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This viewpoint discusses cost-effectiveness estimates for EtranaDez, a gene therapy for hemophilia B, using the Institute for Clinical and Economic Reviews (ICER) framework for single and short-term therapies (SSTs). EtranaDez offers long-term benefits from a single administration, in contrast to the high costs and frequent dosing required by current factor IX prophylaxis. However, the projected gains in health from EtranaDez are small relative to the cost implications of the therapy, and consequently, how the cost offsets associated with EtranaDez are counted has a substantial impact on assessing its cost-effectiveness. Strategies for assessing cost offsets used in the ICER SST framework include a 50/50 cost-sharing model between the health care system and the manufacturer and a cap of $150,000 annually on health care cost offsets. Results from the standard full cost-offset analysis as reported by ICER depicted EtranaDez as a dominant therapy with substantial cost savings compared with factor IX prophylaxis. However, while considering the ICER SST framework, particularly the $150,000 annual cap scenario, the cost-effectiveness was significantly reduced. The incremental cost-effectiveness ratio varied notably between these scenarios, challenging the conventional perception of value of gene therapy in health care. These cost-sharing scenarios highlight the potential of the ICER SST framework to help curtail inefficient health care spending. In cases in which the cost of existing treatment is exceedingly high, the application of such frameworks would improve efficiency in resource allocation, fostering a balance between incentives for innovation and economic sustainability in managed care systems.

DISCLOSURES: Dr Tice and Mr Sarker received ICER grants during the conduct of the study. Dr Moradi, Ms Herce-Hagiwara, Dr Faghim, Dr Agboola, Dr Rind, and Dr Pearson reports grants from Arnold Ventures, grants from Blue Cross Blue Shield of MA, grants from California Healthcare Foundation, grants from The Commonwealth Fund, grants from The Peterson Center on Healthcare, during the conduct of the study; other from Aetna, other from Americas Health Insurance Plans, other from Anthem, other from AbbVie, other from Alnylam, other from AstraZeneca, other from Biogen, other from Blue Shield of CA, other from Cambia Health Services, other from CVS, other from Editas, other from Express Scripts, other from Genentech/Roche, other from GlaxoSmithKline, other from Harvard Pilgrim, other from Health Care Service Corporation, other from Health Partners, other from Johnson & Johnson (Janssen), other from Kaiser Permanente, other from LEO Pharma, other from Mallinckrodt, other from Merck, other from Novartis, other from National Pharmaceutical Council, other from Premera, other from Prime Therapeutics, other from Regeneron, other from Sanofi, other from Spark Therapeutics, other from United Healthcare, other from HealthFirst, other from Pfizer, other from Boehringer-Ingelheim, other from uniQure, other from Evolve Pharmacy Solutions, other from Humana, other from Sun Life, outside the submitted work.

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. ICERs annual policy summit is supported by dues from Aetna, Americas 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, Johnson & Johnson (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.

Objective: To gather real‐world evidence on antiseizure medications (ASMs) treatment patterns and related outcomes in patients with drug‐resistant focal epilepsy. Methods: Medical insurance claims from the start of 2014 till the end of 2019 were used. Patient selection criteria included International Classification of Diseases (ICD) codes followed by documented ASM use. Baseline patient demographics along with ASM and rescue medication use patterns and related patient outcome were documented for first (index) ASM regimen. Patients who failed the first regimen and then failed the second regimen were considered drug resistant. Multivariate analyses were performed to identify risks and other characteristics for positive or negative treatment outcomes. Results: Study cohort consisted of 46 474 patients with a mean age of 47.23 (SD: 16.94). Levetiracetam was the most first‐encountered ASM (37.94%). At baseline, 87.14% were treated with ASMs prior to having study‐confirmed diagnoses. Mental comorbidities were present in 37.86% of patients. After first‐year ASM treatment, 34.61% of patients persisted on their index regimen and 5.91% were seizure‐free. Patients failing first ASM regimen numbered 12 868 (27.69%). Drug‐resistant patients who failed first and then second ASM regimens numbered 6335 (49.23%). Percentages of patients who had successful second treatment and seizure‐free were 21.32 and 3.65, respectively. Initiating patients on lamotrigine or carbamazepine (relative to levetiracetam), baseline use of index ASM, rescue medications, and older age or male gender all lowered the risk for treatment failure. Having higher comorbidity, comorbid mental illness, headache, or neoplasty increased such a risk. Baseline use of index ASM, depressive episode, or anxiety disorder all entailed higher risk of failing second ASM treatment. Significance: Overall, reported findings indicated that patient history at baseline and the early selection of an ASM all influenced treatment outcomes. Findings pointed to the complex nature of ASM treatment in drug‐resistant focal epilepsy patients calling for additional research to identify the optimal treatment to achieve beneficial patient outcomes. [ABSTRACT FROM AUTHOR]

Objective: Men who work in skilled and unskilled trades and labor occupations (i.e., blue‐collar occupations), have high rates of obesity and associated comorbidities but are underrepresented in weight loss programs. A first step in engaging this group is to better understand their preferences for weight loss programs. Methods: Respondents were men working in trade and labor occupations, with overweight/obesity, and an interest in losing weight. A discrete choice experiment was developed, and the data were analyzed using mixed logit model. Respondent characteristics were tested as effect modifiers. Results: Respondents (N = 221, age (M ± SD) 45.0 ± 12.6, BMI 33.3 ± 6.3, 77% non‐Hispanic white) working in a variety of occupations (construction 31%, manufacturing 30%, transportation 25%, maintenance/repair 14%) participated in this study. Results indicate preferences for programs that encourage making smaller dietary changes, are delivered online, and do not incorporate competition. Results were consistent across sensitivity analyses and most respondent groups. Conclusions: The results suggest specific ways to make weight loss programs more appealing to men in trade and labor occupations. Using experimental methods to quantify preferences using larger, more representative samples would further assist in tailoring behavioral weight loss programs for under‐reached populations. [ABSTRACT FROM AUTHOR]

There are significant differences in dentists' hours worked by sex and by age. Women's entry into the dental work force is significant and has helped maintain the supply of dentists.