Alternative frameworks

To address the limitations of traditional assessments of value using CEA, alternative value assessment frameworks have been developed to help decision makers account for the “reasonableness” of their decisions. Organizations such as the European Society of Medical Oncology have developed modified assessment frameworks to determine the value of new pharmaceuticals, enabling a patient-centered approach with shared decision making; however, these disease-specific frameworks are not consistent with population-level decision making (Reference Campolina20). The updated value framework of the Institute for Clinical and Economic Review reflects various “contextual considerations” and “other benefits and disadvantages” of therapies, including disease severity, lack of alternatives, novel mechanism of action, and health disparities. While this framework is designed to address pricing and insurance coverage issues in a market-based health system, it does consider specific differences in evidence standards and unique aspects to the development, early evaluation, regulatory approval, uptake, and patterns of all health technologies, including pharmaceuticals, devices, digital health technologies, diagnostics, and delivery systems (21).

Multiple criteria decision analyses (MCDAs) have also been proposed as a means of overcoming the limitations of CEAs by applying explicit criteria that address localized needs and priorities. Scores or weightings associated with these criteria are ranked or rated, with multiple factors combined into a single value that, through a deliberative, collaborative process, can be used to reach consensus and support healthcare decision making (Reference Howard, Scott, Ju, McQueen and Scuffham22). However, the lack of a validated MCDA framework represents a significant challenge, especially around uncertainties in the weighting and scoring of the criteria used and the potential for a lack of consensus among participants (Reference Wahlster, Goetghebeur, Kriza, Niederländer and Kolominsky-Rabas23). The role of patient and public involvement methods, such as citizens juries, in deciding which criteria should be included in an MCDA, especially technologies for rare disease or an unmet clinical need, should be explored; however, this may lead to (further) delays in accessing new technologies.

Decision-making bodies such as the Scottish Medicines Consortium and the National Institute for Health and Care Excellence introduced the use of modifiers as an alternative to the consideration of the results of traditional CEAs. Modifiers are designed to accommodate the approval and funding of technologies that may result in substantial changes in life expectancy or quality of life (QoL) by considering additional factors. This allows for greater flexibility in decision making, especially around high-cost drugs or technologies that treat severe, rare, or debilitating disease, end-of-life care, or those targeting pediatric populations. The use of modifiers may allow decision makers to accept more uncertainty in economic analyses where there is limited efficacy data available, or a higher cost per QALY (Reference Neumann, Garrison and Willke13;Reference Charlton24–26). The use of modifiers at the appraisal stage has, however, been criticized as a means of funding health technologies that benefit the few at a cost to the many, resulting in fewer technologies being approved at an “opportunity cost” to other patients (Reference Oliver and Mott27).

One of the most widely discussed alternative evaluation frameworks is the Professional Society for Health Economics and Outcomes Research’s (ISPOR) “value flower” model, designed to augment rather than replace CEAs (Figure 1) (Reference Lakdawalla, Doshi, Garrison, Phelps, Basu and Danzon28). Value is at the hub of the model, with spokes going out to 12 different elements, with QALYs retained as a core element along with cost offsets or net costs. By considering additional value elements, the value flower aims to better capture and evaluate the benefits and costs of new health technologies from a societal perspective, many of which relate to uncertainty such as equity, severity of disease, scientific spillovers, and value of hope, noting that quantifying and measuring these elements is complex due to their subjective nature (Reference Neumann, Garrison and Willke13;Reference Lakdawalla, Doshi, Garrison, Phelps, Basu and Danzon28). Productivity (ability to work or perform daily activities despite treatment or disease) and adherence-improving factors (adherence to treatment can significantly impact outcomes and costs) are also included despite inconsistent use (Reference Goring, Garrison, Jansen and Briggs29;Reference Garrison, Hartgers-Gubbels and Chambers30).

Figure 1.

The ISPOR value flower, 2018 (Reference Neumann, Garrison and Willke13;Reference Garrison, Hartgers-Gubbels and Chambers30).

Figure 1 Long description

One of the most widely discussed alternative evaluation frameworks is the Professional Society for Health Economics and Outcomes Research’s (ISPOR) “value flower” model, designed to augment rather than replace CEAs. Value is at the hub of the model, with spokes going out to twelve different elements (clockwise): QALYs and net costs are core elements, productivity, adherence-improving factors, reduction in uncertainty, fear of contagion, insurance value, severity of disease, value of hope, real-option value, equity, scientific spillovers.

The BRAVER Roadmap (Reference Theakston, Hofer, Garau and Steuten31), which examined how broader value elements could be integrated into HTA frameworks, exploring the opportunities and barriers to incorporating the societal perspective into HTA in the Asia region, provided the foundation for this year’s APF. It is hoped that by adopting a societal perspective, decision makers can better address both clinical and societal needs, particularly in countries where reliance on informal caregiving is increasing due to rapidly aging populations and an increasing burden of chronic disease, resulting in wider social care expenditure. From an industry point of view, consideration of wider health sector benefits in HTA may help drive and reward innovation with the development of novel therapies by scientific “spillover.” Many of the elements proposed to be included in HTA by the BRAVER study overlap with those proposed by the ISPOR value flower; however, several elements are unique, including the impact on economic activity (economic growth) and on other government sectors (e.g., education), patient indirect costs and consumption, as well as health-related QoL and indirect costs for caregivers. Productivity is considered from both the patient and the informal caregiver’s perspective (Reference Theakston, Hofer, Garau and Steuten31).

As such, discussions at the 2025 APF centered around whether it is time to think beyond traditional economic evaluations using QALYs and consider implementing a societal perspective approach, including all costs and benefits of a health intervention, regardless of who experiences them. This approach would encompass not only direct medical costs to the healthcare system but also indirect costs and benefits, including patient and caregiver time, productivity losses or gains, and broader social and economic impacts (Reference Sittimart, Rattanavipapong and Mirelman16;Reference Drost, van der Putten, Ruwaard, Evers and Paulus32). The key question for HTA agencies and health systems to address is whether the inclusion of societal elements, such as the impact on caregivers, is relevant to the decision-making process and should it be incorporated in HTA, and if so, how and where?

Pre-meeting surveys and breakout group discussions

Nine HTA agencies from eight countries and seven industry participants responded to a pre-meeting survey designed to gauge delegates’ understanding of the societal perspective as well as its implementation in the Asia region. In addition, during the first session of the APF, delegates were asked to provide three words that described what the societal perspective meant to them, with the resulting word cloud showing that “Equity” was at the heart of many responses, followed by “Inclusive” and “Productivity” (Figure 2). Both the survey results and the word cloud served as a springboard for breakout group discussions, which comprised a mix of industry and agency delegates discussing the topics described in Figure 3.

Figure 2.

Word cloud describing the societal perspective.

Figure 2 Long description

APF delegates were asked to provide three words that described what the societal perspective meant to them, with the resulting word cloud showing that “Equity” was at the heart of many responses, followed by “Inclusive” and “Productivity”. Other min responses included Value, Fairness, inclusivity, holistic, family, patient centric, non-health and cost.

Figure 3.

Themes for the breakout group discussions.

Figure 3 Long description

Breakout Group 1 discussed Whose values should a framework reflect? Value to the payer or value to the public? Breakout Group 2 discussed Is there a role for citizen juries when deciding what elements should be included in an HTA? Group 3 discussed Should the societal perspective be considered for alltechnologies or only high-cost ones such as cellular or genetherapies, or immunotherapies? Group 4 discussed Does fairness matter? Should the societal perspective beconsidered equally for all patient populations or only rare orsevere disease? Group 5 discussed The lack of appropriate patient input and the lack of data wereidentified as major issues by industry and agencies. How do we solve these problems? Group 6 discussed What is the potential impact (positive or negative) of broadeningvalue criteria not only on health budget but on the overallbudget, and patient access.

Most survey respondents from agencies and industry were either very or moderately familiar with the concept of the societal perspective (89 and 86 percent, respectively) and felt that its inclusion was moderately or very important (89 and 100 percent, respectively). To this end, Group-1 discussed the perspective an evaluation framework should reflect. The primary focus of both well-developed (e.g., the Philippines and Japan) and developing HTA systems (e.g., India and Cambodia) was evaluations conducted from the payer’s perspective, with recommendations mainly based on CEA. However, countries with well-developed HTA systems include additional qualitative data to support these analyses, especially when considering rare diseases or population-wide programs such as vaccination. Only Thailand described the development of HTA guidelines to incorporate the societal perspective, noting the limitations and difficulties around data collection, especially direct and indirect medical costs. One of the key recommendations from Group-1 was to involve key stakeholders early, seeking endorsement when developing value frameworks.

There was greater variation between agency and industry responses when asked which elements should be considered in an economic evaluation conducted from the societal perspective. Most agencies favored the inclusion of severity of disease, impact on family or caregivers, productivity and affordability, and benefits to society, closely followed by equity and disparity in health outcomes. All industry respondents wanted the long-term societal impact and the impact on family or caregivers to be considered, followed by productivity and affordability, and benefits to society (86 and 71 percent, respectively). Interestingly, only one industry respondent identified scientific spillover as an element that should be considered.

Citizen juries have been suggested as a means of providing an informed public or patient perspective on how the value of new technologies should be determined to guide funding decisions, with care taken to recruit a representative panel with adequate levels of health literacy to ensure the results are generalizable to the greater population (Reference Haji Ali Afzali, Street, Merlin and Karnon33). There was some discussion by Breakout Group-2 as to whether citizen juries should comprise patients who would benefit from the intervention in question, or the general public, and it was agreed that both would provide a valuable perspective. Group-2 agreed that citizen juries should be considered crucial to ensure patient-centered policy, providing valuable insights into certain societal perspective elements due to their lived experience; however, their involvement should only be part of the process. Despite a willingness to consider citizen juries, serious challenges were raised, particularly regarding the capability and capacity of HTA agencies to implement this approach and the level of participants’ health literacy.

When the survey asked respondents to identify the greatest challenges to incorporating the societal perspective into HTA evaluations, all agencies cited the limited availability of appropriate data, especially data estimating the short- and long-term costs associated with many of the societal perspective elements and patient-reported outcomes. Finding ways for consistent patient involvement in HTA would better inform the inclusion of broader outcomes and social values in HTA, providing unique insights into the potential benefits or disadvantages of a health technology (Reference Kinchin, Walshe and Normand34). Breakout Group-5 discussed this issue and potential solutions. The availability and quality of data were identified as a major issue, with data often being fragmented, held by different institutions, and lacking linkage. A short Industry poster session held during the APF demonstrated that published data are available, suggesting that perhaps the greatest issue is not so much the lack of data but the inability to access appropriate data.

Further issues identified by Group-5 included the exclusion of broader societal benefits from the base case of most health systems, together with payers’ uncertainty about how to elicit and use patient data in assessments, representing a major systemic barrier. Patient engagement is inconsistent, with patients often having limited awareness of opportunities, a lack of understanding of the process or health system, and often lacking the capability or empowerment to engage. Patient recruitment for input can be challenging, with concerns around data privacy and ownership. Potential solutions suggested by the group include adopting a life cycle approach to evidence generation, starting early with clear research questions that will generate appropriate evidence. Multi-stakeholder engagement should be fostered, as well as building capabilities and capacity for patient groups, and partnerships across government, industry, physicians, patients, and other stakeholders should be promoted to codesign solutions. Transparency around the impact of patient evidence needs to be increased, with data shared across jurisdictions to maximize impact. Similarly, a siloed approach to data should be avoided by adopting a whole-of-government approach to data access across all departments (e.g., criminal justice, education), as well as developing clear guidance on the use of real-world evidence.

Other major challenges identified by the survey included a lack of capacity, including funding, staffing levels, and increased time pressure to conduct evaluations with the application of the societal perspective (56 percent), and the lack of appropriate patient input, political will, and expertise or training (44 percent). Similar challenges were identified by industry members, who highlighted the importance of considering the overall budget impact by conducting broad, multi-stakeholder engagement with sectors other than health, such as the education, finance, and criminal justice sectors. The survey identified an additional obstacle: the lack of consensus on which technologies (all or only high-cost ones) or which diseases (all or only severe, rare, or pediatric conditions) should be assessed from a societal perspective. The literature suggested that pilot studies, perhaps restricted to specific disease areas like rare diseases, may be helpful to demonstrate the inclusion of the societal perspective (Reference Theakston, Hofer, Garau and Steuten31).

Group-3 tackled the first of these challenges, discussing the elements of value in HTA, and factors of the societal perspective that are important, such as productivity, family spillovers, insurance value, severity of disease, and scientific spillovers (see background paper). Consideration of these elements may capture the broader impact of health technologies beyond direct medical costs. Although Group-3 agreed that the societal perspective should be considered for high-cost technologies, ideally it should be considered for all health technologies; however, the depth and breadth of the application of the societal perspective should be tailored to the technology and decision context. Group-3 felt that it is particularly important to broaden the assessment of societal impacts for lower-cost technologies that have the potential to affect large populations, such as vaccination programs, to better inform resource allocation and ensure healthcare investments align with societal values, even when individual vaccines are relatively inexpensive. Conversely, for high-cost technologies, greater emphasis should be placed on factors such as productivity loss and disease severity. Group-4 reported an ad hoc and inconsistent application of the societal perspective, driven by limited HTA capacity and funding, insufficient data, a lack of methodological standardization, and unresolved prioritization dilemmas related to the need for equitable resource allocation across the health system. A key recommendation of Groups-1 and -4 was the phased inclusion of the societal perspective using a mixed-methodology approach, incorporating both qualitative and quantitative evidence, applied to prioritized disease areas, with expansion as the system matures over a suggested 5-year timeline. Fundamental to achieving this is the need for methodological standardization with the development of a framework and protocols that incorporate patient-reported outcomes, allowing inclusion of “imperfect” but insightful data. Multi-stakeholder engagement should be encouraged and include ministries beyond health as well as public deliberation, helping to shift the mindset of decision makers from focusing on costs to focusing on “health investment.”

Group-6 identified some of the positive impacts of broadening value criteria, including better capturing the value of innovation to ensure more efficient allocation of healthcare resources, greater inclusion of diverse stakeholder perspectives in the decision-making process, and having a more patient-centered approach. However, the group also identified several potential challenges. There is a risk of exacerbating inequities if broader value elements are not consistently or appropriately captured. Ensuring transparency throughout the assessment process will require a significant shift in mindset among decision makers to support the adoption of an expanded value framework. In addition, resource and methodological challenges and the increased complexity of assessments may potentially result in HTA bottlenecks, possibly delaying access to new technologies. A key recommendation from Group-6 was to strengthen stakeholder engagement to ensure that meaningful evidence is captured, thereby improving the acceptance of broader value criteria and promoting greater transparency in decision making.