Digital health & low-value care.

Digital health advances offer a multitude of possibilities to improve public health and individual wellbeing. Little attention has been paid, however, to digital health's potential to create low-value care - the reduction of which is increasingly appreciated as a policy priority. This commentary provides a framework to illustrate the potential for consumer-facing digital health to generate three distinct categories of low-value care; 1) ineffective care because it is underdeveloped, 2) inefficient care because it supplements rather than substitutes, or 3) unwanted care because it is not aligned with clinician and patient preferences. We offer specific policy recommendations to reduce each type of low-value care.

PAYER PERSPECTIVES ON FUTURE ACCEPTABILITY OF COMPARATIVE EFFECTIVENESS AND RELATIVE EFFECTIVENESS RESEARCH.

OBJECTIVES: Our objective was to gather perspectives from payers on how comparative effectiveness research (CER) in the United States and relative effectiveness (RE) research in Europe will impact evidentiary standards for access decisions of new drugs by 2020. METHODS: We conducted semi-structured interviews with fourteen senior officials representing public and private payers, health technology assessment groups, and pricing and reimbursement bodies in the United States and Europe. An online survey assessed current use of CER/RE evidence and potential trends that might influence its use for decision making by 2020. A semi-structured interview elicited payers' definitions of CER/RE and was structured around four hypothetical cases resembling drugs expected to be more common or poised to create policy challenges by 2020. Topics included acceptance of study designs and analytic methods associated with CER/RE. A systematic content review was done to extract relevant information. RESULTS: According to key informants, randomization will remain an essential component for assessing comparative or relative effectiveness. They anticipate greater use of policy levers such as conditional reimbursement or prior authorization to manage diffusion of new drugs. Case studies provided important insights into situations when certain types of CER evidence may be acceptable (e.g., observational data when differences between drugs are largely convenience). CONCLUSIONS: Industry perceptions that CER/RE will change payers' evidentiary requirements in the future are consistent with our findings. Growing investment in payers' own data and increased reliance on policy tools to control diffusion of new drugs may also influence the type of evidence industry will be required to produce by 2020.

Futurescapes: evidence expectations in the USA for comparative effectiveness research for drugs in 2020.

AIM: Explore key factors influencing future expectations for the production of evidence from comparative effectiveness research for drugs in the USA in 2020 and construct three plausible future scenarios. MATERIALS & METHODS: Semistructured key informant interviews and three rounds of modified Delphi with systematic scenario-building methods. RESULTS & CONCLUSION: Most influential key factors were: health delivery system integration; electronic health record development; exploitation of very large databases and mixed data sources; and proactive patient engagement in research. The scenario deemed most likely entailed uneven development of large integrated health systems with pockets of increased provider risk for patient care, enhanced data collection systems, changing incentives to do comparative effectiveness research and new opportunities for evidence generation partnerships.

Futurescapes: expectations in Europe for relative effectiveness evidence for drugs in 2020.

AIM: Explore key factors influencing future expectations for the production of evidence of relative effectiveness (RE) for drugs in Europe in 2020; construct three plausible future scenarios for RE evidence generation. MATERIALS & METHODS: Semi-structured key informant interviews and three rounds of modified Delphi to gather expert perspectives and develop future scenarios. RESULTS & CONCLUSION: Most influential factors were degree of regulator use of postmarketing authorization (postlaunch) efficacy studies and adaptive licensing; degree of pan-European health technology assessment body coordination in reviewing prelaunch evidence and demanding postlaunch studies; the nature of regulator - health technology assessment body interaction. The most likely scenario entailed some change with postlaunch regulatory studies driving the likely nature of RE evidence generated.

The future of comparative effectiveness and relative efficacy of drugs: an international perspective.

Drug development takes place in a global marketplace, albeit with the USA and EU markets currently dominating. In the USA, demands for comparative effectiveness research have gained traction against a backdrop of health delivery reform, while European stakeholders deliberate the role of relative effectiveness in health technology assessment, trying to reduce the duplication of effort by regulators and health technology assessment bodies. In both arenas, drug-makers are faced with mounting drug development costs, and uncertainty over the types of evidence acceptable for a growing list of stakeholders. This article reports and compares future scenarios for evidence expectations for drugs for the USA and EU in 2020. The similarities, differences, and joint implications of the scenarios are considered to create an view of future evidence generation for drugs developed for these markets.

Medical and pharmacy coverage decision making at the population level.

Medicare is one of the largest health care payers in the United States. As a result, its decisions about coverage have profound implications for patient access to care. In this commentary, the authors describe how Medicare used evidence on heterogeneity of treatment effects to make population-based decisions on health care coverage for implantable cardiac defibrillators. This case is discussed in the context of the rapidly expanding availability of comparative effectiveness research. While there is a potential tension between population-based and patient-centered decision making, the expanded diversity of populations and settings included in comparative effectiveness research can provide useful information for making more discerning and informed policy and clinical decisions.

Recommendations for the design of Phase 3 pharmaceutical trials that are more informative for patients, clinicians, and payers.

BACKGROUND: Pharmaceutical pragmatic clinical trials (PCTs) are designed to provide the type of evidence that is desired by patients, clinicians and payers but too often missing from traditional regulatory trials. PURPOSE: This paper presents framework for designing pragmatic trials incorporating evidence desired by post-regulatory decision makers while remaining within acceptable standards for regulatory approval. METHODS: Following a stakeholder meeting convened in May of 2009 to identify gaps in information collected in Phase 3 trials, CMTP staff and the authors drafted recommendations for Pragmatic Phase 3 Pharmaceutical Trials. This draft was circulated first to technical working group members for their comments. After revising the document based on these comments, it was distributed electronically to other select experts and then made available for public comment. The final version of the EGD appears on the CMTP website. RESULTS: The process resulted in a set of 10 recommendations for conducting Phase 3 trials that met regulatory needs while addressing information important to physicians, patients, payers, and policy-makers. These recommendations encompassed three primary areas: generalizability from the trial participants to the clinical population of interest; effectiveness relative to active comparators; and consistently measured relevant outcomes for coverage and treatment decisions. LIMITATIONS: While stakeholders were involved throughout the process, not all recommendations will meet the needs of all stakeholders. CONCLUSIONS: Pragmatic trial design need not be deferred until a product is in widespread use. Incremental movement toward the more pragmatic design of Phase 3 trials is desirable.

Performance-based risk-sharing arrangements-good practices for design, implementation, and evaluation: report of the ISPOR good practices for performance-based risk-sharing arrangements task force.

There is a significant and growing interest among both payers and producers of medical products for agreements that involve a "pay-for-performance" or "risk-sharing" element. These payment schemes-called "performance-based risk-sharing arrangements" (PBRSAs)-involve a plan by which the performance of the product is tracked in a defined patient population over a specified period of time and the amount or level of reimbursement is based on the health and cost outcomes achieved. There has always been considerable uncertainty at product launch about the ultimate real-world clinical and economic performance of new products, but this appears to have increased in recent years. PBRSAs represent one mechanism for reducing this uncertainty through greater investment in evidence collection while a technology is used within a health care system. The objective of this Task Force report was to set out the standards that should be applied to "good practices"-both research and operational-in the use of a PBRSA, encompassing questions around the desirability, design, implementation, and evaluation of such an arrangement. This report provides practical recommendations for the development and application of state-of-the-art methods to be used when considering, using, or reviewing PBRSAs. Key findings and recommendations include the following. Additional evidence collection is costly, and there are numerous barriers to establishing viable and cost-effective PBRSAs: negotiation, monitoring, and evaluation costs can be substantial. For good research practice in PBRSAs, it is critical to match the appropriate study and research design to the uncertainties being addressed. Good governance processes are also essential. The information generated as part of PBRSAs has public good aspects, bringing ethical and professional obligations, which need to be considered from a policy perspective. The societal desirability of a particular PBRSA is fundamentally an issue as to whether the cost of additional data collection is justified by the benefits of improved resource allocation decisions afforded by the additional evidence generated and the accompanying reduction in uncertainty. The ex post evaluation of a PBRSA should, however, be a multidimensional exercise that assesses many aspects, including not only the impact on long-term cost-effectiveness and whether appropriate evidence was generated but also process indicators, such as whether and how the evidence was used in coverage or reimbursement decisions, whether budget and time were appropriate, and whether the governance arrangements worked well. There is an important gap in the literature of structured ex post evaluation of PBRSAs. As an innovation in and of themselves, PBRSAs should also be evaluated from a long-run societal perspective in terms of their impact on dynamic efficiency (eliciting the optimal amount of innovation).

Improving the efficiency and effectiveness of pragmatic clinical trials in older adults in the United States.

Pragmatic clinical trials (PCTs) seek to improve the generalizability and increase the statistical power of traditional explanatory trials. They are a major tenet of comparative effectiveness research. While a powerful study design, PCTs have been limited by high cost, modest efficiency, and limited ability to fill relevant evidence gaps. Based on an American Reinvestment and Recovery Act (ARRA) supported meeting of national stakeholders, we propose several innovations and future research that could improve the efficiency and effectiveness of such studies focused in the U.S. Innovations discussed include optimizing the use of community based practices through partnership with Practice Based Research Networks (PBRNs), using information technology to simplify PCT subject recruitment, consent and randomization processes, and utilizing linkages to large administrative databases, such as Medicare, as a mechanism to capture outcomes and other important PCT variables with lower subject and research team burden. Testing and adaptation of such innovations to PCT are anticipated to improve the public health value of these increasingly important studies.

Looking at CER from Medicare's perspective.

BACKGROUND: Comparative effectiveness research (CER) is rapidly adding to the amount of data available to health care coverage and payment decision makers. Medicare's decisions have a large effect on coverage and reimbursement policies throughout the health insurance industry and will likely influence the entire U.S. health care system; thus, examining its role in integrating CER into policy is crucial. OBJECTIVES: To describe the potential benefits of CER to support payment and coverage decisions in the Medicare program, limitations on its use,the role of the Centers for Medicare & Medicaid Services (CMS) in improving the infrastructure for CER, and to discuss challenges that must be addressed to integrate CER into CMS's decision-making process. SUMMARY: A defining feature of CER is that it provides the type of evidence that will help decision makers, such as patients, clinicians, and payers,make more informed treatment and policy decisions. Because CMS is responsible for more than 47 million elderly and disabled beneficiaries, the way that Medicare uses CER has the potential to have a large impact on public and individual health. Currently many critical payment and coverage decisions within the Medicare program are made on the basis of poor quality evidence, and CER has the potential to greatly improve the quality of decision making. Despite common misconceptions, CMS is not prohibited by law from using CER apart from some reasonable limitations. CMS is,however, required to support the development of the CER infrastructure by making their data more readily available to researchers. While CER has substantial potential to improve the quality of the agency's policy decisions,challenges remain to integrate CER into Medicare's processes. These challenges include statutory ambiguities, lack of sufficient staff and internal resources to take advantage of CER, and the lack of an active voice in setting priorities for CER and study design. CONCLUSION: Although challenges exist, CER has the potential to greatly enhance CMS's ability to make decisions regarding coverage and payment that will benefit both the agency and their patient population.

A translation table for patient-centered comparative effectiveness research: guidance to improve the value of research for clinical and health policy decision-making.

This article provides background and context for a series of papers stemming from a collaborative effort by Outcome Sciences, Inc., the National Pharmaceutical Council and the Center for Medical Technology Policy to use a stakeholder-driven process to develop a decision tool to select appropriate methods for comparative effectiveness research. The perceived need and origins of the 'translation table' concept for method selection are described and the legislative history and role of the Patient-Centered Outcomes Research Institute are reviewed. The article concludes by stressing the significance of this effort for future health services and clinical research, and the importance of consulting end-users--patients, providers, payers and policy-makers--in the process of defining research questions and approaches to them.

Incorporating stakeholder perspectives in developing a translation table framework for comparative effectiveness research.

This project used a stakeholder-driven process to understand the factors that drive the selection of study designs for comparative effectiveness research (CER). The project assembled a diverse stakeholder committee to explore the basis of a translation framework and gathered input through surveys, interviews and an in-person meeting. Stakeholders recommended different study designs for the CER topic areas and identified different outcomes as the most important outcomes to study in each area. During the discussions, stakeholders described a variety of factors that influenced their study design recommendations. The stakeholder activities resulted in the identification of several key themes, including the need to have a highly specific detailed research question before discussing appropriate designs and the need to use multiple studies, potentially of different designs, to address the CER topic areas. The insights and themes from this project may inform efforts to develop a translation table.

Access with evidence development: the US experience.

The concept of access with evidence development (AED), also known as 'coverage with evidence development' in the Medicare programme, has long been discussed as a policy option for ensuring more appropriate use of new technologies in the US. This article provides a comprehensive overview of more than 10 years of US experience with AED, both in the public and private healthcare sectors. Beginning with a discussion of the successes of private plans' conditional coverage for high-density chemotherapy for autologous bone marrow transplants for metastatic breast cancer and Medicare's conditional coverage of lung-volume-reduction surgery in the 1990s, the article moves on to describe how Medicare worked to codify AED as one of its coverage policy options in the early part of this decade. More recent private and public sector initiatives are also discussed, including an overview of barriers to implementing AED. Despite the complexity of political, financial and ethical issues faced in implementation, AED is now a permanent fixture of US coverage policy. Future initiatives within the Medicare programme and with private payers in the US are much more likely to succeed by relying upon the simple but consequential principles laid out at a Summit convened in Banff, Alberta, Canada in 2009 and presented in another article in this issue.

Impact of Medicare Part D on access to and cost sharing for specialty biologic medications for beneficiaries with rheumatoid arthritis.

OBJECTIVE: Many worry that the use of specialty tiering for biologic disease-modifying antirheumatic drugs (DMARDs) by Medicare Part D plans imposes a heavy financial burden on beneficiaries with rheumatoid arthritis (RA). To date, no one has examined the cost-sharing structures for biologic DMARDs in Part D plans or the resulting cost burden for patients. METHODS: We followed 14,929 vulnerable, low-income patients with RA who were enrolled in the Medicare Replacement Drug Demonstration (MRDD) in 2005. As the MRDD population transitioned into Part D in 2006, we examined correlates of Part D enrollment and compared the cost-sharing provisions for biologic DMARDs in the Medicare Advantage and stand-alone plans. We simulated the out-of-pocket costs of beneficiaries under 3 cost-sharing scenarios. RESULTS: Eighty-one percent of MRDD beneficiaries with RA enrolled in Part D. Enrollment predictors were female sex (odds ratio [OR] 1.48, 95% confidence interval [95% CI] 1.32-1.67), prior MRDD benefit use (OR 2.29, 95% CI 2.04-2.58), other self-reported drug coverage (OR 1.53, 95% CI 1.36-1.71), and receiving an MRDD subsidy (OR 2.00, 95% CI 1.74-2.30). Compared with stand-alone plans, Medicare Advantage plans had lower deductibles, lower premiums, and fewer prior authorization, step therapy, and quantity limit restrictions. However, approximately 75% of all plans used coinsurance as the preferred form of cost sharing. Out-of-pocket costs exceeded $4,000 annually in all cost-sharing scenarios. CONCLUSION: Most MRDD beneficiaries with RA enrolled in Part D. Although plans assume some costs for biologic DMARDs, the majority of costs are shifted to beneficiaries and to Medicare. Such cost shifting may place these medications out of the beneficiary's financial reach and expose Medicare to high financial liability.

The heterogeneity of schizophrenia in disease states.

PREVIOUS PRESENTATION: Some of the contents of this paper have been previously presented at the 16th Annual Meeting of the International Society for Technology Assessment in Health Care June 20, 2000 in the Hague, Netherlands and at the 21st Annual Meeting of the Society for Medical Decision Making as a poster on October 3, 1999 in Reno, NV. BACKGROUND: Studies of schizophrenia treatment often oversimplify the array of health outcomes among patients. Our objective was to derive a set of disease states for schizophrenia using the Positive and Negative Symptom Assessment Scale (PANSS) that captured the heterogeneity of symptom responses. METHODS: Using data from a 1-year clinical trial that collected PANSS scores and costs on schizophrenic patients (N=663), we conducted a k-means cluster analyses on PANSS scores for items in five factor domains. Results of the cluster analysis were compared with a conceptual framework of disease states developed by an expert panel. Final disease states were defined by combining our conceptual framework with the empirical results. We tested its utility by examining the influence of disease state on treatment costs and prognosis. RESULTS: Analyses led to an eight-state framework with varying levels of positive, negative, and cognitive impairment. The extent of hostile/aggressive symptoms and mood disorders correlated with severity of disease states. Direct treatment costs for schizophrenia vary significantly across disease states (F=27.47, df=7, p<0.0001), and disease state at baseline was among the most important predictors of treatment outcomes. CONCLUSION: The disease states we describe offer a useful paradigm for understanding the links between symptom profiles and outcomes.

Public preferences for health states with schizophrenia and a mapping function to estimate utilities from positive and negative symptom scale scores.

BACKGROUND: Schizophrenia is a common severe syndrome with a highly variable pattern of symptoms. The medications used to treat this disorder are expensive and may cause severe adverse effects. Little is known about how the public perceives health outcomes in schizophrenia or the potential adverse effects of antipsychotic medication. This complicates the use of standard cost-effectiveness analysis to set priorities for health resource allocation. OBJECTIVE: In this study, we measured utility weights for a set of health states derived from Positive and Negative Symptom Scale (PANSS) scores, and a set of health states that included the presence of common adverse effects of medication, thus creating utility mapping function for clinical data. METHODS: We presented a convenience sample of members of a large commercial Internet survey panel with digital video materials portraying eight different patterns of schizophrenia with varying levels of positive, negative and cognitive symptoms, and five common adverse effects of antipsychotic medications. We then elicited their standard gamble (SG) and visual analog scale (VAS) ratings using iMPACT3 computer program, with an automated error repair feature, deliberately over-sampling minority ethnic groups in the panel. We censored subjects with uncorrected errors in ratings and estimated utilities for each state by re-weighting responses to match United States population demographics. SUBJECTS: 620 well-educated, ethnically diverse volunteers (54% Caucasian), who spanned a broad range of age groups and geographical regions of the US participated in this study. RESULTS: Because of evidence of bias in ratings, 175 (29%) subjects with internal inconsistencies in ratings were censored from estimates of mean population utilities. In the remaining 441 subjects, SG utilities, re-weighted to match US population demographic profiles, ranged from 0.88 for mild schizophrenia to 0.47 extremely severe schizophrenia. Variability in the types of symptoms exhibited (positive, negative or cognitive) was less important to participants than the overall severity of each state. Modest reductions in symptoms (for example, from severe to moderate or moderate to mild) were associated with relatively large changes in utility (0.12-0.19). Adverse effects decreased utilities for states by amounts ranging from 0.09 (pseudo-parkinsonism) to 0.05 (obesity). CONCLUSIONS: The public views schizophrenia is a very disabling syndrome and treatments that reduce symptoms produce important gains in utility, even if they do not induce a complete remission. Adverse effects significantly reduce utilities for states; therefore, cost-effectiveness analyses for this disorder should take adverse effects of treatment into consideration.

The implementation of Medicare's outpatient prospective payment system and specific concerns for rural hospitals.

The transition to Medicare's new prospective payment system for hospital outpatient services has arguable been the most complex and difficult programmatic change in the history of Medicare (Federal Register, 2002). Concern about its adverse effects led to holding rural hospitals with 100 beds or fewer harmless from the financial consequences of the new payment system for the first three years. However, small rural hospitals were not held harmless from implementing the outpatient prospective payment system (OPPS). Many outside observers felt that small rural hospitals would be ill-equipped to handle the immensity of change required, and that claim denials or delays caused by inaccurate claims submissions might have a disproportionate effect on smaller hospitals. There were also reports about difficulties with the interim payment system that had been designed to ensure small hospitals did not lose money during the first three years. This policy brief describes issues that arose in implementing OPPS during the first years of the program, identifies specific implementation concerns for small rural hospitals, and raises issues that may warrant further research or policy action.