Experiences and Interest in Value-Based Payment Arrangements for Medical Products Among Medicaid Agencies: An Exploratory Analysis.

Policy Points State Medicaid experience with value-based payment (VBP) arrangements for medical products is still relatively limited, and states face a number of challenges in designing and implementing such arrangements, particularly because of the resource-intensive nature of arrangements and data needed to support measurement of desired outcomes. A number of success factors and opportunities to support VBP arrangement efforts were identified through this study, including leveraging established venues or processes for collaboration with manufacturers, engaging external and internal partners in VBP efforts to bolster capabilities, acquiring access to new data sources, and utilizing annual renegotiation of contracts to allow for adjustments. CONTEXT: To date, uptake of value-based payment (VBP) arrangements for medical products and knowledge of their design and impact have been mainly concentrated among private payers. Interest and activity are expanding to Medicaid; however, their experiences and approaches to VBP arrangements for medical products are not well characterized. METHODS: This study sought to characterize the use of VBP arrangements for medical products among state Medicaid agencies through the use of a two-staged, mixed-methods approach. A survey and semistructured interviews were conducted to gain an understanding of state experiences with VBP arrangements for medical products. The survey and interviews were directed at senior leaders from nine states through the survey, with respondents from seven of these states additionally participating in the semistructured interviews. FINDINGS: Although experience with VBP arrangements for medical products among states varied, there were similarities across their motivations and general processes or phases employed in their design and implementation. States collectively identified a number of significant challenges to VBP arrangements, such as manufacturer engagement, outcomes measurement, and the time, expertise, and resources required to design and implement them. We outline a range of strategies to help address these gaps and make it easier for states to pursue VBP arrangements, including more direct engagement from the Center for Medicare and Medicaid Services, state-to-state peer learning and collaboration, data infrastructure and sharing, and additional research to inform fit-for-purpose VBP arrangement approaches. CONCLUSIONS: Findings from this study suggest that it may be easier for states to pursue VBP arrangements for medical products if there is greater clarity on processes employed that support design and implementation as well as effective strategies to address common challenges associated with contract negotiations. As states gain more experience, it will be important to monitor the design and implementation of common VBP arrangements to assess impact on the Medicaid program and the populations it serves.

Beta amyloid PET scans for dementia diagnoses: Practice and research implications from CARE-IDEAS.

Beta amyloid PET scans are a minimally invasive biomarker that may inform Alzheimer's disease (AD) diagnosis. The Caregiver's Reactions and Experience (CARE) study, an IDEAS supplement, aimed to understand experiences of PET scan recipients and their care partners regarding motivations for scans, reporting and interpreting results, and impact of results. Patients with mild cognitive impairment or dementia who agreed to join the CARE-IDEAS study and their care partners participated in a baseline survey and follow-up survey approximately 18 months later, supplemented by in-depth qualitative interviews with subsets of participants. Patients who received scans and volunteered for follow-up research were more likely to be male, better educated, and have higher income than the general population. Survey information was merged with Medicare data. This article integrates findings from several CARE-IDEAS publications and provides implications for practice and research. Although most participants accurately reported scan results, they were often confused about their meaning for prognosis. Some participants reported distress with results, but there were no significant changes in measured depression, burden, or economic strain over time. Many respondents desired more information about prognosis and supportive resources. Scan results were not differentially associated with changes in service use over time. Findings suggest a need for carefully designed and tested tools for clinicians to discuss risks and benefits of scans and their results, and resources to support patients and care partners in subsequent planning. Learning of scan results provides a point-of-contact that should be leveraged to facilitate shared decision-making and person-centered longitudinal AD care.

Elevated Amyloid-ß PET Scan and Cognitive and Functional Decline in Mild Cognitive Impairment and Dementia of Uncertain Etiology.

BACKGROUND: Elevated amyloid-ß (Aß) on positron emission tomography (PET) scan is used to aid diagnosis of Alzheimer's disease (AD), but many prior studies have focused on patients with a typical AD phenotype such as amnestic mild cognitive impairment (MCI). Little is known about whether elevated Aß on PET scan predicts rate of cognitive and functional decline among those with MCI or dementia that is clinically less typical of early AD, thus leading to etiologic uncertainty. OBJECTIVE: We aimed to investigate whether elevated Aß on PET scan predicts cognitive and functional decline over an 18-month period in those with MCI or dementia of uncertain etiology. METHODS: In 1,028 individuals with MCI or dementia of uncertain etiology, we evaluated the association between elevated Aß on PET scan and change on a telephone cognitive status measure administered to the participant and change in everyday function as reported by their care partner. RESULTS: Individuals with either MCI or dementia and elevated Aß (66.6% of the sample) showed greater cognitive decline compared to those without elevated Aß on PET scan, whose cognition was relatively stable over 18 months. Those with either MCI or dementia and elevated Aß were also reported to have greater functional decline compared to those without elevated Aß, even though the latter group showed significant care partner-reported functional decline over time. CONCLUSIONS: Elevated Aß on PET scan can be helpful in predicting rates of both cognitive and functional decline, even among cognitively impaired individuals with atypical presentations of AD.

Continuity of care (COC) and amyloid-ß PET scan: the CARE-IDEAS study.

BACKGROUND: High continuity of care (COC) is associated with better clinical outcomes among older adults. The impact of amyloid-ß PET scan on COC among adults with mild cognitive impairment (MCI) or dementia of uncertain etiology is unknown. METHODS: We linked data from the CARE-IDEAS study, which assessed the impact of amyloid-ß PET scans on outcomes in Medicare beneficiaries with MCI or dementia of uncertain etiology and their care partners, to Medicare claims (2015-2018). We calculated a participant-level COC index using the Bice-Boxerman formula and claims from all ambulatory evaluation and management visits during the year prior to and following the amyloid-ß PET scan. We compared baseline characteristics by scan result (elevated or non-elevated) using standardized differences. To evaluate changes in COC, we used multiple regression models adjusting for sociodemographics, cognitive function, general health status, and the Charlson Comorbidity Index. RESULTS: Among the 1171 cohort members included in our analytic population, the mean age (SD) was 75.2 (5.4) years, 61.5% were male and 93.9% were non-Hispanic white. Over two-thirds (68.1%) had an elevated amyloid-ß PET scan. Mean COC for all patients was 0.154 (SD = 0.102; range = 0-0.73) prior to the scan and 0.158 (SD = 0.105; range = 0-1.0) in the year following the scan. Following the scan, the mean COC index score increased (95% CI) by 0.005 (-0.008, 0.019) points more for elevated relative to not elevated scan recipients, but this change was not statistically significant. There was no association between scan result (elevated vs. not elevated) or any other patient covariates and changes in COC score after the scan. CONCLUSION: COC did not meaningfully change following receipt of amyloid-ß PET scan in a population of Medicare beneficiaries with MCI or dementia of uncertain etiology. Future work examining how care continuity varies across marginalized populations with cognitive impairment is needed.

Amyloid-ß PET Scan Results Disclosure and Care-Partner Emotional Well-Being Over Time.

BACKGROUND: Diagnostic tests, such as amyloid-ß positron emission tomography (PET) scans, can increase appropriate therapeutic management for the underlying causes of cognitive decline. To evaluate the full utility of this diagnostic tool, information is needed on whether results from amyloid-ß PET scans influence care-partner outcomes. OBJECTIVE: This study examines the extent to which previous disclosure of elevated amyloid (suggestive of Alzheimer's disease (AD) etiology) versus not-elevated amyloid (not suggestive of AD etiology) is associated with changes in care-partner wellbeing. METHODS: The study used data derived from a national longitudinal survey of Medicare beneficiaries (n = 921) with mild cognitive impairment (MCI) or dementia and their care-partners. Care-partner wellbeing outcomes included depressive symptoms (PHQ-8), subjective burden (4-item Zarit burden score), and a 3-item measure of loneliness. Change was measured between 4 (Time 1) and 18 (Time 2) months after receiving the scan results. Adjusted linear regression models regressed change (Time 2-Time 1) in each outcome on scan result. RESULTS: Care-partners were primarily white, non-Hispanic, college-educated, and married to the care recipient. Elevated amyloid was not associated with statistically significant Time 1 differences in outcomes or with statistically significant changes in depressive symptoms 0.22 (-0.18, 0.61), subjective burden 0.36 (-0.01, 0.73), or loneliness 0.15 (-0.01, 0.32) for care-partners from one time point to another. CONCLUSION: Given advances in AD biomarker testing, future research in more diverse samples is needed to understand the influence of scan results on care-partner wellbeing across populations.

Navigating the high costs of cancer care: opportunities for patient engagement.

Over the past decade, the financial burden of cancer care on patients and their families has garnered increased attention. Many of the potential solutions have focused on system-level interventions such as adopting value-based payment models and negotiating drug prices; less consideration has been given to actions at the patient level to address cancer care costs. We argue that it is imperative to develop and support patient-level strategies that engage patients and consider their preferences, values and individual circumstances. Opportunities to meet these aims and improve the economic experience of patients in oncology are discussed, including: shared decision-making and communication, financial navigation and treatment planning, digital technology and alternative care pathways, and value-based insurance design.

Lay abstract The financial burden of cancer care on patients and their families is a growing problem and action is critically needed to alleviate the high costs of such care. So far, potential solutions have focused on system-level interventions, with less consideration given to solutions at the patient level. This review argues that it is imperative to develop and support patient-level strategies that engage patients. Next, the review presents evidence of the interplay between patient preferences and values and the costs of cancer care. Finally, opportunities to enhance engagement and improve the economic experience of patients in oncology are discussed, including: shared decision-making and communication, financial navigation and treatment planning, digital technology and alternative care pathways, and value-based insurance design.

Value-based arrangements may be more prevalent than assumed.

OBJECTIVES: To better understand the prevalence of US value-based payment arrangements (VBAs), their characteristics, and the factors that facilitate their success or act as barriers to their implementation. STUDY DESIGN: Surveys were administered to a convenience sample of subject matter experts who were senior representatives from payer organizations and biopharmaceutical manufacturers. These data were supplemented with qualitative interviews in a subsample of survey respondents. METHODS: Descriptive statistics, including percentages for categorical values and mean (SD) and median (interquartile range) for continuous variables, were assessed for quantitative questions. Trained reviewers collated responses to free-text survey questions and the qualitative interviews to identify themes. RESULTS: Of the 25 respondents, 1 manufacturer and 4 payers reported not having explored or negotiated any VBAs. Subsequently, questionnaire results from 11 biopharmaceutical manufacturers and 9 payers who had experience with VBAs were analyzed. More than 70% of VBAs implemented between 2014 and 2017 were not publicly disclosed. Furthermore, although consideration of VBAs as a coverage and payment tool is increasing, VBA implementation is relatively low, with manufacturers and payers reporting that approximately 33% and 60% of early dialogues translate into signed VBA contracts, respectively. Respondents' reasoning for VBA negotiation process breakdowns generally differed by sector and reflected each sector's respective priorities. CONCLUSIONS: This study reveals that the majority of VBAs are not publicly disclosed, which could underestimate their true prevalence and impact. Given the effort required to implement a VBA, future arrangements would likely benefit from a framework or other evaluative tool to help assess VBA pursuit desirability and guide the negotiation and implementation process.

Advancing Value Assessment in the United States: A Multistakeholder Perspective.

Rising costs without perceived proportional improvements in quality and outcomes have motivated fundamental shifts in health care delivery and payment to achieve better value. Aligned with these efforts, several value assessment frameworks have been introduced recently to help providers, patients, and payers better understand the potential value of drugs and other interventions and make informed decisions about their use. Given their early stage of development, it is imperative to evaluate these efforts on an ongoing basis to identify how best to support and improve them moving forward. This article provides a multistakeholder perspective on the key limitations and opportunities posed by the current value assessment frameworks and areas of and actions for improvement. In particular, we outline 10 fundamental guiding principles and associated strategies that should be considered in subsequent iterations of the existing frameworks or by emerging initiatives in the future. Although value assessment frameworks may not be able to meet all the needs and preferences of stakeholders, we contend that there are common elements and potential next steps that can be supported to advance value assessment in the United States.

The role of hospital payments in the adoption of new medical technologies: an international survey of current practice.

This study examined the role of prospective payment systems in the adoption of new medical technologies across different countries. A literature review was conducted to provide background for the study and guide development of a survey instrument. The survey was disseminated to hospital payment systems experts in 15 jurisdictions. Fifty-one surveys were disseminated, with 34 returned. The surveys returned covered 14 of the 15 jurisdictions invited to participate. The majority (71%) of countries update the patient classification system and/or payment tariffs on an annual basis to try to account for new technologies. Use of short-term separate or supplementary payments for new technologies occurs in 79% of countries to ensure adequate funding and facilitate adoption. A minority (43%) of countries use evidence of therapeutic benefit and/or costs to determine or update payment tariffs, although it is somewhat more common in establishing short-term payments. The main barrier to using evidence is uncertain or unavailable clinical evidence. Almost three-fourths of respondents believed diagnosis-related group systems incentivize or deter technology adoption, depending on the particular circumstances. Improvements are needed, such as enhanced strategies for evidence generation and linking evidence of value to payments, national and international collaboration and training to improve existing practice, and flexible timelines for short-term payments. Importantly, additional research is needed to understand how different payment policies impact technology uptake as well as quality of care and costs.

Improving medical device regulation: the United States and Europe in perspective.

CONTEXT: Recent debates and events have brought into question the effectiveness of existing regulatory frameworks for medical devices in the United States and Europe to ensure their performance, safety, and quality. This article provides a comparative analysis of medical device regulation in the two jurisdictions, explores current reforms to improve the existing systems, and discusses additional actions that should be considered to fully meet this aim. Medical device regulation must be improved to safeguard public health and ensure that high-quality and effective technologies reach patients. METHODS: We explored and analyzed medical device regulatory systems in the United States and Europe in accordance with the available gray and peer-reviewed literature and legislative documents. FINDINGS: The two regulatory systems differ in their mandate and orientation, organization, pre- and postmarket evidence requirements, and transparency of process. Despite these differences, both jurisdictions face similar challenges for ensuring that only safe and effective devices reach the market, monitoring real-world use, and exchanging pertinent information on devices with key users such as clinicians and patients. To address these issues, reforms have recently been introduced or debated in the United States and Europe that are principally focused on strengthening regulatory processes, enhancing postmarket regulation through more robust surveillance systems, and improving the traceability and monitoring of devices. Some changes in premarket requirements for devices are being considered. CONCLUSIONS: Although the current reforms address some of the outstanding challenges in device regulation, additional steps are needed to improve existing policy. We examine a number of actions to be considered, such as requiring high-quality evidence of benefit for medium- and high-risk devices; moving toward greater centralization and coordination of regulatory approval in Europe; creating links between device identifier systems and existing data collection tools, such as electronic health records; and fostering increased and more effective use of registries to ensure safe postmarket use of new and existing devices.

The politics of comparative effectiveness research: lessons from recent history.

Efforts to support and use comparative effectiveness research (CER), some more successful than others, have been promulgated at various times over the last forty years. Following a resurgence of interest in CER, recent health care reforms provided substantial support to strengthen its role in US health care. While CER has generally captured bipartisan support, detractors have raised concerns that it will be used to ration services and heighten government control over health care. Such concerns almost derailed the initiative during passage of the health care reform legislation and are still present today. Given recent investments in CER and the debates surrounding its development, the time is ripe to reflect on past efforts to introduce CER in the United States. This article examines previous initiatives, highlighting their prescribed role in US health care, the reasons for their success or failure, and the political lessons learned. Current CER initiatives have corrected for many of the pitfalls experienced by previous efforts. However, past experiences point to a number of issues that must still be addressed to ensure the long-term success and sustainability of CER, including adopting realistic aims about its impact, demonstrating the impact of Patient-Centered Outcomes Research Institute (PCORI) and communicating the benefits of CER, and maintaining strong political and stakeholder support.

Medical technology as a key driver of rising health expenditure: disentangling the relationship.

Health care spending has risen steadily in most countries, becoming a concern for decision-makers worldwide. Commentators often point to new medical technology as the key driver for burgeoning expenditures. This paper critically appraises this conjecture, based on an analysis of the existing literature, with the aim of offering a more detailed and considered analysis of this relationship. Several databases were searched to identify relevant literature. Various categories of studies (eg, multivariate and cost-effectiveness analyses) were included to cover different perspectives, methodological approaches, and issues regarding the link between medical technology and costs. Selected articles were reviewed and relevant information was extracted into a standardized template and analyzed for key cross-cutting themes, ie, impact of technology on costs, factors influencing this relationship, and methodological challenges in measuring such linkages. A total of 86 studies were reviewed. The analysis suggests that the relationship between medical technology and spending is complex and often conflicting. Findings were frequently contingent on varying factors, such as the availability of other interventions, patient population, and the methodological approach employed. Moreover, the impact of technology on costs differed across technologies, in that some (eg, cancer drugs, invasive medical devices) had significant financial implications, while others were cost-neutral or cost-saving. In light of these issues, we argue that decision-makers and other commentators should extend their focus beyond costs solely to include consideration of whether medical technology results in better value in health care and broader socioeconomic benefits.

Evolving reimbursement and pricing policies for devices in Europe and the United States should encourage greater value.

Rising health care costs are an international concern, particularly in the United States, where spending on health care outpaces that of other industrialized countries. Consequently, there is growing desire in the United States and Europe to take a more value-based approach to health care, particularly with respect to the adoption and use of new health technology. This article examines medical device reimbursement and pricing policies in the United States and Europe, with a particular focus on value. Compared to the United States, Europe more formally and consistently considers value to determine which technologies to cover and at what price, especially for complex, costly devices. Both the United States and Europe have introduced policies to provide temporary coverage and reimbursement for promising technologies while additional evidence of value is generated. But additional actions are needed in both the United States and Europe to ensure wise value-based reimbursement and pricing policies for all devices, including the generation of better pre- and postmarket evidence and the development of new methods to evaluate value and link evidence of value to reimbursement.

Valuing end-of-life care in the United States: the case of new cancer drugs.

New cancer therapies offer the hope of improved diagnosis to patients with life-threatening disease. Over the past 5-10 years, a number of specialty drugs have entered clinical practice to provide better systemic therapy for advanced cancers that respond to few therapeutic alternatives. To date, however, such advances have been only modestly effective in extending life and come with a high price tag, raising questions about their value for money, patient access and implications for health care costs. This article explores some of the key issues present in valuing end-of-life care in the United States in the case of advanced cancer drugs, from the difficult trade-offs between their limited health benefits and high costs to the technical, political and social challenges in assessing their value and applying such evidence to inform policy and practice. A number of initial steps are discussed that could be pursued to improve the value of advanced cancer care.

Comparative effectiveness research: the experience of the National Institute for Health and Clinical Excellence.

PURPOSE: To assess the relevance of the experience of the National Institute for Health and Clinical Excellence (NICE) in the United Kingdom to the comparative effectiveness research (CER) initiative in the United States. METHODS: The activities of NICE were reviewed to assess its experience in analytic methods, engagement with stakeholders, communication of findings, and implementation of recommendations. RESULTS: The main lessons for the United States from the experience of NICE relate to how the institute has gathered, synthesized, and used information on the clinical and cost effectiveness of health care interventions. The experience of NICE suggests that ways will have to be found to reconcile the differing stakeholder perspectives on the value of health care. Given the emphasis in the United States on being patient centered, there will be situations where patients' expectations for the provision of care far exceed that which payers feel should be made available on grounds of value for money. Explicit restrictions on access to care based on CER like those found in the United Kingdom are unlikely, but alternative solutions, such as value-based reimbursement, will need to be pursued if unnecessary expenditures are to be avoided. It will also be important that the CER initiative show some impact on the use of health care resources. The longer that NICE has been in existence in the United Kingdom, questions about its impact have been more frequently asked, given the resources devoted to its activities. CONCLUSION: Although there are distinct differences between the health systems of the United Kingdom and United States, lessons can be learned from examining the successes and challenges experienced by NICE.

Reflections on the evolution of health technology assessment in Europe.

Health technology assessment (HTA) has assumed an increasing role in health systems in recent years, with many countries establishing agencies or programmes to evaluate health technology and other interventions to inform policy decisions and clinical practice. This paper reflects upon its development and evolution in Europe over the last decade, with a focus on England, France, Germany and Sweden. In particular, we explore how HTA has evolved over time as well as its impact on policy and practice. While countries share many of the same objectives, there are differences in the way HTA agencies and programmes are organised, operate, and influence decision making. Despite these differences, all systems are faced with opportunities and challenges related to stakeholder involvement and acceptance, the suitability and transparency of assessment requirements and methods, balancing evidence and values in decision making, and demonstrating impact.