Value Assessment Framework Research Articles

Current Value Frameworks—What's New?

Current Value Frameworks—What's New?

PDG10 ALIGNING VALUE IN REGULATORY REVIEW, PRACTICE PATHWAYS, AND HEALTH TECHNOLOGY ASSESSMENT

The recent proliferation of value assessment frameworks for innovative medicines, proposing a range of approaches and methods, has confused many potential users. This policy analysis explores their differences and similarities, and the implications for regulatory and related types of health technology assessment. Approach: We review the objectives and methods of seven different value frameworks including those of the American Society of Clinical Oncology (ASCO), the Institute for Clinical and Economic Research (ICER), and the National Comprehensive Cancer Network (NCCN), and four others. Findings: We find that these frameworks differ substantially in their place and role in the health care system—their “decision context.” Some (e.g., ASCO, NCCN) aim to support clinical shared decision-making. Others (e.g., NCCN) support the development of treatment guidelines and clinical pathways. Finally, others (e.g. ICER) support decisions about inclusion and reimbursement in the health plan benefit package. Clinical value in regulatory benefit-risk balance is a fourth decision context. Net health benefit (NHB) is the key element that links the decisions in this hierarchy of decisions. Regulators judge NHB (benefit-risk balance). Payers assess whether the NHB is worth any additional cost. Health plan managers develop guidelines, pathways, and utilization management tools (e.g., prior authorization), considering both NHB and cost, to provide efficient stewardship of health plan resources. At the shared decision-making level, the focus should be on the physician as the agent helping the patient to assess their individual, tailored NMB based on individual health state preferences. We argue that the four key decision contexts—regulatory benefit-risk analysis, coverage and reimbursement, treatment guidelines and clinical pathways, and clinical shared decision-making—can be aligned by using the QALY as a central value element in each of these related decision contexts, augmented by other value elements, such as insurance value and the value of knowing.

Value-Based Pricing for Emerging Gene Therapies: The Economic Case for a Higher Cost-Effectiveness Threshold.

While one-time gene replacement therapies may offer transformative innovation for the management of ultrarare, health-catastrophic diseases, they also pose challenges to the current U.S. health care system. Historically, the United States and other countries have demonstrated a willingness to support higher prices for health gains in rare diseases. However, payers may be ill-prepared to address reimbursement based on single administrations associated with gene therapies. As yet, there is no consensus on how to appropriately reward gene therapy innovation. The purpose of this article is to characterize challenges for traditional approaches to assessing the value of one-time gene replacement therapies and to provide a health economic rationale for a higher value-based cost-effectiveness threshold (CET). There is a general recognition that ultrarare, health-catastrophic conditions should be judged against a higher CET. The Institute for Clinical and Economic Review in the United States has discussed a range of up to $500K per quality-adjusted life-year (QALY) gained for ultrarare diseases, and the National Institute for Health and Care Excellence in the United Kingdom has described a variable threshold up to £300,000 per QALY depending on the magnitude of the health gains. In practice, health technology assessment decision makers often make comparisons to "benchmarks" to justify both standard and extraordinary CETs. We briefly review and present a list of relevant benchmarks. We also sketch out how a broader concept of value could provide the basis for higher CETs for some ultrarare diseases. This approach is outlined by the recent International Society for Pharmacoeconomics and Outcomes Research Special Task Force on Value Assessment Frameworks. In addition to the QALY gains, other elements of value related to uncertainty may also be important. They include insurance value, severity of disease, real option value, value of hope, and equity. A gene therapy currently in development for the treatment of spinal muscular atrophy (SMA) provides an exemplar for discussing the issues that accompany one-time gene replacement therapies. It is imperative that we find a consensus on how to appropriately reward value created by these gene therapies to incentivize appropriate risk taking and investments by their developers-a higher CET would, by economic logic, support a higher value-based price. If consensus on appropriate rewards cannot be found for safe and effective gene therapies for diseases such as SMA with clear criticality and unmet need, it will be even more difficult to do so for diseases where the value provided is less apparent. DISCLOSURES: Funding for the writing of this article was provided by AveXis Pharmaceuticals, which reviewed the manuscript and contributed feedback during manuscript development. The authors had final editorial control. Jackson and Paul are employees of MME, a biopharmaceutical consulting firm that received funding from AveXis for work on this project. Jackson and Paul also report consulting fees from numerous other biopharmaceutical companies outside of this project. Garrison reports consulting fees from AveXis for work on this project and advisory/consultancy fees from BioMarin, Roche, Novartis, and Pfizer unrelated to this project. Kenston is a former employee of AveXis and reports consulting fees from AveXis for this project and for other projects outside of this work.

Approaches to Capturing Value in Oncology.

This article sets out to describe different value frameworks in the field of new developments in oncology. Since the costs of new oncological therapies follow a steep path, their implementation and financing demand a thorough assessment. This is an ambitious task due to the complex nature of oncological treatments within overall health policy. Five value frameworks were reviewed: European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, American Society of Clinical Oncology (ASCO) Value Framework (version 2.0), National Comprehensive Cancer Network (NCCN) Evidence Blocks, Memorial Sloan Kettering Cancer Center DrugAbacus, and the Institute for Clinical and Economic Review Value Assessment Framework. They are all based on a large set of criteria. However, all these frameworks differ considerably in their outcomes. Among the main differences one has to cite are the inclusion of costs and the use of different outcomes, as well as the fact that they address different target stakeholders, etc. Despite these shortcomings, the value frameworks serve the necessity to introduce more rationality in health decision making seen from the perspective of physicians, patients, and financing bodies.

Acute myeloid leukemia in the elderly: what constitutes treatment value?

Treatment options for patients with acute myeloid leukemia (AML), who are unfit for induction chemotherapy are unsatisfactory. Overall survival (OS) superiority has not been demonstrated in randomized controlled trials (RCT) in this population, challenging the value of available therapies. We sought to assess the relative value of approved therapies using value-assessment tools. Clinical, safety, quality-of-life (QOL), supportive care, and resource utilization outcomes data were abstracted from RCTs and examined using value-assessment frameworks. Three RCTs, one each of azacitidine, decitabine, and low-dose cytarabine were identified. OS was not statistically significant and secondary outcomes including response rates, rates of transfusion independence, the frequency of hospitalizations and changes in QOL were reported differently across trials. Value-assessment tools considered OS as the primary efficacy endpoint without consideration to response rates. The NCCN Evidence BlocksTM were most successful in considering secondary endpoints. With the move toward value-based care, understanding how these value tools apply to AML patients is critical.

PHP286 - RESPONSES OF ISPOR MEMBERS TO THE RECOMMENDATIONS OF THE SPECIAL TASK FORCE ON US VALUE ASSESSMENT FRAMEWORKS

The concept of value based healthcare as a metaphor for assessing new therapeutic interventions has yet to identify additional metrics besides cost-effectiveness to inform public and private decision making. An ISPOR Special Task Force on Value Assessment Frameworks has recently been investigating the drivers in reimbursement decisions. Delegates at the ISPOR Europe 2017 Conference were invited to complete a questionnaire that specifically addressed current opinion on value assessment. This random survey attracted over 80 delegates from 24 countries, nearly 60% of whom considered cost-utility analysis to be a necessary component of a Value Assessment Framework. There was little agreement on whether a single transparent threshold for cost-effectiveness was appropriate, with many respondents suggesting a threshold that is much higher than the current cost/QALY. Over half (55%) of respondents considered surrogate endpoints to be acceptable for reimbursement decisions, whilst two thirds (64%) felt that MCDA might be a replacement for the QALY. Over half (53%) also felt that patients should play a more active role in health technology assessments, and risk sharing was considered to be an acceptable method of cost containment by over 70%. Finally, the majority of respondents (74%) felt it appropriate for manufacturers to command high prices for orphan drugs. In a country analysis, albeit limited by small numbers, there was general congruence between Europe and the US, apart from the need for cost-effectiveness analysis (60 vs 36% in favour, respectively). The Special Task Force has now recommended a refinement of the QALY to reflect societal values, and consider MCDA to be a viable alternative, whilst value assessment should incorporate both patient and population perspectives. The findings of this survey of ISPOR delegates were consistent with these recommendations, which illustrates the interest in the need for a Value Assessment Framework to aid reimbursement decisions.

AMCP Partnership Forum: Managing Care in the Wave of Precision Medicine.

Precision medicine, the customization of health care to an individual's genetic profile while accounting for biomarkers and lifestyle, has increasingly been adopted by health care stakeholders to guide the development of treatment options, improve treatment decision making, provide more patient-centered care, and better inform coverage and reimbursement decisions. Despite these benefits, key challenges prevent its broader use and adoption. On December 7-8, 2017, the Academy of Managed Care Pharmacy convened a group of stakeholders to discuss these challenges and provide recommendations to facilitate broader adoption and use of precision medicine across health care settings. These stakeholders represented the pharmaceutical industry, clinicians, patient advocacy, private payers, device manufacturers, health analytics, information technology, academia, and government agencies. Throughout the 2-day forum, participants discussed evidence requirements for precision medicine, including consistent ways to measure the utility and validity of precision medicine tests and therapies, limitations of traditional clinical trial designs, and limitations of value assessment framework methods. They also highlighted the challenges with evidence collection and data silos in precision medicine. Interoperability within and across health systems is hindering clinical advancements. Current medical coding systems also cannot account for the heterogeneity of many diseases, preventing health systems from having a complete understanding of their patient population to inform resource allocation. Challenges faced by payers, such as evidence limitations, to inform coverage and reimbursement decisions in precision medicine, as well as legal and regulatory barriers that inhibit more widespread data sharing, were also identified. While a broad range of perspectives was shared throughout the forum, participants reached consensus across 2 overarching areas. First, there is a greater need for common definitions, thresholds, and standards to guide evidence generation in precision medicine. Second, current information silos are preventing the sharing of valuable data. Collaboration among stakeholders is needed to support better information sharing, awareness, and education of precision medicine for patients. The recommendations brought forward by this diverse group of experts provide a set of solutions to spur widespread use and application of precision medicine. Taken together, successful adoption and use of precision medicine will require input and collaboration from all sectors of health care, especially patients. DISCLOSURES This AMCP Partnership Forum and the development of the proceedings document were supported by Amgen, Foundation Medicine, Genentech, Gilead, MedImpact, National Pharmaceutical Council, Precision for Value, Sanofi, Takeda, and Xcenda.

AMCP Partnership Forum: Managing Care in the Wave of Precision Medicine.

Precision medicine, the customization of health care to an individual's genetic profile while accounting for biomarkers and lifestyle, has increasingly been adopted by health care stakeholders to guide the development of treatment options, improve treatment decision making, provide more patient-centered care, and better inform coverage and reimbursement decisions. Despite these benefits, key challenges prevent its broader use and adoption. On December 7-8, 2017, the Academy of Managed Care Pharmacy convened a group of stakeholders to discuss these challenges and provide recommendations to facilitate broader adoption and use of precision medicine across health care settings. These stakeholders represented the pharmaceutical industry, clinicians, patient advocacy, private payers, device manufacturers, health analytics, information technology, academia, and government agencies. Throughout the 2-day forum, participants discussed evidence requirements for precision medicine, including consistent ways to measure the utility and validity of precision medicine tests and therapies, limitations of traditional clinical trial designs, and limitations of value assessment framework methods. They also highlighted the challenges with evidence collection and data silos in precision medicine. Interoperability within and across health systems is hindering clinical advancements. Current medical coding systems also cannot account for the heterogeneity of many diseases, preventing health systems from having a complete understanding of their patient population to inform resource allocation. Challenges faced by payers, such as evidence limitations, to inform coverage and reimbursement decisions in precision medicine, as well as legal and regulatory barriers that inhibit more widespread data sharing, were also identified. While a broad range of perspectives was shared throughout the forum, participants reached consensus across 2 overarching areas. First, there is a greater need for common definitions, thresholds, and standards to guide evidence generation in precision medicine. Second, current information silos are preventing the sharing of valuable data. Collaboration among stakeholders is needed to support better information sharing, awareness, and education of precision medicine for patients. The recommendations brought forward by this diverse group of experts provide a set of solutions to spur widespread use and application of precision medicine. Taken together, successful adoption and use of precision medicine will require input and collaboration from all sectors of health care, especially patients. DISCLOSURES This AMCP Partnership Forum and the development of the proceedings document were supported by Amgen, Foundation Medicine, Genentech, Gilead, MedImpact, National Pharmaceutical Council, Precision for Value, Sanofi, Takeda, and Xcenda.

A Health Economics Approach to US Value Assessment Frameworks—Introduction: An ISPOR Special Task Force Report [1

Concerns about rising spending on prescription drugs and other areas of health care have led to multiple initiatives in the United States designed to measure and communicate the value of pharmaceuticals and other technologies for decision making. In this section we introduce the work of the International Society for Pharmacoeconomics and Outcomes Research Special Task Force on US Value Assessment Frameworks formed to review relevant perspectives and appropriate approaches and methods to support the definition and use of high-quality value frameworks. The Special Task Force was part of the International Society for Pharmacoeconomics and Outcomes Research Initiative on US Value Assessment Frameworks, which enlisted the expertise of leading health economists, concentrating on what the field of health economics can provide to help inform the development and use of value assessment frameworks. We focus on five value framework initiatives: the American College of Cardiology/American Heart Association, the American Society of Clinical Oncology, the Institute for Clinical and Economic Review, the Memorial Sloan Kettering Cancer Center, and the National Comprehensive Cancer Network. These entities differ in their missions, scope of activities, and methodological approaches. Because they are gaining visibility and some traction in the United States, it is essential to scrutinize whether the frameworks use approaches that are transparent as well as conceptually and methodologically sound. Our objectives were to describe the conceptual bases for value and its use in decision making, critically examine existing value frameworks, discuss the importance of sound conceptual underpinning, identify key elements of value relevant to specific decision contexts, and recommend good practice in value definition and implementation as well as areas for further research.

ISPOR’s Initiative on US Value Assessment Frameworks: Seeking a Role for Health Economics

ISPOR’s Initiative on US Value Assessment Frameworks: Seeking a Role for Health Economics

A Health Economics Approach to US Value Assessment Frameworks—Summary and Recommendations of the ISPOR Special Task Force Report [7

This summary section first lists key points from each of the six sections of the report, followed by six key recommendations. The Special Task Force chose to take a health economics approach to the question of whether a health plan should cover and reimburse a specific technology, beginning with the view that the conventional cost-per-quality-adjusted life-year metric has both strengths as a starting point and recognized limitations. This report calls for the development of a more comprehensive economic evaluation that could include novel elements of value (e.g., insurance value and equity) as part of either an “augmented” cost-effectiveness analysis or a multicriteria decision analysis. Given an aggregation of elements to a measure of value, consistent use of a cost-effectiveness threshold can help ensure the maximization of health gain and well-being for a given budget. These decisions can benefit from the use of deliberative processes. The six recommendations are to: 1) be explicit about decision context and perspective in value assessment frameworks; 2) base health plan coverage and reimbursement decisions on an evaluation of the incremental costs and benefits of health care technologies as is provided by cost-effectiveness analysis; 3) develop value thresholds to serve as one important input to help guide coverage and reimbursement decisions; 4) manage budget constraints and affordability on the basis of cost-effectiveness principles; 5) test and consider using structured deliberative processes for health plan coverage and reimbursement decisions; and 6) explore and test novel elements of benefit to improve value measures that reflect the perspectives of both plan members and patients.

ISPOR’s Initiative on US Value Assessment Frameworks: The Use of Cost-Effectiveness Research in Decision Making among US Insurers

ISPOR’s Initiative on US Value Assessment Frameworks: The Use of Cost-Effectiveness Research in Decision Making among US Insurers

ISPOR’s Initiative on US Value Assessment Frameworks: An Industry Perspective

ISPOR’s Initiative on US Value Assessment Frameworks: An Industry Perspective

ISPOR’s Initiative on US Value Assessment Frameworks: A Missed Opportunity for ISPOR and Patients

ISPOR’s Initiative on US Value Assessment Frameworks: A Missed Opportunity for ISPOR and Patients

Review of Recent US Value Frameworks—A Health Economics Approach: An ISPOR Special Task Force Report [6

The sixth section of our Special Task Force (STF) report reviews and comments on recent US-oriented value assessment frameworks, specifically those published by the American College of Cardiology/American Heart Association, the Institute for Clinical and Economic Research, the American Society of Clinical Oncology, the National Comprehensive Cancer Network, and the Memorial Sloan Kettering Cancer Center. We review published commentaries that address the validity, reliability, and conceptual underpinnings of these frameworks. We find common themes of critique regarding the strengths and limitations across frameworks. Particular shortcomings of some frameworks pose greater threats to their face validity and utility compared with others. The most significant limitations include lack of clear perspective (e.g., patient vs. health plan) and poor transparency in accounting for costs and benefits. We then review how each framework adheres to core STF recommendations, with particular emphasis on whether the framework can be used to support coverage decisions by health insurers, and whether it adheres to core principles of cost-effectiveness analysis. The Institute for Clinical and Economic Research framework most closely adheres to core STF recommendations. Others have significant limitations that vary widely from framework to framework. We also review how the frameworks follow STF recommendations for addressing potentially relevant issues beyond cost-effectiveness analysis - for example, equity in resource allocation and patient heterogeneity. Finally, we review whether and how each framework uses value thresholds and addresses affordability concerns. We conclude with suggestions for further research, particularly in the areas of testing the measurement and use of novel elements of value and deliberative processes.

DECISION-COMPONENTS OF NICE'S TECHNOLOGY APPRAISALS ASSESSMENT FRAMEWORK.

Value assessment frameworks have gained prominence recently in the context of U.S. healthcare. Such frameworks set out a series of factors that are considered in funding decisions. The UK's National Institute of Health and Care Excellence (NICE) is an established health technology assessment (HTA) agency. We present a novel application of text analysis that characterizes NICE's Technology Appraisals in the context of the newer assessment frameworks and present the results in a visual way. A total of 243 documents of NICE's medicines guidance from 2007 to 2016 were analyzed. Text analysis was used to identify a hierarchical set of decision factors considered in the assessments. The frequency of decision factors stated in the documents was determined and their association with terms related to uncertainty. The results were incorporated into visual representations of hierarchical factors. We identified 125 decision factors, and hierarchically grouped these into eight domains: Clinical Effectiveness, Cost Effectiveness, Condition, Current Practice, Clinical Need, New Treatment, Studies, and Other Factors. Textual analysis showed all domains appeared consistently in the guidance documents. Many factors were commonly associated with terms relating to uncertainty. A series of visual representations was created. This study reveals the complexity and consistency of NICE's decision-making processes and demonstrates that cost effectiveness is not the only decision-criteria. The study highlights the importance of processes and methodology that can take both quantitative and qualitative information into account. Visualizations can help effectively communicate this complex information during the decision-making process and subsequently to stakeholders.

Resolving Lingering Problems or Continued Support for Pseudoscience? The ICER Value Assessment Update

The Institute for Clinical and Economic Review (ICER) released its updated value assessment framework in mid-2017. This included refinements to its conceptual structure and modifications to methods of collecting and assessing evidence. Consequent to this release, a number of authors have commented on the updated value framework, addressing the question of whether the latest framework represents a major revision or merely attempts to resolve lingering problems. The purpose of this commentary is twofold: (i) to revisit what are considered to be fundamental flaws in the ICER value assessment framework and (ii) to question whether or not post-release critiques of the value framework address the fundamental weaknesses in the ICER approach: the absence of credible, evaluable and replicable claims for the benefits and harms of a therapy intervention. The commentary argues that while ICER sees the purpose of its value assessment framework as forming ‘the backbone of rigorous, transparent evidence reports’ in placing ‘scientific methods of evidence analysis at the heart of a clearer and more transparent process’ it falls far short of these ideals. Rather, in attempting to replicate in the US health care environment the evaluation framework mandated by the National Institute for Health and Care Excellence (NICE) in the UK, the ICER falls into the trap of generating value claims for product impact that fail to meet the standards of normal science.
 
 Type: Commentary

Transparency, Imaginary Worlds and ICER Value Assessments

The Institute for Clinical and Economic Review (ICER) is seen as offering a credible platform for evaluating the pricing policies for pharmaceutical products and devices. Over the past few years ICER has presented a stream of reports, many of which have recommended substantial price discounts where the results of a lifetime cost-per-QALY modeling suggests they are out of line with notional willingness to pay thresholds and arbitrary budget constraints. At the same time, there have been growing concerns over the lack of transparency in the ICER value assessment process, focusing in particular on the refusal by ICER to allow access to its value assessment modeling framework. The purpose of this brief commentary is to point out that the position taken by ICER over model access is not defensible; the arguments given are specious. This ongoing refusal undercuts the ICER claim to be independent and the credibility of ICER recommendations for price discounting. The solution is for ICER to commit to a transparent process of value assessment, allowing in particular access to its models and for the ICER model to be subject to an independent assessment. At the same time, manufacturers and other stakeholders should have access to the model with the opportunity to challenge the model through developing model frameworks which they feel better represent product value. This advocacy, it should be noted, does not reflect acceptance of the ICER lifetime cost-per-QALY value assessment framework. Health care decision makers would be better served by a value assessment framework that provided short-term credible, evaluable and replicable claims, facilitating meaningful feedback to decision makers, and not on the construction of simulated imaginary worlds.
 Conflict of Interest: None
 
 Type: Commentary

A Comparative Review of Value Assessment Frameworks In Germany, France, England, Australia, and South Korea: An Industry Perspective

A Comparative Review of Value Assessment Frameworks In Germany, France, England, Australia, and South Korea: An Industry Perspective

Trends In Emerging Non-Hta Drug Value Assessment Frameworks

Trends In Emerging Non-Hta Drug Value Assessment Frameworks