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Objective: Changes to the Medicaid Drug Rebate Program (MDRP) determination of Medicaid Best Price (MBP) enables Value-based purchasing arrangements (VBPAs) to address financial uncertainty. This study estimates the likely effectiveness of MDRP-enabled VBPAs for chronically dosed medicines., Methods: Monte Carlo simulations examined: Multiple Best Prices and Bundled Sales MBP approaches authorized under MDRP and a third National Pooling approach using payment misalignment; needed payer size for practical participation; and the resulting potential number of covered lives under a VBPA as evaluation metrics., Results: Both Multiple Best Prices and National Pooling enable VBPAs for 95% of scenarios (including all 5i chronic products with ≥1,000 treated patients per year), with 75% of those with payment misalignment ≤9%. National pooling for retail drugs has less participation and worse misalignment (5i: 95% contracted, 75% ≤9% misalignment; retail: 71%, 66%). Bundled Sales performed worst (5i: 40%, 75% ≤9%; retail: 31%, 88%) due to rebate volatility risk of breaking best price and Average Manufacturer Price impact. Medicaid sees worse misalignment for the 60% drug performance scenarios because of comparison to the statutory rebate (23.1%)., Conclusion: The Multiple Best Prices approach has the lowest misalignment and could be applied to most chronic therapies, even rare ones.

Background: Cell and gene therapies promise durable benefits but face financial challenges from the uncertainty in their performance. Value-based purchasing arrangements (VBPAs) can address uncertainty but have been inhibited in the US by the Medicaid Drug Rebate Program (MDRP) approach to determining Medicaid Best Price (MBP) rebates. The likely effectiveness of MDRP reform proposals enabling VBPAs is examined in this study for durable cell and gene therapies., Methods: Monte Carlo simulations examined three potential reforms (Multiple Best Prices, Bundled Sales, and National Pooling) to determine the impact on payment misalignment, the required payer size to participate, and the percentage of total lives covered by a VBPA., Results: Simulation results suggest that 11% to 54% of commercial US lives would be feasibly covered by a VBPA depending on reform type and condition size. MPB reform achieved the highest commercial contracted percentage and lowest misalignment for commercial payers compared to National Pooling and Bundled Sales. State Medicaid plan results suggest lower extreme misalignment across all successfully contracted instances than commercial payers., Conclusions: The Multiple Best Prices will likely enable VBPAs for many durable cell and gene therapies and larger payers. Further reforms may be needed to extend VBPAs to ultra-orphan conditions.

BACKGROUND: US health plans are adopting benefit designs that shift greater financial burden to patients through higher deductibles, additional copay tiers, and coinsurance. Prior systematic reviews found that higher cost was associated with reductions in both appropriate and inappropriate medications. However, these reviews were conducted prior to contemporary benefit design and medication utilization. OBJECTIVE: To assess the relationship and factors associated with cost-sharing and (1) medication adherence, (2) clinical outcomes, (3) health care resource utilization (HRU), and (4) costs. METHODS: A systematic review of literature published between January 2010 and August 2020 was conducted to identify the relationship between cost-sharing and medication adherence, clinical outcomes, HRU, and health care costs. Data were extracted using a standardized template and were synthesized by key questions of interest. RESULTS: From 1,995 records screened, 79 articles were included. Most studies, 71 of 79 (90%), reported the relationship between cost-sharing and treatment adherence, persistence and/or discontinuation; 16 (20%) reported data on cost-sharing and HRU or medication initiation, 11 (14%) on costsharing and health care costs, and 6 (8%) on cost-sharing and clinical outcomes. The majority of publications found that, regardless of disease area, increased cost-sharing was associated with worse adherence, persistence, or discontinuation. The aggregate data suggested the greater the magnitude of cost-sharing, the worse the adherence. Among studies examining clinical outcomes, cost-sharing was associated with worse outcomes in 1 study and the remaining 3 found no significant differences. Regarding HRU, higher-cost-sharing trended toward decreased outpatient and increased inpatient utilization. The available evidence suggested higher cost-sharing has an overall neutral to negative impact on total costs. Studies evaluating elimination of copays found either decreased or no impact in total costs. CONCLUSIONS: The published literature shows consistent impacts of higher cost sharing on initiation and continuation of medications, and the greater the cost-sharing, the worse the medication adherence. The evidence is limited regarding the impact of cost-sharing on clinical outcomes, HRU, and costs. Limited evidence suggests increased cost-sharing is associated with more inpatient care and less outpatient care; however, a neutral to no difference was suggested for other outcomes. Although increased costsharing is intended to decrease total costs, studies evaluating reducing or eliminating cost-sharing found that total costs did not rise. Today's growing cost-containment environment should carefully consider the broader impact cost-sharing has on treatment adherence, clinical outcomes, resource use, and total costs. It may be that cost-sharing is a blunt, rather than precise, tool to curb health care costs, affecting both necessary and unnecessary health care use. DISCLOSURES: This study and the development of this article were funded by the National Pharmaceutical Council. Mr Sils is an employee of the National Pharmaceutical Council. Dr Graff is a former employee of the National Pharmaceutical Council. Drs Fusco and Kistler and Ms Ruiz are employees of Xcenda. Xcenda received funding to conduct the literature review.

BACKGROUND: Expenditures on specialty medications for autoimmune conditions (SpRx-AIC) have increased considerably in recent years, raising affordability concerns for employers and other plan sponsors and resulting in greater patient cost-sharing. Among those commercially insured, prior studies have shown differential patterns of health care utilization in association with wage, though no data are available for SpRx-AIC. Notably, out-of-pocket costs associated with SpRx-AIC have been shown to impact medication adherence, particularly for low-income households. OBJECTIVE: To assess the association of wage status on SpRx-AIC and health care services use and cost among employees with employer-sponsored health insurance. METHODS: Employee health care claims and wage data were obtained from the IBM Watson MarketScan database for calendar year 2018. Midyear employee wage data were used as a basis for allocating employees into annual income quartiles: $47,000 or less, $47,001-$71,000, $71,001-$106,000, and $106,001 or more. The lowest quartile was further divided into 2 groups ($35,000 or less and $35,001-$47,000) to better evaluate subgroup differences at lower wage levels. Outcomes included monthly days supply of SpRx-AIC, medication discontinuation rates (medication cessation for ≥ 90 days), proportion of days covered (PDC), medical services utilization rates per 1,000, and allowed payment amounts. Generalized linear regressions were used to assess differences while adjusting for patient characteristics, including age, gender, plan type, region, median household income, deductible amount, comorbidity index, and psychiatric diagnostic scores. RESULTS: From a sample of more than 2 million enrollees, 148,761 (7.2%) were identified as having an autoimmune disorder of interest. Of those, 17,096 (11.5%) had filled at least one SpRx-AIC prescription. Following adjustment, SpRx-AIC use was significantly less among the lowest wage group compared with the highest wage group (10.1% vs 11.7%; P < 0.0001). Days supply was significantly lower in the lowest wage group (244.4 vs 258.0; P < 0.001), as was PDC (0.74 vs 0.76; P < 0.001). In the lowest wage group, medical services utilization was significantly higher for inpatient admissions (0.08 vs 0.05; P = 0.002) and emergency department visits (0.52 vs 0.16; P < 0.0001). There were no significant differences among wage groups in SpRx-AIC discontinuation, outpatient services use, or health care costs. CONCLUSIONS: Low-wage employees with autoimmune conditions are significantly less likely to use an SpRx-AIC and have a lower monthly supply and PDC when SpRx-AIC was used. They are more likely to be admitted to the hospital and have more emergency department visits. These findings raise concerns about employer benefit design inequities for SpRx-AIC access and the resulting potential adverse impact on health care costs and employee functional status. DISCLOSURES : National Pharmaceutical Council, Genentech, and TrialCard provided funding support for this study, with funding administered by the National Alliance of Healthcare Purchaser Coalitions. Genentech and TrialCard provided comments regarding the final manuscript draft; National Pharmaceutical Council employees were actively engaged in study design, analysis and interpretation of results, and manuscript preparation. Dr Sherman is a consultant to National Alliance of Healthcare Purchaser Coalitions. Mr Sils and Ms Westrich were employees of the National Pharmaceutical Council at time of study. Ms Kamen is an employee of IBM Watson Health.

Introduction: The study estimated the extent to which drug innovations over the past 30 years may have improved outcomes for six diseases., Areas Covered: We analyzed six diseases (ischemic heart disease, lung cancer, breast cancer, human immunodeficiency virus [HIV] infection, type 2 diabetes mellitus, and rheumatoid arthritis [RA]) with significant mortality or morbidity for which there have been major drug innovations over the past 30 years. We used U.S. data from the Global Burden of Disease (GBD) database and a patient registry to perform counterfactual time-series analyses predicting the improved health outcomes that may have been associated with major drug innovations. For 5 conditions using data from the GBD study, years of life lost per individual with the condition could have been higher by 17.1% (breast cancer) to 660.6% (HIV infection) in 2017 had the major drug innovations not been introduced. For RA, using patient registry data, patients' functional status could have been 11.5% worse had biological therapies not been introduced., Expert Opinion: Policies targeting drug prices should be broadened to consider the price and value of all health-care services. The societal importance of the pharmaceutical industry's ability to respond rapidly to emerging diseases should be recognized.

RASSF1A may be the most frequently inactivated tumor suppressor identified in human cancer so far. It is a proapoptotic Ras effector and plays an important role in the apoptotic DNA damage response (DDR). We now show that in addition to DDR regulation, RASSF1A also plays a key role in the DNA repair process itself. We show that RASSF1A forms a DNA damage-regulated complex with the key DNA repair protein xeroderma pigmentosum A (XPA). XPA requires RASSF1A to exert full repair activity, and RASSF1A-deficient cells exhibit an impaired ability to repair DNA. Moreover, a cancer-associated RASSF1A single-nucleotide polymorphism (SNP) variant exhibits differential XPA binding and inhibits DNA repair. The interaction of XPA with other components of the repair complex, such as replication protein A (RPA), is controlled in part by a dynamic acetylation/deacetylation cycle. We found that RASSF1A and its SNP variant differentially regulate XPA protein acetylation, and the SNP variant hyperstabilizes the XPA-RPA70 complex. Thus, we identify two novel functions for RASSF1A in the control of DNA repair and protein acetylation. As RASSF1A modulates both apoptotic DDR and DNA repair, it may play an important and unanticipated role in coordinating the balance between repair and death after DNA damage., (Copyright © 2015, American Society for Microbiology. All Rights Reserved.)