Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program.

INTRODUCTION: The United States has begun assessing the value of pharmaceuticals to inform negotiated prices in the Medicare program. Given strong political objections in the United States to the use of QALYs, Medicare will need to adopt an alternative approach to measuring value. AREAS COVERED: In this narrative review, we identified six alternative approaches to measuring value (equal value life-years, health years in total, generalized risk-adjusted cost-effectiveness, severity weighting based on absolute or proportional shortfall, comparative effectiveness based on conventional clinical endpoints, and comparative effectiveness based on both conventional endpoints and patient-centric value elements) and five criteria for assessing these approaches (responsiveness to concerns about discrimination, feasibility, transparency, flexibility, and the ability to incorporate factors beyond traditional value elements). EXPERT OPINION: Four of the alternatives are broadly aligned with the cost-effectiveness framework, but none fully addresses all aspects of the stated concerns that QALYs may be used to unintentionally implement discrimination. We note, however, that the extent to which these concerns lead to discrimination in practice is unknown. Finally, we recommend an approach for measuring value in terms of comparative effectiveness that combines quantitative ranking and weighting of distinct criteria (including patient-centric value elements) with deliberation.

Cost and Return on Investment of a Team-Based Palliative Care Program for Parkinson Disease.

Implementation of palliative care (PC) in neurology settings may improve symptom control and quality of life and reduce acute care admissions. The benefits of team-based PC for patients with Parkinson disease have been established through rigorous evidence standards including randomized controlled trials. However, evidence on implementation costs and return on investment (ROI) is unknown and may guide other providers and systems considering this model of care. We applied time-driven activity-based costing with reimbursable visits calculated using Medicare reimbursement rates in Colorado and current procedural technology codes to 2 outpatient clinics at the University of Colorado Hospital: neurology PC and movement disorders. Per-patient ROI was calculated as the ratio of the incremental difference in financial revenues divided by the incremental difference in investment to expand PC services. The cost per new patient was $154 and $98 for neuropalliative and movement disorders clinics, respectively. Established patient visit costs were $82 and $41 for the neuropalliative care and movement disorders clinics, respectively. The neurology PC clinic had per-patient revenue for new and established visits of $297 and $147, respectively, compared with $203 and $141 for new and established visits, respectively, at the comparator clinic. Based on our assumptions, for every $1 invested in expanding PC services, a projected $1.68 will be recouped by the hospital system for new patient visits, and $0.13 will be recouped for established patient visits. These amounts are context dependent, and a calculator was created to allow other systems to estimate costs and ROI. Our results suggest that in an academic medical setting, both neurology PC and movement disorders clinics provided increased revenue to the health system. Opportunities to improve ROI include efficient allocation of personnel to new and established visits, expanding telemedicine, and other cost offsets for complex patients not estimated in this analysis. ROI may also be greater in health systems that benefit from cost savings such as accountable care organizations. Our approach may be applied to other novel care models. Future research efforts will focus on estimating the continued sustainability of this innovative outpatient care model.

Stakeholder perception of pharmaceutical value: A multicriteria decision analysis pilot case study for value assessment in the United States.

BACKGROUND: Recent attention to value frameworks has highlighted limitations of current conventional value and health technology assessment (V/HTA) methods (eg, cost-effectiveness). Multicriteria decision analysis (MCDA) has potential as a supplemental tool to incorporate additional value criteria into conventional value assessment. OBJECTIVE: To conduct a pilot study to illustrate the impact of an MCDA approach on the value perceptions of hypothetical treatment profiles from a multistakeholder panel. METHODS: Participants voted on value perceptions of 2 hypothetical treatments with similar cost-effectiveness evidence: Treatment A for aggressive B-cell non-Hodgkin lymphoma in adults and treatment B for episodic migraine in adults. Participants voted treatments A and B as low, intermediate, or high value before and after a weighting exercise on prespecified, additional value criteria. Weights from participants were used to calculate treatment-specific MCDA scores from 0 (least favorable) to 100 (most favorable) and were presented to participants for a second value-perception vote. Analyses compared changes in value perceptions within treatments A and B post-MCDA exercise. RESULTS: Before considering MCDA scores for treatment A, 0% of participants considered it to be low, 52% intermediate, and 48% high value. After considering MCDA scores for treatment A, 4% considered it low, 29% intermediate, and 67% high value. Both before and after considering MCDA scores for treatment B, 13%, considered it low, 57% intermediate, and 30% high value. Mean MCDA scores for treatments A and B were 67 and 63, respectively. Of all stakeholders, 41% altered their perception of value for treatment A (9% negatively and 32% positively) and, separately, 45% for treatment B (23% both negatively and positively) after considering MCDA scores. CONCLUSIONS: With nearly half of participants altering their perception of value after consideration of additional value criteria, findings support the need for a more inclusive and flexible value assessment process. DISCLOSURES: This study was funded by The National Pharmaceutical Council. Dr Perfetto was employed by the National Health Council (NHC) at the time this work was completed, and all honoraria and consulting and travel fees were paid to the NHC. The NHC is a not-for-profit, membership organization. It is supported through membership dues and sponsorship funds. The complete list of members and sponsors is located on the NHC's website at www.nationalhealthcouncil.org. She is also an advisor for the Brain Injury Association of America, Dan Lewis Foundation, and Canter for Medical Technology Policy.

Cost of Hospitalizations for Leading Foodborne Pathogens in the United States: Identification by International Classification of Disease Coding and Variation by Pathogen.

Foodborne illness is a continuing public health problem in the United States. Seven pathogens-Campylobacter, Clostridium perfringens, Shiga toxin-producing Escherichia coli O157, Listeria monocytogenes, nontyphoidal Salmonella, norovirus, and Toxoplasma gondii-are estimated to cause >90% of the foodborne illnesses, hospitalizations, and deaths attributed to 31 known pathogens. The purpose of this article was to inform estimates of the cost of hospitalizations associated with these pathogens using National Inpatient Survey data from January 2012 through September 2015. The article explored two methodological issues. First, is it more appropriate to use hospitalizations identified using principal or all diagnosis codes when estimating cost? Second, should pathogen-specific or overall mean cost estimates be used? After excluding C. perfringens because of low sample size, the remaining six pathogens included in the analysis were associated with 17,102 hospital discharge records. Of these 55% have the pathogen listed as a principal diagnosis (FBP-PD), ranging from 6% for T. gondii to 68% for nontyphoidal Salmonella. The mean per-case cost of records with the pathogen listed as a secondary diagnosis (FBP-SD) was 2.7 times higher than FBP-PD. FBP-SD were also more severe than FBP-PD with longer lengths of stay, increasing loss of function, and increasing risk of mortality. Severity was the main driver of cost. We also found severity of illness and cost of hospitalizations vary by pathogen. Based on identifying cases with a pathogen in either FBP-PD or FBP-SD, we found mean per-case hospitalization cost across the six pathogens included in this study was $17,515, ranging from $11,552 for Campylobacter to $34,206 for norovirus. In summary, if only FBP-PD cases were used to estimate cost, estimates would likely underestimate hospitalization costs among those cases with a pathogen-specific diagnosis. Because these foodborne pathogens varied in severity of illness, the mean cost of hospitalizations also varied significantly by pathogen.

Prognostic predictors relevant to end-of-life palliative care in Parkinson's disease and related disorders: a systematic review.

Parkinson's disease and related disorders (PDRD) are the second most common neurodegenerative disease and a leading cause of death. However, patients with PDRD receive less end-of-life palliative care (hospice) than other illnesses, including other neurologic illnesses. Identification of predictors of PDRD mortality may aid in increasing appropriate and timely referrals. To systematically review the literature for causes of death and predictors of mortality in PDRD to provide guidance regarding hospice/end-of-life palliative care referrals. We searched MEDLINE, PubMed, EMBASE and CINAHL databases (1970-2020) of original quantitative research using patient-level, provider-level or caregiver-level data from medical records, administrative data or survey responses associated with mortality, prognosis or cause of death in PDRD. Findings were reviewed by an International Working Group on PD and Palliative Care supported by the Parkinson's Foundation. Of 1183 research articles, 42 studies met our inclusion criteria. We found four main domains of factors associated with mortality in PDRD: (1) demographic and clinical markers (age, sex, body mass index and comorbid illnesses), (2) motor dysfunction and global disability, (3) falls and infections and (4) non-motor symptoms. We provide suggestions for consideration of timing of hospice/end-of-life palliative care referrals. Several clinical features of advancing disease may be useful in triggering end-of-life palliative/hospice referral. Prognostic studies focused on identifying when people with PDRD are nearing their final months of life are limited. There is further need for research in this area as well as policies that support need-based palliative care for the duration of PDRD.

Evaluation of Prophylactic Heparin Dosage Strategies and Risk Factors for Venous Thromboembolism in the Critically Ill Patient.

BACKGROUND: Venous thromboembolism (VTE) occurs frequently in critically ill patients without heparin prophylaxis. Although heparin prevents VTE, VTEs occur frequently despite prophylaxis. A higher heparin dosage may be more effective for preventing VTE. METHODS: A retrospective study was conducted using the Premier Incorporated Perspective Database to evaluate comparatively the effects of different heparin prophylaxis dosing strategies in the critically ill patient. Critically ill adult patients who were mechanically ventilated for at least 1 day and had an intensive care unit (ICU) length of stay of at least 2 days were included. Patients received 5000 units of heparin either twice/day or 3 times/day. The primary outcome was development of a new VTE. Key secondary outcomes included clinically important bleeding, thrombocytopenia, and mortality. Patients were propensity matched to control for confounding. Multivariable analysis was conducted for VTE risk factors. RESULTS: The study included 30,800 patients from 374 hospitals who were propensity matched by heparin dosage. New VTE occurred in 6.16% of patients treated with 3 times/day heparin versus 6.23% with twice/day heparin (p=0.8). No significant differences in the incidence of pulmonary embolism (0.91% vs 0.8%, p=0.29) or deep vein thrombosis (5.56% vs 5.70% p=0.59) were observed between the two types of heparin dosing. No differences were observed between the two types of heparin dosing in in-hospital mortality (15.8% vs 15.15%), bleeding (0.23% vs 0.33%), or thrombocytopenia (5.19% vs 5.34%, p>0.08 for all), respectively. Risk factors associated with VTE included intraabdominal and urinary tract infections, loop diuretics, malnutrition, obesity, thrombocytopenia, paralytics, vasopressors, female sex, peripheral vascular disease, sepsis, neutropenia, and end-stage renal disease. Antiplatelet therapy, heart failure, diabetes, and substance abuse were associated with reduced VTE (p<0.05 for all). CONCLUSIONS: In critically ill patients, prophylactic dosing of heparin 3 times/day versus twice/day was not associated with differences in new VTE or safety outcomes. Several modifiable VTE risk factors were identified.

Economic Value of Improved Accuracy for Self-Monitoring of Blood Glucose Devices for Type 1 and Type 2 Diabetes in England.

OBJECTIVE: The objective was to model clinical and economic outcomes of self-monitoring blood glucose (SMBG) devices with varying error ranges and strip prices for type 1 and insulin-treated type 2 diabetes patients in England. METHODS: We programmed a simulation model that included separate risk and complication estimates by type of diabetes and evidence from in silico modeling validated by the Food and Drug Administration. Changes in SMBG error were associated with changes in hemoglobin A1c (HbA1c) and separately, changes in hypoglycemia. Markov cohort simulation estimated clinical and economic outcomes. A SMBG device with 8.4% error and strip price of £0.30 (exceeding accuracy requirements by International Organization for Standardization [ISO] 15197:2013/EN ISO 15197:2015) was compared to a device with 15% error (accuracy meeting ISO 15197:2013/EN ISO 15197:2015) and price of £0.20. Outcomes were lifetime costs, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs). RESULTS: With SMBG errors associated with changes in HbA1c only, the ICER was £3064 per QALY in type 1 diabetes and £264 668 per QALY in insulin-treated type 2 diabetes for an SMBG device with 8.4% versus 15% error. With SMBG errors associated with hypoglycemic events only, the device exceeding accuracy requirements was cost-saving and more effective in insulin-treated type 1 and type 2 diabetes. CONCLUSIONS: Investment in devices with higher strip prices but improved accuracy (less error) appears to be an efficient strategy for insulin-treated diabetes patients at high risk of severe hypoglycemia.

Can Economic Model Transparency Improve Provider Interpretation of Cost-effectiveness Analysis? Evaluating Tradeoffs Presented by the Second Panel on Cost-effectiveness in Health and Medicine.

The Second Panel on Cost-Effectiveness in Health and Medicine convened on December 7, 2016 at the National Academy of Medicine to disseminate their recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses (CEAs). Following its summary, panel proceedings included lengthy discussions including the field's struggle to disseminate findings efficiently through peer-reviewed literature to target audiences. With editors of several medical and outcomes research journals in attendance, there was consensus that findings of cost-effectiveness analyses do not effectively reach other researchers or health care providers. The audience members suggested several solutions including providing additional training to clinicians in cost-effectiveness research and requiring that cost-effectiveness models are made publicly available. However, there remains the questions of whether making economic modelers' work open-access through journals is fair under the defense that these models remain one's own intellectual property, or whether journals can properly manage the peer-review process specifically for cost-effectiveness analyses. In this article, we elaborate on these issues and provide some suggested solutions that may increase the dissemination and application of cost-effectiveness literature to reach its intended audiences and ultimately benefit the patient. Ultimately, it is our combined view as economic modelers and clinicians that cost-effectiveness results need to reach the clinician to improve the efficiency of medical practice, but that open-access models do not improve clinician access or interpretation of the economics of medicine.

Finding Resolution for the Responsible Transparency of Economic Models in Health and Medicine.

The Second Panel on Cost-Effectiveness in Health and Medicine recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses has a number of questions unanswered with respect to the implementation of transparent, open source code interface for economic models. The possibility of making economic model source code could be positive and progressive for the field; however, several unintended consequences of this system should be first considered before complete implementation of this model. First, there is the concern regarding intellectual property rights that modelers have to their analyses. Second, the open source code could make analyses more accessible to inexperienced modelers, leading to inaccurate or misinterpreted results. We propose several resolutions to these concerns. The field should establish a licensing system of open source code such that the model originators maintain control of the code use and grant permissions to other investigators who wish to use it. The field should also be more forthcoming towards the teaching of cost-effectiveness analysis in medical and health services education so that providers and other professionals are familiar with economic modeling and able to conduct analyses with open source code. These types of unintended consequences need to be fully considered before the field's preparedness to move forward into an era of model transparency with open source code.

Economic differences in direct and indirect costs between people with epilepsy and without epilepsy.

OBJECTIVE: To provide generalizable estimates of economic burden in epilepsy and nonepilepsy populations and a comprehensive accounting for employment-based lost productivity associated with epilepsy in current US health care systems as compared with other chronic diseases. RESEARCH DESIGN: We use the nationally representative data source (Medical Expenditure Panel Survey) from 1998 to 2009 to create a retrospective cohort of people diagnosed with epilepsy by a health professional and a comparison cohort of people with no epilepsy. MEASURES: Health care utilization and direct costs for all components of treatment, including prescription medications, wages, employment, educational attainment, family income, and lost productivity were outcomes. RESULTS: We observed economic disparities associated with epilepsy in the United States despite high rates of modern treatments (89% on anticonvulsant therapies). Only 42% of the people with epilepsy over age 18 reported employment compared with 70% of people with no epilepsy; among those, people with epilepsy reported missing an average of 12 days of work because of illness or injury as compared with 4 days in the nonepilepsy cohort. Holding other variables constant, people with epilepsy had a loss of productivity of $9504 in 2011 dollars compared with people with no epilepsy. In comparison, diabetes was associated with annual average lost productivity valued at $3358 and depression at $3182. CONCLUSIONS: Lost wage-based productivity associated with epilepsy was nearly equal to combined wage losses associated with diabetes, depression, anxiety, and asthma together. To evaluate societal burden of illness, results illustrate the importance of indirect costs in addition to treated prevalence and direct medical costs.

An evaluation of the impact of patient cost sharing for antihypertensive medications on adherence, medication and health care utilization, and expenditures.

OBJECTIVE: To assess the impact of patient cost-sharing for antihypertensive medications on the proportion of days covered (PDC) by antihypertensive medications, medical utilization, and health care expenditures among commercially insured individuals assigned to different risk categories. METHODS: Participants were identified from the Consolidated Health Cost Guidelines (CHCG) database (January 1, 2006-December 31, 2008) based on a diagnosis (index) claim for hypertension, continuous enrollment ≥12 months pre- and post-index, and no prior claims for antihypertensive medications. Participants were assigned to: low-risk group (no comorbidities), high-risk group (1+ selected comorbidities), or very high-risk group (prior hospitalization for 1+ selected comorbidities). The relationship between patient cost sharing and PDC by antihypertensive medications was assessed using standard linear regression models, controlling for risk group membership, and various demographic and clinical factors. The relationship between PDC and health care service utilization was subsequently examined using negative binomial regression models. RESULTS: Of the 28,688 study patients, 66% were low risk. The multivariate regression model supported a relationship between patient cost sharing per 30-day fill and PDC in the following year. For every US$1.00 increase in cost sharing, PDC decreased by 1.1 days (P < 0.0001). Significant predictors of PDC included high risk, older age, gender, Charlson Comorbidity Index score, geography, and total post-index insurer- and patient-paid costs. An increase in PDC was associated with a decrease in all-cause and hypertension-related inpatient, outpatient, and emergency room visits and medical, pharmacy, and total costs. CONCLUSIONS: The trend has been for managed care organizations and employers to require patients to bear a greater out-of-pocket burden for health care resources consumed. This study illustrates the potential adverse effects of higher patient cost sharing among patients with hypertension stratified by different risk levels. A decrease in PDC was predictive of higher resource utilization and health care costs, which should be of interest to payers and employers alike.

Use of diagnostic tests and procedures for disease-modifying therapy users and non-disease-modifying therapy users with multiple sclerosis.

We examined the use of 23 diagnostic procedures and monitoring tests for users of disease-modifying therapy (DMT) and non-DMT users with multiple sclerosis (MS). The Medstat MarketScan(®) Commercial Claims and Encounters database (2003-2007), which is composed of medical and pharmacy claims for approximately 8 million beneficiaries from 45 US commercial health plans, was used to identify DMT users with an index claim for an MS drug and a 6-month baseline period without MS drugs. Patients were followed for 12 months. Logistic regression models were used to estimate differences in rates and proportion of patients receiving procedures and tests between cohorts. Baseline rates for DMT users (n = 12,455) included MRIs (76.8%), spinal taps (15.7%), neuropsychological testing (4.7%), chemistry panels (61.4%), complete blood cell counts (76.7%) and liver function tests (60.5%). Relative to non-DMT users (n = 25,534), DMT users were more likely to receive an MRI, neuropsychological testing, chemistry panels, complete blood cell counts and liver function tests.

Economic grand rounds: psychological distress and depression associated with job loss and gain: the social costs of job instability.

The authors used Medical Expenditure Panel Survey data for 81,097 respondents in 2004-2007, a period of economic expansion, to examine psychological distress among depressed and nondepressed persons in four categories: employed (73%), unemployed (23%), recent job loss (4%), and recent job gain (<1%). Depressed persons who experienced job loss or unemployment were significantly more distressed than depressed persons who were employed. Among depressed persons, on all measures of distress except one (worthlessness), unadjusted distress levels for those who gained a job were higher than for those who had lost a job. Measurements of the social costs of job instability need to account for costs related to unemployment and underemployment.