Determining priority indicators of utility for genomic testing in rare disease: A Delphi study.

PURPOSE: Determining the value of genomic tests in rare disease necessitates a broader conceptualization of genomic utility beyond diagnostic yield. Despite widespread discussion, consensus toward which aspects of value to consider is lacking. This study aimed to use expert opinion to identify and refine priority indicators of utility in rare disease genomic testing. METHODS: We used 2 survey rounds following Delphi methodology to obtain consensus on indicators of utility among experts involved in policy, clinical, research, and consumer advocacy leadership in Australia. We analyzed quantitative and qualitative data to identify, define, and determine priority indicators. RESULTS: Twenty-five experts completed round 1 and 18 completed both rounds. Twenty indicators reached consensus as a priority in value assessment, including those relating to prognostic information, timeliness of results, practical and health care outcomes, clinical accreditation, and diagnostic yield. Whereas indicators pertaining to discovery research, disutility, and factors secondary to primary reason for testing were considered less of a priority and were removed. CONCLUSION: This study obtained expert consensus on different utility indicators that are considered a priority in determining the value of genomic testing in rare disease in Australia. Indicators may inform a standardized approach to evidence generation and assessment to guide future research, decision making, and implementation efforts.

Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi-criteria decision analysis.

INTRODUCTION: The value of gene therapies for haemophilia needs to be assessed holistically. AIM: To determine the value of etranacogene dezaparvovec (ED) compared to current extended half-life (EHL) recombinant factors (rFIX), using multi-criteria decision analysis (MCDA). METHOD: MCDA EVIDEM methodology adapted to orphan drugs was used, with nine quantitative criteria and four contextual criteria. The MCDA framework was rated by 28 multidisciplinary experts. Descriptive statistics were performed for quantitative and qualitative criteria. RESULTS: Haemophilia B (HB) was considered a severe disease (mean ± SD: 4.3 ± 0.7) with some unmet needs (mean ± SD 3.3 ± 0.9). Experts found ED more effective (mean ± SD 2.0 ± 2.3) and provide better quality of life (QoL) (mean ± SD: 1.8 ± 1.5) than the comparative HB treatments but with safety uncertainties (mean ± SD -1.2 ± 1.8). ED could lead to medical cost and non-medical cost savings over time (mean ± SD: 1.6 ± 2.0 and 2.0 ± 1.5, respectively). The quality of the evidence was high (mean ± SD: 3.9 ± 0.9). ED was considered aligned with the priorities of the National Health System (NHS) and the specific interests of patients. ED's value contribution was 0.45 (+1 = highest value). CONCLUSIONS: ED brings added value in the treatment of moderately severe and severe HB (sHB) compared to current EHL rFIX, addressing the severity of the disease and increasing efficacy and patients' QoL especially related to the single dose and low bleeding rate. Concerns about long-term safety need to be addressed.

Scoring the Value Assessment Framework for China: A Factorial Survey.

OBJECTIVES: This study aimed to develop the scoring functions for the recently developed value assessment framework (VAF) for China, which comprises 12 attributes. METHODS: We implemented a factorial survey among Chinese healthcare stakeholders from July to September 2022. A total of 240 hypothetical drug value profiles described by the VAF were grouped into 60 blocks and randomly assigned to respondents. Each respondent was assigned with 1 block, each presented in 3 disease scenarios of different levels of severity. For each profile, respondents were asked to assess the drug's value on a scale from 0 (lowest) to 10 (highest) and make 1 of the 3 insurance recommendations: cover, to be negotiated for coverage, or reject. Linear and logistic mixed-effects models were used to develop scoring functions for aggregating the value attributes. RESULTS: A total of 365 respondents participated in the survey. 3968 responses from 331 respondents were included in the analysis. Most of the included respondents were under 45 (n = 256, 77.3%), females (n = 208, 62.8%), living in urban areas (n = 296, 89.4%), and with a bachelor's degree or higher (n = 303, 91.5%). Health benefits and safety carried more weights than other attributes in the scoring functions across disease scenarios. The value and probability of entering negotiation or receiving insurance coverage for the attribute profiles for severe/critical disease were higher than for mild/moderate disease. CONCLUSIONS: The scoring functions of the VAF can be used to assess the value of a drug and its probability of entering negotiation or receiving insurance coverage in China.

Multistakeholder Perceptions of Additional Value Elements for United States Value Assessment of Health Interventions.

OBJECTIVES: Limitations in conventional cost-effectiveness methods have led to calls for incorporation of additional value elements in assessments of health technologies. However, gaps remain in how additional value elements may inform decision making. This study aimed to prioritize additional value elements from the perspective of healthy individuals without a specific condition or indicated for a specific treatment in the United States among a multistakeholder panel and compare the importance of perspective-specific value elements. METHODS: Additional value elements were prioritized in 2 phases: (1) we identified and categorized additional value elements in a targeted literature review, and (2) we convened a multistakeholder group-based preference elicitation study (N = 28) to evaluate the description of each value element and rank and generate normalized weights of each value element for its significance in value assessment. The importance of additional value elements was also weighted relative to patient-centric value elements. RESULTS: The rank and weight of contextual value elements among 28 stakeholders were "severity of the disease" (26.2%), "disadvantaged and vulnerable target populations highly represented" (21.8%), "broader economic impact" (17.3%), "risk protection" (13.8%), "rarity of the disease" (11.3%), and "novel mechanism of action" (9.7%). Relative weight of the additional value elements versus patient-centric value elements was 52% and 48%, respectively. CONCLUSIONS: Study findings may inform priority setting for value frameworks and emerging US government assessments. The group-based elicitation method is repeatable and useful for structured deliberative processes in value assessment and may help improve the consistency and predictability of what is important to stakeholders.

Documenting the Full Value of Vaccination: A Systematic Review of Value Frameworks.

OBJECTIVES: Economic evaluations of vaccination may not fully account for nonhealth patient impacts on families, communities, and society (ie, broader value elements). Omission of broader value elements may reflect a lack of established measurement methodology, lack of agreement over which value elements to include in economic evaluations, and a lack of consensus on whether the value elements included should vary by vaccination type or condition. We conducted a systematic review of value frameworks to identify broader value elements and measurement guidance that may be useful for capturing the full value of vaccination. METHODS: We searched Ovid MEDLINE, PubMed, Embase, and the gray literature to identify value frameworks for all health interventions, and we extracted information on each framework's context, value elements, and any available guidance on how these elements should be measured. We used descriptive statistics to analyze and compare the prevalence of broader value elements in vaccination value frameworks and other healthcare-related value frameworks. RESULTS: Our search identified 62 value frameworks that met inclusion criteria, 9 of which were vaccination specific. Although vaccination frameworks included several broader value elements, such as reduced transmissibility and public health benefits, the elements were represented inconsistently across the frameworks. Vaccination frameworks omitted several value elements included in nonvaccination-specific frameworks, including dosing and administration complexity and affordability. In addition, guidance for measuring broader value elements was underdeveloped. CONCLUSIONS: Future efforts should further evaluate inclusion of broader value elements in economic evaluations of vaccination and develop standards for their subsequent measurement.

Mapping Health Technology Assessment Agency Approaches for Biosimilar Value Assessment: An ISPOR Special Interest Group Report.

OBJECTIVES: A systematic literature review undertaken by the ISPOR Biosimilar Special Interest Group highlighted that limited guidance exists on how to assess biosimilars value and on appropriate economic evaluation techniques. This study described current health technology assessment (HTA) agency approaches for biosimilar value assessment. METHODS: Semi-structured interviews (n = 16) were carried out with HTA experts in Africa, America, Asia, Australia, and Europe to investigate current HTA practices for biosimilars. Data categorization was based on a thematic analysis approach. Findings from the qualitative data analysis were interpreted in view of relevant published literature. RESULTS: Our research suggests that in systems in which frameworks for biosimilar regulatory approval are well established, HTA agencies can accept the regulators' comparability exercise, and reimbursement decisions can generally be based on price comparisons. This approach is accepted in practice and allows streamlining of biosimilars value assessment. Nevertheless, conducting HTAs for biosimilars can be relevant when (1) the originator is not reimbursed, (2) the biosimilar marketing authorization holder seeks reimbursement for indications/populations, pharmaceutical forms, methods and routes of administration that differ with respect to the originator, and (3) a price premium is sought for a biosimilar based on an added-value claim. Further, HTA agencies' role conducting class-review updates following biosimilar availability can support greater patients' access to biologics. CONCLUSIONS: Internationally, there are differences in how national competent authorities on pricing and reimbursement of pharmaceuticals perceive HTA's role for biosimilars. Therefore, HTA agencies are encouraged to issue clear guidance on when and how to conduct HTAs for biosimilars, and on which economic techniques to apply.

Systematic Review of Outcomes for Assessment of Medication Adherence Enhancing Interventions: An ISPOR Special Interest Group Report.

OBJECTIVES: The lack of universal guidance on outcome measures for evaluating medication adherence enhancing interventions (MAEIs) poses a challenge for assessing their effectiveness. This literature review aimed to provide a systematic overview of outcome measures currently used for the value assessment of MAEIs. METHODS: We followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines and searched MEDLINE, PsycINFO, Scopus, CINAHL, and Academic Search Complete for randomized and nonrandomized clinical trials, prospective cohort studies, model-based economic evaluations, and value frameworks published in English between January 2010 and September 2020. Two independent reviewers screened all titles and abstracts, followed by a full-text review. Due to the large number of relevant studies, data extraction was limited to articles published between January 2018 and September 2020. We collected data on the general characteristics of the study, the type of intervention, and the outcomes measured. RESULTS: We screened 14 685 records and identified 308 articles for data extraction. Behavioral interventions were the most common (n = 143), followed by educational interventions (n = 110) and mixed-method interventions (n = 73). Outcomes were clustered into 7 categories with medication adherence (n = 286) being the most frequently measured, followed by clinical outcomes (n = 155), health-related quality of life (n = 57), resource use (n = 43), patient satisfaction (n = 31), economic outcomes (n = 18), and other outcomes (n = 76). CONCLUSIONS: Various outcomes measures have been used to evaluate MAEIs, with only a small number of studies exploring economic and patient-reported outcomes. Future research is warranted to develop a consensus-based set of criteria for assessing MAEIs to facilitate the comparison of interventions and enable informed decision making.

Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making.

OBJECTIVES: A health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multicriteria decision analysis approach. METHODS: We constructed a multicriteria-decision-analysis-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (N = 23), we refined previously generated criteria and developed rank-based weights. We established a criterion-pertinent scoring rubric for assessing incremental benefits of new drugs. Three clinicians independently assessed comprehension by pilotscoring 9 drugs. We then validated CATCH for 2 childhood cancer therapies through structured deliberation with an expert panel (N = 10), obtaining individual scores, consensus scores, and verbal feedback. Analyses included descriptive statistics, thematic analysis, exploratory disagreement indices, and sensitivity analysis. RESULTS: The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median disagreement indices = 0.34): Effectiveness, Child-specific Health-related Quality of Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, and Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panelists endorsed the framework's key modifiers of value. Modes of their individual prescores aligned closely with deliberative consensus scores. CONCLUSIONS: We iteratively developed a value assessment framework that captures dimensions of child-specific health and nonhealth gains. CATCH could improve the richness and relevance of HTA decision making for children in Canada and comparable health systems.

Patient-Informed Value Elements in Cost-Effectiveness Analyses of Major Depressive Disorder Treatment: A Literature Review and Synthesis.

OBJECTIVES: Prior work identified 6 key value elements (attributes of treatment and desired outcomes) for individuals living with major depressive disorder (MDD) in managing their condition: mode of treatment, time to treatment helpfulness, MDD relief, quality of work, interaction with others, and affordability. The objective of our study was to identify whether previous cost-effectiveness analyses (CEAs) for MDD treatment addressed any of these value elements. A secondary objective was to identify whether any study engaged patients, family members, and caregivers in the model development process. METHODS: We conducted a systematic literature review to identify published model-based CEAs. We compared the elements of the published studies with the MDD patient value elements elicited in prior work to identify gaps and areas for future research. RESULTS: Of 86 published CEAs, we found that 7 included patient out-of-pocket costs, and 32 included measures of productivity, which were both priorities for individuals with MDD. We found that only 2 studies elicited measures from patients for their model, and 2 studies engaged patients in the modeling process. CONCLUSIONS: Published CEA models for MDD treatment do not regularly include value elements that are a priority for this patient population nor do they include patients in their modeling process. Flexible models that can accommodate elements consistent with patient experience are needed, and a multistakeholder engagement approach would help accomplish this.

Reflections on co-creating a model for the value assessment of artificial intelligence technologies.

AIMS: A multidisciplinary group of experts and patients developed the Model for ASsessing the value of Artificial Intelligence (MAS-AI) to ensure an evidence-based and patient-centered approach to introducing artificial intelligence technologies in healthcare. In this article, we share our experiences with meaningfully involving a patient in co-creating a research project concerning complex and technically advanced topics. METHODS: The co-creation was evaluated by means of initial reflections from the research team before the project started, in a continuous logbook, and through semi-structured interviews with patients and two researchers before and after the active co-creation phase of the project. RESULTS: There were initial doubts about the feasibility of including patients in this type of project. Co-creation ensured relevance to patients, a holistic research approach and the debate of ethical considerations. Due to one patient dropping out, it is important to foresee and support the experienced challenges of time and energy spent by the patient in future projects. Having a multidisciplinary team helped the collaboration. A mutual reflective evaluation provided insights into the process which we would otherwise have missed. CONCLUSIONS: We found it possible to create complex and data-intense research projects with patients. Including patients benefitted the project and gave researchers new perspectives on their own research. Mutual reflection throughout the project is key to maximise learning for all parties involved.

A Systematic Literature Review of Gaps and Challenges in Value Assessment of Biosimilars: An ISPOR Special Interest Group Report.

OBJECTIVES: This study aims to provide an overview of the gaps and challenges in the value assessment of biosimilars and to identify potential approaches to address them. METHODS: A multidisciplinary, international team of biosimilar experts identified gaps and challenges. A systematic review was conducted of the peer-reviewed literature in PubMed, EMBASE, Web of Science Core Collection, EBSCOhost Business Source Complete; and of the gray literature. Preliminary results were presented at ISPOR conferences and this article benefited from 2 review rounds among ISPOR Biosimilar Special Interest Group members. RESULTS: Given that a biosimilar is highly similar to its reference biologic, health technology assessment agencies should accept the comparability exercise approved by regulatory authorities and, thus, conduct a price comparison when biosimilar reimbursement is requested for the same indication as the reference biologic. If the reference biologic is not reimbursed or is not the standard of care, a full economic evaluation of the biosimilar versus a relevant comparator needs to be conducted. To date, little consideration has been given to specific challenges, such as how biosimilar value assessment can account for the nocebo effect, potential differences between biologic-naive and biologic-experienced patients, the availability of intravenous and subcutaneous administration forms or different administration devices for the same active compound, value-added services, and the contribution of biosimilars for generating health gain at the population level. CONCLUSIONS: There is a need to gather further insights in the methodology of value assessment for biosimilars, and health technology assessment agencies need to develop more elaborate guidance on biosimilar value assessment in specific circumstances.

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.

Longitudinal analysis of shoulder arthroplasty utilization, clinical outcomes, and value: a comparative assessment of changes in improvement over 15 years with a single platform shoulder prosthesis.

BACKGROUND: The goal of this longitudinal analysis of anatomic total shoulder arthroplasty (aTSA) and reverse total shoulder arthroplasty (rTSA) utilization from 2007 to 2021 is to quantify changes in clinical outcomes, cost, and value, resulting from the introduction and adoption of new shoulder arthroplasty (SA) technologies. METHODS: We analyzed an international database of a single SA prosthesis (Equinoxe; Exactech, Inc.; Gainesville, FL, USA) for all clinical sites that have continuously enrolled cases from 2007 to 2021 to compare changes in primary aTSA and primary rTSA utilization and outcomes across 3, 5-year cohorts based upon the date of implantation. A value analysis was conducted across the 5-year implantation cohorts, with value measured by the ratio of each postoperative outcome measure at 24-36 months and 36-60 months after surgery, and the average implant selling price each year for the U.S. sites in constant 2007 U.S. dollars. These measures of value were compared between cohorts to quantify the impact of new technology introduced over the study period. RESULTS: A dramatic increase in rTSA utilization was observed across the 6 sites over the 15-year study period, along with a rapid adoption of new aTSA and rTSA technologies. The average patient receiving primary aTSA and primary rTSA changed over the 15-year study period, with significant shifts in diagnosis, comorbidities, and preoperative functional status. A comparison of postoperative results demonstrated that both aTSA and rTSA clinical and radiographic outcomes showed improvement relative to 2007-2011. Over this 15-year study period, the average aTSA implant selling price has been relatively stable while the average rTSA implant selling price has significantly declined. As a result, the value associated with the Equinoxe rTSA significantly increased for nearly every outcome measure at 24-36 months and 36-60 months after surgery, while value associated with the Equinoxe aTSA stayed relatively constant from 2007 to 2021. CONCLUSION: Our 6042-patient longitudinal analysis quantified numerous changes in utilization, outcomes, and value across 6 clinical sites over the 15-year study period. Rapid adoption of new aTSA and rTSA technologies was observed and clinical and radiographic outcomes improved relative to 2007-2011. These clinical improvements, in combination with steady aTSA and declining rTSA implant prices, have driven rTSA value to continuously increase while aTSA value has been maintained at a high-level over the 15-year study period with this particular SA system, even when considering the cost and adoption of new technologies.

Toward comprehensive value assessment for Alzheimer's disease innovations.

INTRODUCTION: Assessing medical technologies for Alzheimer's disease (AD) creates challenges for current methods of value assessment. New value assessment approaches for AD are also needed. METHODS: We adapted concepts from health economics to help guide decision makers to more informed decisions about AD therapies and diagnostics. RESULTS: We propose a value framework based on five categories: perspective, value elements, analysis, reporting, and decision making. AD value assessments should include the perspective of the patient-caregiver dyad. We propose a broader array of value elements than currently used. Analytics and decision methods can synthesize evidence for all elements of value. Decisions should use a "deliberative appraisal" approach informed by the composite evidence and be transparently reported. DISCUSSION: Using the proposed framework, the value of forthcoming innovations for AD may be more thoroughly assessed for and by all stakeholders. It can guide decision makers to carefully consider all relevant elements of value contributing to more holistic and transparent decision making. RESEARCH HIGHLIGHTS: Alzheimer's disease challenges common methods of evaluating medical technology. Using current methods, new AD innovations might not be appropriately valued. Poor value assessments will adversely affect patient access to AD innovations. A full AD value framework expands perspective, elements, analysis, decision-making, reporting.

[Expert consensus on core indicators for lifecycle value assessment of Chinese patent medicine].

The establishment of core indicators for assessment plays an important role in carrying out the lifecycle value assessment of Chinese patent medicine, which are developed based on the concepts such as clinical value oriented, paying attention to the human use experience, and whole process quality control. To this end, the Specialty Committee of Data Monitoring and Decision Making of the World Federation of Chinese Medicine Societies organized experts to draft the Expert Consensus on Core Indicators for Lifecycle Value Assessment of Chinese Patent Medicine based on the research including Chinese Medicine Registration Review Evidence System in Combination of Traditional Chinese Medicine Theory, Human Use Experience, and Clinical Trials(GZY-FJS-2022-206) by National Administration of Traditional Chinese Medicine. This consensus proposed 92 core indicators from four stages, including new drug R&D project approval, pre-clinical research, new drug marketing authorization, and post-marketing, combining the assessment purposes and needs of different stakeholders from different dimensions such as clinical needs, clinical positioning, human use experience, effectiveness, safety, quality control, innovation, accessibility, and suitability. This consensus also interpreted the indicators to clearly elucidate the core elements of the value assessment of Chinese patent medicine in different R&D stages and guided the stakeholders to identify, analyze, and assess the value of Chinese patent medicine in the R&D and use process based on the core indicators in a scientific, objective, and standardized approach. This consensus is expected to play an important role in the high-quality new drug development, drug pricing and compensation of Chinese patent medicine, the development of clinical pathways, and rational clinical application.

The Cost-Effectiveness of Remdesivir for Hospitalized Patients With COVID-19.

OBJECTIVES: This study aimed to estimate the cost-effectiveness of remdesivir, the first novel therapeutic to receive Emergency Use Authorization for the treatment of hospitalized patients with COVID-19, and identify key drivers of value to guide future pricing and reimbursement efforts. METHODS: A Markov model evaluated the cost-effectiveness of remdesivir in patients hospitalized with COVID-19 from a US healthcare sector perspective. A lifetime time horizon captured potential long-term costs and outcomes. Model outcomes included discounted total costs, life-years, and quality-adjusted life-years (QALYs). Remdesivir was modeled as an addition to standard of care and compared with standard of care alone, including dexamethasone for patients requiring respiratory support. COVID-19 hospitalizations were assumed to be reimbursed through a single payment based on the respiratory support received alongside a remdesivir carveout payment in the base case. Sensitivity and scenario analyses identified key drivers. RESULTS: At a unit price of $520 per vial and assuming no survival benefit with remdesivir, the incremental cost-effectiveness was $298 200/QALY for patients with moderate to severe COVID-19 and $1 847 000/QALY for patients with mild COVID-19. Although current data do not support a survival benefit, if one was assumed, the cost-effectiveness estimate was $50 100/QALY for the moderate to severe population and $103 400/QALY for the mild population. Another key driver included the hospitalization payment structure (per diem vs bundled payment). CONCLUSIONS: With the current evidence available, remdesivir's price is too high to align with its expected health gains for hospitalized patients with COVID-19. Results from this study provide a rationale for iterative health technology assessment.

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.

Should We Consider Including a Value for "Hope" as an Additional Benefit Within Health Technology Assessment?

Health technology assessment (HTA) typically uses average health-related quality of life gain as its main measure of benefit used in economic evaluation. Nevertheless, there have been calls to consider novel aspects of benefit including the "value of hope," defined as a patients' potential preferences for a wider distribution of treatment benefit with a positive skew, in the hope that they will be one of the lucky ones. The value of hope may also derive from feeling hopeful as a positive mental state, which may be missing from current measures of health-related quality of life. The value attributed to feeling hopeful could be related to, or additional to, the value derived from possible risk-seeking preferences. Here, we reflect upon the strength of the case for the inclusion of the "value of hope" taking a critical look at the commonly referenced evidence for including the "value of hope" as risk-seeking preferences. We also draw attention to other conceptions of hope-as an emotion, a cognitive process, or a combination of both-and reflect upon the potential of including these broader notions of hope into HTA. The case for the inclusion of the "value of hope" based on risk-seeking preferences is weak. We suggest research questions that could give further evidence on whether hope is an important missing value from HTA.

The History and Future of the "ISPOR Value Flower": Addressing Limitations of Conventional Cost-Effectiveness Analysis.

OBJECTIVES: Since its publication as part of the 2018 ISPOR Special Task Force (STF) on US Value Assessments, the "ISPOR value flower," with its petals highlighting elements that may be overlooked or underappreciated in conventional drug value assessments, has been discussed and debated. We review the history of the value flower, describe recent developments, and consider implications for future value assessments. METHODS: We discuss various antecedents to the value flower, as well as conceptual and empirical articles published in the past 4 years. RESULTS: Since the publication of the ISPOR STF report, researchers have provided more rigorous theoretical and mathematical foundations for certain novel value elements (eg, severity of illness, value of insurance, value of hope) through "generalized risk-adjusted cost-effectiveness analysis," which incorporates risk aversion in people's preferences and uncertainty in treatment outcomes. Empirical estimates are also emerging to support key elements, such as insurance value, real option value, value of hope, and value of knowing. Although health technology assessment bodies have applied or are considering certain elements (eg, severity modifiers to cost-effectiveness thresholds), other elements have yet to gain traction. CONCLUSIONS: Five years after the STF began its work, the development of novel value measures continues to evolve. Although it is encouraging to see supporting empirical studies emerging, more are needed. Additional efforts are also needed to illustrate how the estimates can be used in the deliberative processes that are integral to health technology assessments.

Value assessment of artificial intelligence in medical imaging: a scoping review.

BACKGROUND: Artificial intelligence (AI) is seen as one of the major disrupting forces in the future healthcare system. However, the assessment of the value of these new technologies is still unclear, and no agreed international health technology assessment-based guideline exists. This study provides an overview of the available literature in the value assessment of AI in the field of medical imaging. METHODS: We performed a systematic scoping review of published studies between January 2016 and September 2020 using 10 databases (Medline, Scopus, ProQuest, Google Scholar, and six related databases of grey literature). Information about the context (country, clinical area, and type of study) and mentioned domains with specific outcomes and items were extracted. An existing domain classification, from a European assessment framework, was used as a point of departure, and extracted data were grouped into domains and content analysis of data was performed covering predetermined themes. RESULTS: Seventy-nine studies were included out of 5890 identified articles. An additional seven studies were identified by searching reference lists, and the analysis was performed on 86 included studies. Eleven domains were identified: (1) health problem and current use of technology, (2) technology aspects, (3) safety assessment, (4) clinical effectiveness, (5) economics, (6) ethical analysis, (7) organisational aspects, (8) patients and social aspects, (9) legal aspects, (10) development of AI algorithm, performance metrics and validation, and (11) other aspects. The frequency of mentioning a domain varied from 20 to 78% within the included papers. Only 15/86 studies were actual assessments of AI technologies. The majority of data were statements from reviews or papers voicing future needs or challenges of AI research, i.e. not actual outcomes of evaluations. CONCLUSIONS: This review regarding value assessment of AI in medical imaging yielded 86 studies including 11 identified domains. The domain classification based on European assessment framework proved useful and current analysis added one new domain. Included studies had a broad range of essential domains about addressing AI technologies highlighting the importance of domains related to legal and ethical aspects.