Bolstering FDA Pathways for Reducing Death and Disease from Tobacco.

This JAMA Forum discusses the US Food and Drug Administration's efforts to implement the 2009 Family Smoking Prevention and Tobacco Control Act through approval of products that demonstrate evidence to help smokers quit and to reduce their risk of relapse.

Reforms Needed to Modernize the US Food and Drug Administration's Oversight of Dietary Supplements, Cosmetics, and Diagnostic Tests.

This JAMA Forum discusses long-awaited reforms that could modernize the US Food and Drug Administration's regulatory processes, promote innovation, and provide US consumers greater assurance that the products they use are safe and reliable.

Understanding Use of Real-World Data and Real-World Evidence to Support Regulatory Decisions on Medical Product Effectiveness.

RWE has potential to provide efficient and relevant information on the effectiveness of medical products, complementing the data generated in clinical trials; however, how RWE can support regulatory decision-making is unclear, potentially limiting its use. The objective of this study was to identify and characterize instances where RWE was included in the evidence package to support the effectiveness of a medical product regulated by U.S. Food and Drug Administration. A retrospective landscape analysis was conducted to identify instances where RWE was submitted to support effectiveness through targeted review of white and gray literature and publicly available FDA reviews of medical products. Trained evaluators examined FDA reviews to determine if and how RWE contributed to regulatory decision-making regarding effectiveness. Evaluators identified 34 instances of RWE submitted between 1954 and 2020, where 26% of instances were for oncology, 18% for hematology, and 12% for neurology. Over 50% of the products were indicated for use in rare disease or pediatric populations. 82% of products where RWE was submitted received an orphan designation. RWE was included in the product label in 59% of instances. Stated reasons indicating why submitted RWE did not significantly contribute to regulatory decision-making included lack of pre-specification of study design and analysis as well as data reliability and relevancy concerns. While there is historical use of RWE to support medical product effectiveness for oncology and rare diseases, potential exists to leverage the strengths of RWE to support other therapeutic areas and capture outcomes that are most relevant to patients.

Hospital-level compliance with the commission on cancer's quality of care measures and the association with patient survival.

BACKGROUND: Quality measurement has become a priority for national healthcare reform, and valid measures are necessary to discriminate hospital performance and support value-based healthcare delivery. The Commission on Cancer (CoC) is the largest cancer-specific accreditor of hospital quality in the United States and has implemented Quality of Care Measures to evaluate cancer care delivery. However, none has been formally tested as a valid metric for assessing hospital performance based on actual patient outcomes. METHODS: Eligibility and compliance with the Quality of Care Measures are reported within the National Cancer Database, which also captures data for robust patient-level risk adjustment. Hospital-level compliance was calculated for the core measures, and the association with patient survival was tested using Cox regression. RESULTS: Seven hundred sixty-eight thousand nine hundred sixty-nine unique cancer cases were included from 1323 facilities. Increasing hospital-level compliance was associated with improved survival for only two measures, including a 35% reduced risk of mortality for the gastric cancer measure G15RLN (HR 0.65, 95% CI 0.58-0.72) and a 19% reduced risk of mortality for the colon cancer measure 12RLN (HR 0.81, 95% CI 0.77-0.85). For the lung cancer measure LNoSurg, increasing compliance was paradoxically associated with an increased risk of mortality (HR 1.14, 95% CI 1.08-1.20). For the remaining measures, hospital-level compliance demonstrated no consistent association with patient survival. CONCLUSION: Hospital-level compliance with the CoC's Quality of Care Measures is not uniformly aligned with patient survival. In their current form, these measures do not reliably discriminate hospital performance and are limited as a tool for value-based healthcare delivery.

Association of ACO Shared Savings Success and Serious Illness Spending.

EXECUTIVE SUMMARY: Accountable care organizations (ACOs) need confidence in their return on investment to implement changes in care delivery that prioritize seriously ill and high-cost Medicare beneficiaries. The objective of this study was to characterize spending on seriously ill beneficiaries in ACOs with Medicare Shared Savings Program (MSSP) contracts and the association of spending with ACO shared savings. The population included Medicare fee-for-service beneficiaries identified with serious illness (N = 2,109,573) using the Medicare Master Beneficiary Summary File for 100% of ACO-attributed beneficiaries linked to MSSP beneficiary files (2014-2016). Lower spending for seriously ill Medicare beneficiaries and risk-bearing contracts in ACOs were associated with achieving ACO shared savings in the MSSP. For most ACOs, the seriously ill contribute approximately half of the spending and constitute 8%-13% of the attributed population. Patient and geographic (county) factors explained $2,329 of the observed difference in per beneficiary per year spending on seriously ill beneficiaries between high- and low-spending ACOs. The remaining $12,536 may indicate variation as a result of potentially modifiable factors. Consequently, if 10% of attributed beneficiaries were seriously ill, an ACO that moved from the worst to the best quartile of per capita serious illness spending could realize a reduction of $1,200 per beneficiary per year for the ACO population overall. Though the prevalence and case mix of seriously ill populations vary across ACOs, this association suggests that care provided for seriously ill patients is an important consideration for ACOs to achieve MSSP shared savings.

Vital Directions For Health And Health Care: Priorities For 2021.

In 2016, in anticipation of the US presidential election and forthcoming new administration, the National Academy of Medicine launched a strategic initiative to marshal expert guidance on pressing health and health care priorities. Published as Vital Directions for Health and Health Care, the products of the initiative provide trusted, nonpartisan, evidence-based analysis of critical issues in health, health care, and biomedical science. The current collection of articles published in Health Affairs builds on the initial Vital Directions series by addressing a set of issues that have a particularly compelling need for attention from the next administration: health costs and financing, early childhood and maternal health, mental health and addiction, better health and health care for older adults, and infectious disease threats. The articles also reflect the current experience with both the coronavirus disease 2019 (COVID-19) pandemic and the health inequities that have been drawn out sharply by COVID-19, as well as the implications going forward for action.

Prospective or retrospective ACO attribution matters for seriously ill patients.

OBJECTIVES: Since 2019, the Medicare Shared Savings Program (MSSP) has allowed accountable care organizations (ACOs) to choose either retrospectively or prospectively attributed ACO populations. To understand how ACOs' choice of attribution method affects incentives for care among seriously ill Medicare beneficiaries, this study compares beneficiary characteristics and Medicare per capita expenditures between prospective and retrospective ACO populations. STUDY DESIGN: This retrospective, cross-sectional analysis describes survival, patient characteristics, and Medicare spending for Medicare fee-for-service beneficiaries identified with serious illness (n = 1,600,629) using 100% Medicare Master Beneficiary Summary and MSSP beneficiary files (2014-2016). METHODS: We used generalized linear models with ACO and year fixed effects to estimate the average within-ACO difference between potential retrospective and prospective ACO populations. RESULTS: Dying in the first 90 days of the performance year was associated with reduced odds of retrospective ACO attribution (odds ratio [OR], 0.24; 95% CI, 0.24-0.25) relative to beneficiaries surviving 270 days or longer. Similarly, hospice use was associated with reduced odds of retrospective assignment (OR, 0.80; 95% CI, 0.79-0.80). Among ACOs that did not achieve shared savings, average per capita Medicare expenditures (after truncation) were $2459 (95% CI, $2192-$2725) higher for prospective vs retrospective ACO populations. The difference was $834 (95% CI, $402-$1266) greater per capita among ACOs that achieved shared savings. CONCLUSIONS: The difference in survival and spending for ACO populations captured by prospective vs retrospective attribution methods means that ACOs may need to employ different care management strategies to improve performance depending on their attribution method.

The Past Decade of Paying for Value : From the Affordable Care Act to COVID-19.

The Affordable Care Act played a major role in transitioning American health care away from fee-for-service payment. We explore the spread of payment reforms since the implementation of the ACA, both nationally and in North Carolina; the corresponding effects on health care costs and quality; and further steps needed to achieve greater transformation.

Correction to: Comparing Associations of State Reopening Strategies with COVID-19 Burden.

In the original version of this paper, an author was misidentified. The corrected author listing appears here, and has been updated in the online version.

Comparing Associations of State Reopening Strategies with COVID-19 Burden.

BACKGROUND: The novel coronavirus disease 2019 (COVID-19) infected over 5 million United States (US) residents resulting in more than 180,000 deaths by August 2020. To mitigate transmission, most states ordered shelter-in-place orders in March and reopening strategies varied. OBJECTIVE: To estimate excess COVID-19 cases and deaths after reopening compared with trends prior to reopening for two groups of states: (1) states with an evidence-based reopening strategy, defined as reopening indoor dining after implementing a statewide mask mandate, and (2) states reopening indoor dining rooms before implementing a statewide mask mandate. DESIGN: Interrupted time series quasi-experimental study design applied to publicly available secondary data. PARTICIPANTS: Fifty United States and the District of Columbia. INTERVENTIONS: Reopening indoor dining rooms before or after implementing a statewide mask mandate. MAIN MEASURES: Outcomes included daily cumulative COVID-19 cases and deaths for each state. KEY RESULTS: On average, the number of excess cases per 100,000 residents in states reopening without masks is ten times the number in states reopening with masks after 8 weeks (643.1 cases; 95% confidence interval (CI) = 406.9, 879.2 and 62.9 cases; CI = 12.6, 113.1, respectively). Excess cases after 6 weeks could have been reduced by 90% from 576,371 to 63,062 and excess deaths reduced by 80% from 22,851 to 4858 had states implemented mask mandates prior to reopening. Over 50,000 excess deaths were prevented within 6 weeks in 13 states that implemented mask mandates prior to reopening. CONCLUSIONS: Additional mitigation measures such as mask use counteract the potential growth in COVID-19 cases and deaths due to reopening businesses. This study contributes to the growing evidence that mask usage is essential for mitigating community transmission of COVID-19. States should delay further reopening until mask mandates are fully implemented, and enforcement by local businesses will be critical for preventing potential future closures.

Frontiers of Upstream Stroke Prevention and Reduced Stroke Inequity Through Predicting, Preventing, and Managing Hypertension and Atrial Fibrillation: A Call to Action From the Value in Healthcare Initiative's Predict & Prevent Learning Collaborative.

Stroke is one of the leading causes of morbidity and mortality in the United States. While age-adjusted stroke mortality was falling, it has leveled off in recent years due in part to advances in medical technology, health care options, and population health interventions. In addition to adverse trends in stroke-related morbidity and mortality across the broader population, there are sociodemographic inequities in stroke risk. These challenges can be addressed by focusing on predicting and preventing modifiable upstream risk factors associated with stroke, but there is a need to develop a practical framework that health care organizations can use to accomplish this task across diverse settings. Accordingly, this article describes the efforts and vision of the multi-stakeholder Predict & Prevent Learning Collaborative of the Value in Healthcare Initiative, a collaboration of the American Heart Association and the Robert J. Margolis, MD, Center for Health Policy at Duke University. This article presents a framework of a potential upstream stroke prevention program with evidence-based implementation strategies for predicting, preventing, and managing stroke risk factors. It is meant to complement existing primary stroke prevention guidelines by identifying frontier strategies that can address gaps in knowledge or implementation. After considering a variety of upstream medical or behavioral risk factors, the group identified 2 risk factors with substantial direct links to stroke for focusing the framework: hypertension and atrial fibrillation. This article also highlights barriers to implementing program components into clinical practice and presents implementation strategies to overcome those barriers. A particular focus was identifying those strategies that could be implemented across many settings, especially lower-resource practices and community-based enterprises representing broad social, economic, and geographic diversity. The practical framework is designed to provide clinicians and health systems with effective upstream stroke prevention strategies that encourage scalability while allowing customization for their local context.

Streamlining and Reimagining Prior Authorization Under Value-Based Contracts: A Call to Action From the Value in Healthcare Initiative's Prior Authorization Learning Collaborative.

Utilization management strategies, including prior authorization, are commonly used to facilitate safe and guideline-adherent provision of new, individualized, and potentially costly cardiovascular therapies. However, as currently deployed, these approaches encumber multiple stakeholders. Patients are discouraged by barriers to appropriate access; clinicians are frustrated by the time, money, and resources required for prior authorizations, the frequent rejections, and the perception of being excluded from the decision-making process; and payers are weary of the intensive effort to design and administer increasingly complex prior authorization systems to balance value and appropriate use of these treatments. These issues highlight an opportunity to collectively reimagine utilization management as a transparent and collaborative system. This would benefit the entire healthcare ecosystem, especially in light of the shift to value-based payment. This article describes the efforts and vision of the multistakeholder Prior Authorization Learning Collaborative of the Value in Healthcare Initiative, a partnership between the American Heart Association and the Robert J. Margolis, MD, Center for Health Policy at Duke University. We outline how healthcare organizations can take greater utilization management responsibility under value-based contracting, especially under different state policies and local contexts. Even with reduced payer-mandated prior authorization in these arrangements, payers and healthcare organizations will have a continued shared need for utilization management. We present options for streamlining these programs, such as gold carding and electronic and automated prior authorization processes. Throughout the article, we weave in examples from cardiovascular care when possible. Although reimagining prior authorization requires collective action by all stakeholders, it may significantly reduce administrative burden for clinicians and payers while improving outcomes for patients.

Improving Cardiovascular Drug and Device Development and Evidence Through Patient-Centered Research and Clinical Trials: A Call to Action From the Value in Healthcare Initiative's Partnering With Regulators Learning Collaborative.

The pipeline of new cardiovascular drugs is relatively limited compared with many other clinical areas. Challenges causing lagging drug innovation include the duration and expense of cardiovascular clinical trials needed for regulatory evaluation and approvals, which generally must demonstrate noninferiority to existing standards of care and measure longer-term outcomes. By comparison, there has been substantial progress in cardiovascular device innovation. There has also been progress in cardiovascular trial participation equity in recent years, especially among women, due in part to important efforts by Food and Drug Administration, National Institutes of Health, American Heart Association, and others. Yet women and especially racial and ethnic minority populations remain underrepresented in cardiovascular trials, indicating much work ahead to continue recent success. Given these challenges and opportunities, the multistakeholder Partnering with Regulators Learning Collaborative of the Value in Healthcare Initiative, a collaboration of the American Heart Association and the Robert J. Margolis, MD, Center for Health Policy at Duke University, identified how to improve the evidence generation process for cardiovascular drugs and devices. Drawing on a series of meetings, literature reviews, and analyses of regulatory options, the Collaborative makes recommendations across four identified areas for improvement. First, we offer strategies to enhance patient engagement in trial design, convenient participation, and meaningful end points and outcomes to improve patient recruitment and retention (major expenses in clinical trials). Second, new digital technologies expand the potential for real-world evidence to streamline data collection and reduce cost and time of trials. However, technical challenges must be overcome to routinely leverage real-world data, including standardizing data, managing data quality, understanding data comparability, and ensuring real-world evidence does not worsen inequities. Third, as trials are driven by evidence needs of regulators and payers, we recommend ways to improve their collaboration in trial design to streamline and standardize efficient and innovative trials, reducing costs and delays. Finally, we discuss creative ways to expand the minuscule proportion of sites involved in cardiovascular evidence generation and medical product development. These actions, paired with continued policy research into better ways to pay for and equitably develop therapies, will help reduce the cost and complexity of drug and device research, development, and trials.

Paying For Value From Costly Medical Technologies: A Framework For Applying Value-Based Payment Reforms.

Innovative medical products offer significant and potentially transformative impacts on health, but they create concerns about rising spending and whether this rise is translating into higher value. The result is increasing pressure to pay for therapies in a way that is tied to their value to stakeholders through improving outcomes, reducing disease complications, and addressing concerns about affordability. Policy responses include the growing application of health technology assessments based on available evidence to determine unit prices, as well as alternatives to volume-based payment that adjust product payments based on predictors or measures of value. Building on existing frameworks for value-based payment for health care providers, we developed an analogous framework for medical products, including drugs, devices, and diagnostic tools. We illustrate each of these types of alternative payment mechanisms and describe the conditions under which each may be useful. We discuss how the use of this framework can help track reforms, improve evidence, and advance policy analysis involving medical product payment.

North Carolina's Health Care Transformation to Value: Progress to Date and Further Steps Needed.

North Carolina has received national attention for its approach to health care payment and delivery reform. Importantly, payment reform alone is not enough to drive systematic changes in care delivery. We highlight the importance of progress in four complementary areas to achieve system-wide payment and care reform.

Advancing Value-Based Models for Heart Failure: A Call to Action From the Value in Healthcare Initiative's Value-Based Models Learning Collaborative.

Heart failure (HF) is a leading cause of hospitalizations and readmissions in the United States. Particularly among the elderly, its prevalence and costs continue to rise, making it a significant population health issue. Despite tremendous progress in improving HF care and examples of innovation in care redesign, the quality of HF care varies greatly across the country. One major challenge underpinning these issues is the current payment system, which is largely based on fee-for-service reimbursement, leads to uncoordinated, fragmented, and low-quality HF care. While the payment landscape is changing, with an increasing proportion of all healthcare dollars flowing through value-based payment models, no longitudinal models currently focus on chronic HF care. Episode-based payment models for HF hospitalization have yielded limited success and have little ability to prevent early chronic disease from progressing to later stages. The available literature suggests that primary care-based longitudinal payment models have indirectly improved HF care quality and cardiovascular care costs, but these models are not focused on addressing patients' longitudinal chronic disease needs. This article describes the efforts and vision of the multi-stakeholder Value-Based Models Learning Collaborative of The Value in Healthcare Initiative, a collaboration of the American Heart Association and the Robert J. Margolis, MD, Center for Health Policy at Duke University. The Learning Collaborative developed a framework for a HF value-based payment model with a longitudinal focus on disease management (to reduce adverse clinical outcomes and disease progression among patients with stage C HF) and prevention (an optional track to prevent high-risk stage B pre-HF from progressing to stage C). The model is designed to be compatible with prevalent payment models and reforms being implemented today. Barriers to success and strategies for implementation to aid payers, regulators, clinicians, and others in developing a pilot are discussed.