09%). In IV analysis, using a matched sample of 11,031 episodes, hospitalizations with ONS use had 14.8% shorter LOS (6.4 vs 7.5 days; 1.1 days [95% CI, 0.2-2.4]). Hospitalizations with ONS use had 9.7% lower cost ($16,552 vs $18,320; $1768 [95% CI, $1924-$1612]). CONCLUSIONS: ONS use was associated with lower LOS and episode cost among pediatric inpatients. ONS use in hospitalized pediatric patients may provide a cost-effective, evidence-based approach to improving pediatric hospital care.

Tutorial on health economics and outcomes research in nutrition.

As healthcare costs climb around the world, public and private payers alike are demanding evidence of a treatment's value to support approval and reimbursement decisions. Health economics and outcomes research, or HEOR, offers tools to answer questions about a treatment's value, as well as its real-world effects and cost-effectiveness. Given that nutrition interventions have to compete for space in budgets along with biopharmaceutical products and devices, nutrition is now increasingly coming to be evaluated through HEOR. This tutorial introduces the discipline of HEOR and motivates its relevance for nutrition. We first define HEOR and explain its role and relevance in relation to randomized controlled trials. Common HEOR study types--including burden of illness, effectiveness studies, cost-effectiveness analysis, and valuation studies--are presented, with applications to nutrition. Tips for critically reading HEOR studies are provided, along with suggestions on how to use HEOR to improve patient care. Directions for future research are discussed.

Reforming medicare's dialysis payment policies: implications for patients with secondary hyperparathyroidism.

OBJECTIVE: To demonstrate how expanding services covered by a "bundled payment" can also expand variation in the costs of treating patients under the bundle, using the Medicare dialysis program as an example. DATA SOURCES/STUDY SETTING: Observational claims-based study of 197,332 Medicare hemodialysis beneficiaries enrolled for at least one quarter during 2006-2008. STUDY DESIGN: We estimated how resource utilization (all health services, dialysis-related services, and medications) changes with intensity of secondary hyperparathyroidism (sHPT) treatment. DATA EXTRACTION METHODS: Using Medicare claims, a patient-quarter level dataset was constructed, including a measure of sHPT treatment intensity. PRINCIPAL FINDINGS: Under the existing, narrow dialysis bundle, utilization of covered services is relatively constant across treatment intensity groups; under a broader bundle, it rises more rapidly with treatment intensity. CONCLUSIONS: The broader Medicare dialysis bundle reimburses providers uniformly, even though patients treated more intensively for sHPT cost more to treat. Absent any payment adjustments or efforts to ensure quality, this flat payment schedule may encourage providers to avoid high-intensity patients or reduce their treatment intensity. The first incentive harms efficiency. The second may improve or worsen efficiency, depending on whether it reduces appropriate or inappropriate treatment.

Early HIV treatment in the United States prevented nearly 13,500 infections per year during 1996-2009.

In recent years, guidelines for HIV treatment have recommended initiation of combination antiretroviral therapy (cART) earlier in the course of the disease than was previously the case. These recommendations stem in part from growing evidence that treatment reduces the risk of sexual transmission. We used an epidemiological model of disease transmission and progression to assess HIV prevention through early treatment-that is, initiation of cART when CD4 white blood cell counts are in excess of 350 cells per cubic millimeter. (CD4 cells are involved in the immune system's defense against tumors and infection; the number of CD4 cells in a cubic millimeter of blood is a standard measure of immune response to antiretroviral therapy.) We estimated that the actual timing of treatment initiation in the United States prevented 188,000 HIV cases in the period 1996-2009. "Very early" treatment (at CD4 counts greater than 500) accounted for four-fifths of the prevented cases. For all of the prevented cases, the losses in life expectancy that were avoided were worth $128 billion, assuming that a life-year has a value of $150,000. These findings underscore the cost-effectiveness of early HIV treatment.

Data-driven decision-making tools to improve public resource allocation for care and prevention of HIV/AIDS.

Public health agencies face difficult decisions when allocating scarce resources to control the spread of HIV/AIDS. Decisions are often made with few local empirical data. We demonstrated the use of the robust decision making approach in Los Angeles County, an approach that is data driven and allows decision makers to compare the performance of various intervention strategies across thousands of simulated future scenarios. We found that the prevailing strategy of emphasizing behavioral risk reduction interventions was unlikely to achieve the policy goals of the national HIV/AIDS strategy. Of the alternative strategies we examined, those that invested most heavily in interventions to initiate antiretroviral treatment and support treatment adherence were the most likely to achieve policy objectives. By employing similar methods, other public health agencies can identify robust strategies and invest in interventions more likely to achieve HIV/AIDS policy goals.

The prospect of a generation free of HIV may be within reach if the right policy decisions are made.

Scientific advances have transformed HIV treatment and prevention, leading to the adoption of an approach that emphasizes broad testing and antiretroviral treatment at earlier stages in the disease, called "test and treat." In addition to clinical benefits, early treatment generates considerable social and economic value. These changes raise the prospect that for the first time since the 1980s, an entire generation might be free of HIV. However, achieving such a goal will require continued scientific advances and the presence of policies and programs to ensure that people living with HIV/AIDS have access to health care and adhere to treatment regimens. This article explores the opportunities and challenges that the Affordable Care Act (ACA) presents for people living with HIV/AIDS and discusses how the act's various components might interact with existing support for people with HIV/AIDS, such as the Ryan White Program. As the ACA's reforms proceed, coordinated state and federal programs must make smart policy choices so that critical access to and affordability of comprehensive care are maintained in the fight against HIV/AIDS.

Can Oral Nutritional Supplements Improve Medicare Patient Outcomes in the Hospital?

We analyzed the effect of oral nutritional supplement (ONS) use on 30-day readmission rates, length of stay (LOS), and episode costs in hospitalized Medicare patients (≥65), and subsets of patients diagnosed with acute myocardial infarction (AMI), congestive heart failure (CHF) or pneumonia (PNA). Propensity-score matching and instrumental variables were used to analyze ONS and non-ONS episodes from the Premier Research Database (2000-2010). ONS use was associated with reductions in probability of 30-day readmission by 12.0% in AMI and 10.1% in CHF. LOS decreases of 10.9% in AMI, 14.2% in CHF, and 8.5% in PNA were associated with ONS, as were decreases in episode costs in AMI, CHF and PNA of 5.1%, 7.8% and 10.6%, respectively. The effect on LOS and episode cost was greatest for the Any Diagnosis population, with decreases of 16.0% and 15.8%, respectively. ONS use in hospitalized Medicare patients ≥65 is associated with improved outcomes and decreased healthcare costs, and is therefore relevant to providers seeking an inexpensive, evidence-based approach for meeting Affordable Care Act quality targets.