Evolving Use of Health Technology Assessment in Medical Device Procurement-Global Systematic Review: An ISPOR Special Interest Group Report.

OBJECTIVES: To review the current academic evidence describing how data from health technology assessments (HTAs) informs procurement decisions for medical devices. METHODS: A systematic literature review was performed to identify relevant studies and criteria used in medical device purchasing or procurement decisions. Included articles were screened for relevancy and risk of bias. The included studies were summarized qualitatively. RESULTS: A total of 292 studies were screened, of which 11 matched the inclusion criteria. Included studies' geographies and HTA maturity varied. Some studies described hospital-level HTA processes, whereas others focused on national-level recommendations. Criteria for procurement decisions included standard HTA factors, such as efficacy, cost, cost-effectiveness, and budget impact; broader issues were also noted, including impact on the organization, ethical aspects, staff workload, and volume. There was little consideration of device-specific characteristics, such as life cycle, learning curve, or incremental technical innovation. Few decisions referred to HTA reports as part of the procurement decision; similarly, few HTA reports included a procurement perspective to help guide the procurement bodies. CONCLUSIONS: There is minimal evidence that notes HTA influencing medical device procurement. Procurement bodies and hospitals may not be incentivized to publish their work and transparency could be improved; further research would better describe the link between HTA and procurement. Such research would enable the HTA agencies to meaningfully assess devices to target procurement bodies and allow device sponsors to prioritize evidence. This could limit redundancy, improve evidence, and ultimately promote savings to healthcare systems and expand access.

What Is Value in Health and Healthcare? A Systematic Literature Review of Value Assessment Frameworks.

OBJECTIVES: This study aimed to investigate how value is defined and measured in existing value assessment frameworks (VAFs) in healthcare. METHODS: We searched PubMed, Embase, the Cochrane Library, and Centre for Reviews and Dissemination from 2008 to 2019. We also performed backward citation chaining of included studies and previously published systematic reviews. Studies reporting the development of a VAF in healthcare were included. For each included framework, we extracted and compared the context, target users, intended use, methods used to identify value attributes, description of the attributes, and attribute scoring approaches. RESULTS: Of the 8151 articles screened, 57 VAFs were included. The value attributes included in 55 VAFs were grouped into 9 categories: health benefits (n = 53, 96%), affordability (n = 45, 82%), societal impact (n = 42, 76%), burden of disease (n = 36, 65%), quality of evidence (n = 32, 58%), cost-effectiveness (n = 31, 56%), ethics and equity (n = 27, 49%), unmet needs (n = 21, 38%), and innovation (n = 15, 27%). The remaining 2 VAFs used broad attributes or user-defined attributes. Literature review was the main approach to identify value attributes in 36 VAFs. Patient or public was engaged through the development of only 11 VAFs. Weighting has been used to score 29 VAFs, of which 19 used the methods of multicriteria decision analysis. CONCLUSIONS: There are substantial variations in defining and measuring value. A noticeable weakness of existing VAFs is that patient or public engagement was generally very limited or missing in framework development process. Existing VAFs tend to aggregate multiple value attributes into a single index for decision making.

Value Assessment Frameworks in Latin America: "Il buono, il brutto e il cattivo".

OBJECTIVES: Using a taxonomy previously developed, we assessed the strengths and limitations of available value assessment frameworks (VAF) in Latin America. METHODS: Systematic review of peer-reviewed journals, gray literature review, and surveys to ISPOR Latin America Industry Committee members were done to identify and select current VAF. Once selected, independent reviewers, organized by pairs, assessed each framework's input, methodology, and outputs. RESULTS: We assessed 7 of 9 VAF in the region, excluding 2 that were not currently in use. The review included 1 framework developed by a regional entity, and 6 country frameworks for either price assessment or to inform reimbursement. Most of these frameworks had a clear definition of the purpose (6 of 7) but could provide more details on the conceptual approach, including perspectives, methods for obtaining preferences, and the ability to incorporate multiple value dimensions (2 of 7). Most lacked information about inclusions/exclusions of elements included in the framework, and whether it assumes a base case comparator and how it is selected. The description of the evaluation of data sources and their scientific validity was inconsistently reported (3 of 7). Few included an assessment of the intervention's effect on total costs of treating a defined population (2 of 7), or a description of how uncertainty could be incorporated (3 of 7). Finally, potential conflicts of interest among those creating the framework are not sufficiently addressed (0 of 7). CONCLUSIONS: In the 7 frameworks assessed in Latin America, there are opportunities to improve dimensions, methods, and scope. Addressing these issues will strengthen these VAF for policy and clinical decision making.

A Systematic Review of the Value Assessment Frameworks Used within Health Technology Assessment of Omics Technologies and Their Actual Adoption from HTA Agencies.

BACKGROUND: Omics technologies, enabling the measurements of genes (genomics), mRNA (transcriptomics), proteins (proteomics) and metabolites (metabolomics), are valuable tools for personalized decision-making. We aimed to identify the existing value assessment frameworks used by health technology assessment (HTA) doers for the evaluation of omics technologies through a systematic review. METHODS: PubMed, Scopus, Embase and Web of Science databases were searched to retrieve potential eligible articles published until 31 May 2020 in English. Additionally, through a desk research in HTA agencies' repositories, we retrieved the published reports on the practical use of these frameworks. RESULTS: Twenty-three articles were included in the systematic review. Twenty-two frameworks, which addressed genetic and/or genomic technologies, were described. Most of them derived from the ACCE framework and evaluated the domains of analytical validity, clinical validity and clinical utility. We retrieved forty-five reports, which mainly addressed the commercial transcriptomic prognostics and next generation sequencing, and evaluated clinical effectiveness, economic aspects, and description and technical characteristics. CONCLUSIONS: A value assessment framework for the HTA evaluation of omics technologies is not standardized and accepted, yet. Our work reports that the most evaluated domains are analytical validity, clinical validity and clinical utility and economic aspects.

Assessing the Joint Value of Genomic-Based Diagnostic Tests and Gene Therapies.

Gene therapy is an emerging type of treatment that may aim to provide a cure to individuals with a genetic mutation known to be causative of a specific disease. A diagnosis of the causative mutation must precede treatment with a in vivo gene therapy. Both achieving a genomic-based diagnosis and treatment with a gene therapy may result in substantial expenditures for health care systems. Uncertainties around the health care costs, risks, and benefits derived from diagnosis and treatment with a subsequent gene therapy suggests a need for developing an evidence base, underpinned by opportunity cost, to inform if, and how, these health technologies should be introduced into health care systems funded by finite budgets. This article discusses why current methods to evaluate health technologies (decision-analytic model-based cost-effectiveness analysis from the perspective of a health care system over a lifetime time horizon) are appropriate to quantify the costs and consequences of using genomic-based diagnostic tests and gene therapies in combination, rather than as separate interventions, within clinical practice. Evaluating the economic impact of test-and-treatment strategies will ensure that the opportunity cost of these health technologies is quantified fully for decision-makers who are responsible for allocating limited resources in health care systems.

Using Nominal Group Technique to Identify Key Attributes of Oncology Treatments for a Discrete Choice Experiment.

Background. Responding to rising oncology therapy costs, multiple value frameworks are emerging. However, input from economists in their design and conceptualization has been limited, and no existing framework has been developed using preference weightings as legitimate indicators of value. This article outlines use of the nominal group technique to identify valued treatment attributes (such as treatment inconvenience) and contextual considerations (such as current life expectancy) to inform the design of a discrete choice experiment to develop a preference weighted value framework for future decision makers. Methods. Three focus groups were conducted in 2017 with cancer patients, oncology physicians, and nurses. Using the nominal group technique, participants identified and prioritized cancer therapy treatment and delivery attributes as well as contextual issues considered when choosing treatment options. Results. Focus groups with patients (n = 8), physicians (n = 6), and nurses (n = 10) identified 30 treatment attributes and contextual considerations. Therapy health gains was the first priority across all groups. Treatment burden/inconvenience to patients and their families and quality of evidence were prioritized treatment attributes alongside preferences for resource use and cost (to patients and society) attributes. The groups also demonstrated that contextual considerations when choosing treatment varied across the stakeholders. Patients prioritized existence of alternative treatments and oncologist/center reputation while nurses focused on administration harms, communication, and treatment innovation. The physicians did not prioritize any contextual issues in their top rankings. Conclusions. The study demonstrates that beyond health gains, there are treatment attributes and contextual considerations that are highly prioritized across stakeholder groups. These represent important candidates for inclusion in a discrete choice experiment seeking to provide weighted preferences for a value framework for oncology treatment that goes beyond health outcomes.

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.