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Background and objectives: Despite existing therapies, people with lupus nephritis progress to kidney failure and have reduced life expectancy. Belimumab and voclosporin are two new disease-modifying therapies recently approved for the treatment of lupus nephritis.\ud \ud \ud \ud Design, setting, participants, & measurements: A de novo economic model was developed to estimate the cost-effectiveness of these therapies, including the following health states: “complete response,” “partial response,” and “active disease” defined by eGFR and proteinuria changes, kidney failure, and death. Short-term data and mean cohort characteristics were sourced from pivotal clinical trials of belimumab (the Belimumab International Study in Lupus Nephritis) and voclosporin (the Aurinia Urinary Protection Reduction Active–Lupus with Voclosporin trial and Aurinia Renal Response in Active Lupus With Voclosporin). Risk of mortality and kidney failure were on the basis of survival modeling using published Kaplan–Meier data. Each drug was compared with the standard of care as represented by the comparator arm in its respective pivotal trial(s) using US health care sector perspective, with a societal perspective also explored.\ud \ud \ud \ud Results: In the health care perspective probabilistic analysis, the incremental cost-effectiveness ratio for belimumab compared with its control arm was estimated to be approximately $95,000 per quality-adjusted life year. The corresponding incremental ratio for voclosporin compared with its control arm was approximately $150,000 per quality-adjusted life year. Compared with their respective standard care arms, the probabilities of belimumab and voclosporin being cost effective at a threshold of $150,000 per quality-adjusted life year were 69% and 49%, respectively. Cost-effectiveness was dependent on assumptions made regarding survival in response states, costs and utilities in active disease, and the utilities in response states. In the analysis from a societal perspective, the incremental ratio for belimumab was estimated to be approximately $66,000 per quality-adjusted life year, and the incremental ratio for voclosporin was estimated to be approximately $133,000 per quality-adjusted life year.\ud \ud \ud \ud Conclusions: Compared with their respective standard care arms, belimumab but not voclosporin met willingness-to-pay thresholds of $100,000 per quality-adjusted life year. Despite potential clinical superiority in the informing trials, there remains high uncertainty around the cost-effectiveness of voclosporin.

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, California Health Care Foundation, The Donaghue Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from AbbVie, Aetna, America's Health Insurance Plans, Anthem, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Evolve Pharmacy, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Humana, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, uniQure, and United Healthcare. Agboola, McKenna, and Pearson are employed by ICER. Lin and Kazi received funding from ICER for work on this report.

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, California Health Care Foundation, The Donaghue Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from AbbVie, Aetna, America's Health Insurance Plans, Anthem, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Evolve Pharmacy, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Humana, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, uniQure, and United Healthcare. Agboola, Rind, Herron-Smith, and Pearson are employed by ICER. Walton and Quach, through the University of Illinois at Chicago, received funding from ICER for development of the economic model described in this report.

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, Commonwealth Fund, California Health Care Foundation, National Institute for Health Care Management (NIHCM), New England St...

DISCLOSURES: Funding for this summary was contributed by the Laura and John Arnold Foundation, National Institute for Health Care Management, California Health Care Foundation, Blue Cross Blue Shie...

DISCLOSURES: Funding for this summary was contributed by the Laura and John Arnold Foundation, Blue Shield of California, and California Health Care Foundation to the Institute for Clinical and Eco...

Rheumatoid arthritis is associated with a societal burden greater than $39 billion annually. Novel treatments, known as targeted immune modulators (TIMs), are expensive but effective, producing improvements in response rates compared with conventional disease-modifying antirheumatic drugs (cDMARDs). Sarilumab, a TIM approved in 2017, shows superior improvements compared with cDMARDs and produced significantly greater likelihood of achieving response and improvement in the Health Assessment Questionnaire Disability Index than adalimumab monotherapy. Although sarilumab monotherapy has shown improvements over cDMARDs and the TIM market leader adalimumab, treatment with sarilumab is costly, with an annual wholesale acquisition cost of $39,000.To estimate the lifetime cost-effectiveness of starting treatment with sarilumab monotherapy for adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to cDMARDs.A sequential treatment cohort model followed a hypothetical cohort from initiation of sarilumab monotherapy until death. The model allowed patients to switch therapies up to 3 times due to effectiveness or adverse events. The first switch was to a TIM within the same treatment category; the second switch was to a TIM within a different treatment category; and the third switch was to a cDMARD. Sarilumab monotherapy was compared with a cDMARD (methotrexate) and the TIM market leader (adalimumab monotherapy). Key risk and benefit evidence came from clinical studies and network meta-analyses of data on radiographic progression and response. We used a lifetime time horizon and the U.S. health sector payer perspective assuming therapy net pricing. We also incorporated loss of productivity to reflect a restricted societal perspective.Over a lifetime time horizon, a treatment pathway starting with sarilumab resulted in 17.16 life-years and 13.66 quality-adjusted life-years (QALYs). Treatment pathways starting with the cDMARD resulted in 16.54 life-years and 11.77 QALYs; treatment pathways starting with adalimumab resulted in 17.05 life-years and 13.35 QALYs. Total costs for sarilumab ($492,000 for payer perspective, $634,000 for societal perspective) were less than total costs for adalimumab ($536,000 for payer perspective, $689,000 for societal perspective) but higher than total costs for the cDMARD ($63,000 for payer perspective, $272,000 for societal perspective). When compared with cDMARD therapy, sarilumab resulted in a cost-effectiveness estimate of $227,000 per QALY gained from the payer perspective and $191,000 per QALYs gained from the societal perspective. When compared with adalimumab, sarilumab was dominant from both perspectives.Sarilumab resulted in better health outcomes than conventional therapy alone. However, its additional cost with assumed class-level net prices led to cost-effectiveness estimates above commonly cited thresholds. When compared with the market leader, sarilumab achieved favorable value. This evaluation informs stakeholders of the value of sarilumab and its alternatives to promote high value practices in health care.Funding for this research was contributed by the Institute for Clinical and Economic Review (ICER). Ollendorf, Chapman, Kumar, Synnott, and Agboola are employees of ICER, an independent organization that evaluates the evidence on the value of health care interventions, which is funded by grants from the Laura and John Arnold Foundation, Blue Shield of California Foundation, and the California HealthCare Foundation. The organization's annual policy summit is supported by dues from Aetna, AHIP, Anthem, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Health Care, Omeda Rx, United Healthcare, Kaiser Permanente, Premera Blue Cross, AstraZeneca, Genentech, GlaxoSmithKline, JohnsonJohnson, Merck, National Pharmaceutical Council, Takeda, Pfizer, Novartis, Lilly, and Humana. This work is an extension of an analysis presented at the New England Comparative Effectiveness Public Advisory Council on March 24, 2017, where the authors received public feedback on the analysis, results, and effect of a value assessment for targeted immune modulators. At the time of presentation, sarilumab was still an investigational product; therefore, a price was not known, so cost-effectiveness estimates were not generated. Since the presentation of that material, additional evidence for sarilumab has become available. The additional evidence has been incorporated into this analysis to present cost-effectiveness estimates for sarilumab.

Funding for this summary was contributed by Arnold Ventures, Commonwealth Fund, California Health Care Foundation, National Institute for Health Care Management (NIHCM), New England States Consortium Systems Organization, Blue Cross Blue Shield of Massachusetts, Harvard Pilgrim Health Care, Kaiser Foundation Health Plan, and Partners HealthCare to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, Health Partners, JohnsonJohnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Agboola, Fluetsch, Rind, and Pearson are employed by ICER. Lin reports support from ICER during work on this economic model and grants from Mount Zion Health Fund, National Institutes of Health (National Cancer Institute and National Heart, Lung, and Blood Institute), and the Tobacco-Related Diseases Research Program, unrelated to this work. Walton reports support from ICER for work on this economic model and unrelated consulting fees from Baxter.

In 2008, tbo-filgrastim was approved as a biosimilar in Europe and then approved in the United States by the FDA in 2012 as a biologic product with 1 similar indication to filgrastim. Because tbo-filgrastim was less expensive than filgrastim, and clinical information and expert opinion supported similarity, the PharmacyTherapeutics Committee of a large health care system approved tbo-filgrastim as the preferred granulocyte-colony stimulating factor (G-CSF) product in March 2014.To (a) assess the use of filgrastim and tbo-filgrastim products by comparing baseline characteristics, setting of care, indication for use, and payer type and (b) understand potential barriers of conversion to tbo-filgrastim.A retrospective evaluation of filgrastim and tbo-filgrastim use was conducted on all patients (N = 204) who received the drugs between July 2015 and December 2015 at the 2 largest hospitals in the health system. Baseline characteristics, indication requiring use of filgrastim or tbo-filgrastim, setting of care, and payer information were collected from electronic medical records, and descriptive analyses were conducted.Overall, G-CSFs were administered to 204 patients for 261 episodes of care (filgrastim and tbo-filgrastim were used in 65 and 196 episodes of care, respectively). Baseline characteristics were similar between the 59 patients who received filgrastim and the 174 patients who received tbo-filgrastim. G-CSF was primarily used in the inpatient setting (163 episodes of care, 63%) with 90% of patients using tbo-filgrastim. In the outpatient setting (98 episodes of care, 38%), filgrastim and tbo-filgrastim were each used by 50% of patients. Tbo-filgrastim was the preferred G-CSF by clinical providers for all indications, except for stem cell mobilization, where filgrastim use was higher (55% vs. 45% of 71 episodes of care). In the outpatient setting, analysis by payers showed that the majority of patients on commercial plans were using filgrastim (58%), while half of Medicare patients were using filgrastim (50%, n = 12). Twelve patients were self-paid, and all were using tbo-filgrastim. Subgroup analysis by hospital showed differences in utilization patterns.Although tbo-filgrastim was the preferred G-CSF in our formulary, 29% of patients continued to receive filgrastim. Conversion to tbo-filgrastim has been largely successful, but extra steps may be needed to achieve full conversion to biosimilars.No outside funding supported this study. Agboola was employed by Partners Healthcare at the time of the study. The authors have nothing to disclose. Study concept and design were contributed equally by Agboola and Reddy. Agboola collected the data, and data interpretation was performed by both authors. The manuscript was written primarily by Agboola, with assistance from Reddy. Both authors revised the manuscript.

Funding for this summary was contributed by the Laura and John Arnold Foundation, Blue Shield of California, and California Health Care Foundation to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, AHIP Anthem, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Health Care, Cambia Health Solutions, United Healthcare, Kaiser Permanente, Premera Blue Cross, AstraZeneca, Genentech, GlaxoSmithKline, JohnsonJohnson, Merck, National Pharmaceutical Council, Prime Therapeutics, Sanofi, Spark Therapeutics, Health Care Service Corporation, Editas, Alnylam, Regeneron, Mallinkrodt, Biogen, HealthPartners, and Novartis. Agboola, Dreitlein, and Pearson are ICER employees. Lin reports personal fees from ICER, during the conduct of this study, and grants from the National Institutes of Health and the California Department of Insurance, outside the submitted work. Carlson and Lubinga report grants from ICER, during the conduct of this study.

Objective Opioid abuse is a significant public health problem in the United States. We evaluate the clinical effectiveness and economic impact of abuse-deterrent formulations (ADF) of opioids relative to non-ADF opioids in preventing abuse. Methods We developed a cost-effectiveness model simulating 2 cohorts of 100 000 noncancer, chronic-pain patients newly prescribed either ADF or non-ADF extended-release (ER) opioids and followed them over 5 years, tracking new events of opioid abuse and opioid-related overdose deaths in addition to tracking 5-year cumulative costs of therapeutic use and abuse of ADF and non-ADF opioids. Patients in each cohort entered the model for therapeutic opioid use from where they could continue in that pathway, discontinue opioid use, or abuse opioids or die of opioid overdose-related or unrelated causes. In addition, one-way sensitivity and scenario analysis were conducted. Results Over a 5-year time period, using ADF opioids prevented an additional 2300 new cases of opioid abuse at an additional cost of approximately $535 million to the healthcare sector. Threshold analyses showed that a 40% decrease in ADF opioid costs was required to attain cost neutrality between the 2 cohorts, whereas a 100% effectiveness in abuse reduction still did not result in cost neutrality. A 43% decrease in diversion with ADFs relative to non-ADFs was required to attain cost neutrality. Including a societal perspective produced results directionally similar to the base-case analysis findings. Conclusion ADF opioids have the potential to prevent new cases of opioid abuse, but at substantially higher costs to the health system.

IntroductionExercises that simulate emergency scenarios are accepted widely as an essential component of a robust Emergency Preparedness program. Unfortunately, the variability in the quality of the exercises conducted, and the lack of standardized processes to measure performance, has limited the value of exercises in measuring preparedness.MethodsIn order to help health organizations improve the quality and standardization of the performance data they collect during simulated emergencies, a model online exercise evaluation toolkit was developed using performance measures tested in over 60 Emergency Preparedness exercises. The exercise evaluation toolkit contains three major components: (1) a database of measures that can be used to assess performance during an emergency response exercise; (2) a standardized data collection tool (form); and (3) a program that populates the data collection tool with the measures that have been selected by the user from the database. The evaluation toolkit was pilot tested from January through September 2014 in collaboration with 14 partnering organizations representing 10 public health agencies and four health care agencies from eight states across the US. Exercise planners from the partnering organizations were asked to use the toolkit for their exercise evaluation process and were interviewed to provide feedback on the use of the toolkit, the generated evaluation tool, and the usefulness of the data being gathered for the development of the exercise after-action report.ResultsNinety-three percent (93%) of exercise planners reported that they found the online database of performance measures appropriate for the creation of exercise evaluation forms, and they stated that they would use it again for future exercises. Seventy-two percent (72%) liked the exercise evaluation form that was generated from the toolkit, and 93% reported that the data collected by the use of the evaluation form were useful in gauging their organization’s performance during the exercise. Seventy-nine percent (79%) of exercise planners preferred the evaluation form generated by the toolkit to other forms of evaluations.ConclusionResults of this project show that users found the newly developed toolkit to be user friendly and more relevant to measurement of specific public health and health care capabilities than other tools currently available. The developed toolkit may contribute to the further advancement of developing a valid approach to exercise performance measurement.AgboolaF, BernardD, SavoiaE, BiddingerPD. Development of an online toolkit for measuring performance in health emergency response exercises. Prehosp Disaster Med. 2015;30(5):503–508.

The aim of this study was to investigate whether prior participation in preparedness exercises was predictive of better performance on objective measures of response. We conducted a statewide tabletop exercise that focused on a hazardous materials (HAZMAT) scenario and assessed performance using our developed evaluation tool. The evaluative score was analyzed in relation to the number of HAZMAT exercises in the past 3 years, participation in prior CHEMPACK-specific exercise, hospital size, teaching status of the hospital, preparedness training experience, and participants years of experience in preparedness activities. Hospitals that had participated in more exercises in the past 3 years performed significantly better than hospitals that had participated in fewer exercises. No significant differences were found between the performance of hospitals in relation to size, teaching status, preparedness training experience, and participants' years of experience in preparedness activities. Our results suggest that more frequent participation in exercises may result in improved overall response.

Log Change of PTH (Month 3-Month 0) by Vitamin D Dose/day. Figure S2. Change in PTH (Month 3- Month 0) by Vitamin D Dose/day. (PDF 201 kb)

Log Change of PTH (Month 3-Month 0) by Vitamin D Dose/day. Figure S2. Change in PTH (Month 3- Month 0) by Vitamin D Dose/day. (PDF 201 kb)

Large-scale public health emergencies require a sophisticated, coordinated response involving multiple entities to protect health and minimize suffering. However, the rarity of such emergencies presents a barrier to gathering observational data about the effectiveness of the public health response before such events occur. For this reason, public health practitioners increasingly have relied on simulated emergencies, known as “exercises” as proxies to test their emergency capabilities. However, the formal evaluation of performance in these exercises, historically has been inconsistent, and there is little research to describe how data acquired from simulated emergencies actually support conclusions about the quality of the public health emergency response system. Over the past six years, we have designed and evaluated more than seventy public health emergency exercises, collaborating with public health agencies, hospitals and others to test a wide variety of systems and their capabilities. Using the data and experience that we gathered, we have developed a conceptual framework that describes the essential elements necessary to consider when applying performance measurement science to public health emergency exercises. We suggest that this framework may assist practitioners and researchers who wish to better measure performance in exercises and to improve public health emergency preparedness.

Many public health and healthcare organizations use formal knowledge management practices to identify and disseminate the experiences gained over time. The “lessons-learned” approach is one such example of knowledge management practice applied to the wider concept of organizational learning. In the field of emergency preparedness, the lessons-learned approach stands on the assumption that learning from experience improves practice and minimizes avoidable deaths and negative economic and social consequences of disasters. In this project, we performed a structured review of AARs to analyze how lessons learned from the response to real-incidents may be used to maximize knowledge management and quality improvement practices such as the design of public health emergency preparedness (PHEP) exercises. We chose as a source of data the “Lessons Learned Information Sharing (LLIS.gov)” system, a joined program of the U.S. Department of Homeland Security DHS and FEMA that serves as the national, online repository of lessons learned, best practices, and innovative ideas. We identified recurring challenges reported by various states and local public health agencies in the response to different types of incidents. We also strived to identify the limitations of systematic learning that can be achieved due to existing weaknesses in the way AARs are developed.

Background: During public health emergencies, public officials are busy in developing communication strategies to protect the population from existing or potential threats. However, a population’s social and individual determinants (i.e. education, income, race/ethnicity) may lead to inequalities in individual or group-specific exposure to public health communication messages, and in the capacity to access, process, and act upon the information received by specific sub-groups- a concept defined as communication inequalities. The aims of this literature review are to: 1) characterize the scientific literature that examined issues related to communication to the public during the H1N1 pandemic, and 2) summarize the knowledge gained in our understanding of social determinants and their association with communication inequalities in the preparedness and response to an influenza pandemic. Methods: Articles were searched in eight major communication, social sciences, and health and medical databases of scientific literature and reviewed by two independent reviewers by following the PRISMA guidelines. The selected articles were classified and analyzed in accordance with the Structural Influence Model of Public Health Emergency Preparedness Communications. Results: A total of 118 empirical studies were included for final review. Among them, 78% were population-based studies and 22% were articles that employed information environment analyses techniques. Consistent results were reported on the association between social determinants of communication inequalities and emergency preparedness outcomes. Trust in public officials and source of information, worry and levels of knowledge about the disease, and routine media exposure as well as information-seeking behaviors, were related to greater likelihood of adoption of recommended infection prevention practices. When addressed in communication interventions, these factors can increase the effectiveness of the response to pandemics. Conclusions: Consistently across studies, a number of potential predictors of behavioral compliance to preventive recommendations during a pandemic were identified. Our findings show the need to include such evidence found in the development of future communication campaigns to ensure the highest rates of compliance with recommended protection measures and reduce communication inequalities during future emergencies.

Response of parathyroid hormone (PTH) to vitamin D supplementation is determined by the baseline PTH level and change in vitamin D status. Conflicting reports in Blacks exist on the PTH response to vitamin D to supplementation. During 3 winters from 2007 to 2010, 328 healthy Blacks (median age, 51 years) living in Boston, MA were randomized into a 4-arm, double-blind trial for 3 months of placebo, 1000, 2000, or 4000 IU of vitamin D3. PTH was measured in 254 participants at baseline and at the end of vitamin D supplementation period. The differences in PTH between baseline and 3 months were 3.93 pg/mL for those receiving placebo, -3.37 pg/mL for those receiving 1000 IU/d, -6.76 pg/mL for those receiving 2000 IU/d, and -8.99 pg/mL for those receiving 4000 IU/d (-2.98 pg/mL for each additional 1000 IU/d of vitamin D3; p