The Bigger Picture: Considering Broader Value Drivers and Perspectives in HEOR

ISPOR Europe this year revolved around the theme of “Generating Evidence Toward Health and Well-Being”, aligning well with ongoing efforts in health economics and outcomes research (HEOR) to consider value elements that extend beyond the healthcare perspective, to broader well-being and societal benefits.

Whilst the “ISPOR value flower” has been a topic of conversation at HEOR conferences since its introduction in 2018, discussions at ISPOR Europe 2024 showed real energy and engagement related to “non-conventional” value drivers. This perhaps reflects the methodological progress that has been made in quantifying broader aspects of value, coinciding with a recent publication by Shafrin et al.,1 which introduced an updated value inventory – the Generalized Cost-Effectiveness Analysis (GCEA) value flower (Figure 1)— described as a “user guide” for HEOR professionals seeking to estimate the value of treatments from a societal perspective.

Here we provide our perspectives on the recent developments, and consider the trade-offs between the benefit of including these additional components of value and the potential risks that their inclusion may pose for manufacturers, HTA bodies and patients.

Figure 1. GCEA value flower

Diagram showing the GCEA value flower

Source: Shafrin et al., 2024.1

Beneficiary: Equity

Perspectives from Matt Griffiths – Global Head of HTA

The intersection of health equity and HEOR continued to be an important theme at this year’s conference. As well as general commentary on the hopes for Joint Clinical Assessment to provide more equitable access to medicines across the European Union, there were a number of breakout sessions and podium presentations dedicated to discussing or demonstrating available methods for incorporating equity concerns in HTA and/or cost-effectiveness analysis. Some of these methods, such as Distributional Cost-Effectiveness Analysis (DCEA), are well-developed and gaining increasing awareness amongst the HEOR community but, as highlighted across the conference sessions, their use is currently limited by 1) data availability; 2) lack of decision-makers formally incorporating these methods as part of their value assessment framework.

However, the landscape is starting to shift here, with ICER and CADTH (now the CDA) having published reports on equity in the last couple of years. Notably, in January 2025, NICE are due to consult on a modular update to their manual, which is set to provide NICE’s latest view and guidance on considerations for when and how to include distributional impacts in assessments. I currently chair the Health Equity Research Special Interest Group at ISPOR and moderated their Open Meeting at this year’s conference. Discussions at this meeting between stakeholders spanning HTA bodies, industry, academics and consultancies demonstrated the growing interest in health equity, but confirmed that many stakeholders feel a need for stronger and clearer signals on the role of equity in value assessment, as well as more training and case studies for how to conduct these analyses.

Beneficiary: Family and Caregiver Spillover

Perspectives from Elizabeth Parke – Senior Analyst in HTA

One of the first issue panels at ISPOR this year provided some interesting perspectives on capturing the broader effects of treatments on family and caregiver spillover (i.e. how treatments impact the well-being of friends and family members), with a focus on whether economic evaluations should account for bereavement when considering caregiver health-related quality of life (HRQoL).2 Currently, many HTA agencies accept the application of a caregiver disutility as a spillover effect, but either do not recommend or do not provide guidance on capturing any further impact a technology might have on caregiver HRQoL. This may, however, result in a failure to capture important nuances in the true lived family and caregiver experience, and in particular may underestimate the value of a novel intervention in providing more time with family. The exclusion of a bereavement effect can also paradoxically suggest that a patient’s death might be a ‘utility improving event’ for caregivers. This contradicts the widely accepted belief that grief significantly impacts wellbeing and introduces what Mott et al. termed the “carer-QALY trap”.3 Incorporating a bereavement effect on caregiver HRQoL could help offset this effect and reduce the risk of undervaluing life-extending treatments, by reducing the implied utility benefit for caregivers following a patient’s death, particularly in the short term.

It is important, however, to acknowledge the unresolved challenges associated with the inclusion of a bereavement effect on caregiver HRQoL; these include uncertainty in the duration and severity of the effect, when it might be appropriate to include and whether it should be applied beyond the patient’s death. These questions gained particular interest from NICE attendees and appear to be a growing focus among the research community, with several interesting study findings presented by the panellists. Notably, one of the panellists from Zorginstituut Nederland (ZIN; Dutch HTA body) highlighted a recent review conducted within ZIN (where the economic evaluation base case includes a societal perspective) that explored potentially including bereavement effects. However, current guidance remains that these should be excluded, linking to the challenges outlined above and echoing the consensus that further work is needed before this can be effectively integrated into economic evaluations. Nevertheless, could the growing interest in this area signal that developments, including specific guidance on this topic from other HTA agencies, may be coming?

In my view, considering bereavement effects on carer HRQoL—whether quantitatively or initially on a qualitative basis whilst research is sparse—would represent a positive step forward. Its current exclusion, whilst being justified to some degree based on the complexities involved, may underestimate the true value of new life-extending technologies for caregivers and affected families. Looking forwards, clearer guidance from HTA bodies on the application of bereavement effects would be needed to ensure these can be captured in a consistent way. Furthermore, I appreciate the need for this effect to be incorporated in a simple and transparent way (e.g. by applying a carer disutility on patient death), to avoid introducing further complexity and to ensure consistency in decision-making. I believe developments such as these would support economic modelling that captures a more holistic perspective of value, whilst also ensuring its incorporation is not time- and resource-intensive, resulting in potential delays to patient access.

Environmental Sustainability: Are We Ignoring a Key Component of Value?

Perspectives from Naman Kochar – Senior Analyst

The need to minimise environmental impact is becoming increasingly relevant across sectors globally. However, in healthcare, it is notable that environmental considerations are not yet central to established value frameworks like the ISPOR or GCEA value flowers.4 This omission highlights ongoing debates about the role of environmental impact in healthcare decision-making, which were noted by our Scientific Director Lucy Eddowes at ISPOR Europe 2023 and have historically not reached consensus.1, 5 Discussions have progressed one year on, but key questions remain that the sector needs to consider.

Are environmental factors relevant to healthcare?

Healthcare providers seem to think so – for instance, National Health Service England (NHSE) has set ambitions targets of net zero carbon emissions by 2045.6 Additionally, ignoring environmental factors may risk transferring healthcare-related emission burdens onto younger and future populations, subsequently impacting health equity – which (unlike environmental impact) is covered by both the ISPOR and GCEA value frameworks.

How important are environmental factors when assessing treatment value?

During an issue panel on what health economists could learn from environmental economists’ modelling methodologies, 60% of attendees were unwilling to sacrifice healthy life years for environmental improvements.7 Reflecting findings from the NICE Listens Public Engagement Programme,8 these results suggest that sustainability is supported by wider society if it does not negatively affect patient care. The ‘general public’s reluctance to trade-off direct treatment benefits for environmental advantages suggests that the current exclusion of environmental considerations from value frameworks is justified to some degree. However, these perspectives may fail to fully consider the potential adverse effects of environmental impacts, such as long-term pollutant discharge, on population health.

Healthcare system often have different priorities to the public – for instance, research presented at the conference showed that NICE’s severity modifier framework may not be well-aligned with public prioritisation of health gains.9 Lofty targets such as those set by NHSE suggest that environmental considerations may hold greater priority for healthcare decision-makers than for wider society. In fact, environmental impact has recently been recognised by NICE as an important consideration in decisions surrounding topic prioritisation as part of the integrated topic prioritisation process.10, 11

Future evidence generation and discussion should be conducted to understand whether environmental considerations should be integrated into healthcare decision-making. For instance, large-scale discrete-choice experiments (DCEs) could yield more insights into how willing patients are to prioritise environmental benefits over treatment-related over benefits (if at all). Multistakeholder discussions should include healthcare providers, industry and the public to address potential priority disparities and inform health policy alignment.

How far are we from considering environmental value in HTA?

Beyond the question of whether environmental value should even be considered in value judgements, conference discussions highlighted that there remained a major need for methodological consensus on its integration into decision-making.

For instance, for cost-effectiveness-driven decision-making, it was unclear whether environmental considerations should feed into intervention QALYs, costs, or both. If a QALY-based approach is taken, the large-scale DCEs described above would likely be needed to derive utility values associated with environmental impact, possibly leveraging the approaches taken in small-scale exploratory analyses.12

If a cost-based approach is taken, for example by monetising CO2-equivalent emissions, methodological consensus would also be needed here. This was reflected by lengthy conference discussions with particular focus on:7

  1. The appropriate rules for discounting environmental impacts, given high upfront costs and long-term benefits beyond individual patient lifetimes
  2. Whether the carbon footprint of supply chains should be monetised and considered in decision-making, given that they can evolve over time to reduce environmental impact

Beyond this, the research of our colleagues revealed the considerable impact on ICERs of using different tariffs for monetising emissions.13 These are just a few of several areas of uncertainty in economic modelling approaches.

Even if methodological consensus is achieved, securing payer acceptance will be a likely rate-determining step. Encouragingly, HTA bodies in France and Canada have already initiated projects to explore incorporating environmental criteria in healthcare assessments,14, 15 and my hope is that other bodies follow suit. Looking forward, formal collaborations between these HTA bodies and industry and academia will be needed to align on best practice. Leveraging environmental modellers’ expertise and using formal platforms like ISPOR’s Environmental Sustainability Special Interest Group could also facilitate this.

How else could environmental impact be better considered?

As the HTA method evolution described above will likely only bear fruit in the long-term, I believe that alternative approaches are needed in the interim. Manufacturers could more proactively include environmental factors qualitatively in HTA submissions, and payers should consider these when making value judgements.

More holistic policy changes were also suggested at the conference, such as incentivising greener technologies during procurement processes or implementing carbon taxes for manufacturers.7, 16 However, I feel that policymakers need to consider broader implications on industry, such as competitive imbalances, if similar measures are not applied in other sectors, to avoid stifling innovation.

Striking the Balance: When Should We Reflect the Bigger Picture?

HTA is grounded on the principle that the assessment of new technologies should be fair. If new technologies do provide value for key stakeholders outside of current HTA frameworks, then these aspects of value should be considered. For healthcare providers, broader value elements could reflect policy objectives or public health needs. Meanwhile, for the general public, inclusion of broader value elements could ensure that decision making is grounded on factors most aligned with societal preferences for health and wellbeing, enhancing patient-centric care and cultivating trust in healthcare systems. Ultimately, industry should be encouraged and incentivised to deliver technologies that best address patient, public and healthcare needs.

However, as Lotte Steuten from the Office for Health Economics noted, the healthcare provider perspective remains predominant among European HTA bodies.17 While organisations like NICE and ZIN are open to quantifying some broader value elements (e.g. through applying severity modifiers and recognising productivity impacts), the majority of HTA bodies are not. Additionally, the question remains: how significantly would these value elements influence decision-making, even if fully captured? Including broader elements might only marginally improve fairness in value assessments, whilst conversely increasing costs for manufacturers (through the need for additional evidence generation), introducing additional complexity and uncertainty in HTA decision-making, and thereby potentially delaying patient access. Robust evidence and methods are needed to ensure these benefits can be captured accurately and consistently, and can be evaluated efficiently.

To balance the trade-off between capturing broader elements of value fully and the need for pragmatism in health-care decision making, HTA bodies should seek to implement processes that allow consideration of these additional elements specifically in situations where they would significantly impact decision-making, without imposing excessive resource demands for evidence generation and appraisal.

References

  1. Shafrin J, Kim J, Cohen JT, et al. Valuing the Societal Impact of Medicines and Other Health Technologies: A User Guide to Current Best Practices. Forum Health Econ Policy 2024;27:29-116.
  2. Issue Panel 206. Should Health Technology Assessment Include the Bereavement Effect on Health-Related Quality of Life? What Difference Could It Make to Decisions About Life-Extending Treatments?Presented at ISPOR Europe Congress, Barcelona, Spain. 2024.
  3. Mott DJ, Schirrmacher H, Al-Janabi H, et al. Modelling Spillover Effects on Informal Carers: The Carer QALY Trap. Pharmacoeconomics 2023;41:1557-1561.
  4. Lakdawalla DN, Doshi JA, Garrison LP, Jr., et al. Defining Elements of Value in Health Care—A Health Economics Approach: An ISPOR Special Task Force Report [3]. Value in Health 2018;21:131-139.
  5. Costello Medical. ISPOR Europe 2023 Conference Report: Environmental Responsibility and Patient Voice in Access and HEOR. 2023. Available here. Last accessed: December 2024.
  6. NHS England. Delivering a net zero NHS. 2024. Available here. Last accessed: December 2024.
  7. Issue Panel 208. Lessons From Climate Change Models: What Can Health Economists Learn From Environmental Economists’ Modeling Methodologies?Presented at ISPOR Europe Congress, Barcelona, Spain. 2024.
  8. NICE Listens. Prioritisation Dialogue Report. 2024. Available here. Last accessed: December 2024.
  9. H. Hayes. HTA277: Is NICE Too Severe With Severity? Exploring How Well NICE’s Severity Modifier Aligns With UK Preferences. Presented at ISPOR Europe Congress, Barcelona, Spain. 2024.
  10. National Institute of Health and Care Excellence. NICE-Wide Topic Prioritisation: The Manual. 2024. Available here. Last accessed: December 2024.
  11. Costello Medical. Public Consultation on NICE Integrated Topic Prioritisation and Strategic Principles: What’s Set To Change? 2024. Available here. Last accessed: December 2024.
  12. M. Taylor. PCR51: Are we willing to trade off our own health to save the planet? An exploratory discrete choice experiment. Presented at ISPOR Europe Congress, Barcelona, Spain. 2024.
  13. A. Smith. HTA251: Investigating the Quantitative Effect of Integrating Environmental Impacts Into Economic Evaluations: To What Extent Could Environmental Sustainability Influence Decision Making in Health Technology Assessment? Presented at ISPOR Europe Congress, Barcelona, Spain. 2024.
  14. Canada’s Drug Agency. Criteria For Conducting Environmental Assessments in CADTH HTAs. 2024. Available here. Last accessed: December 2024.
  15. Haute Autorité de Santé. Environmental Health Roadmap: Reinforcing the HAS’ Commitment to Environmental Issues in the Context of its Missions. 2023. Available here. Last accessed: December 2024.
  16. V. Ardito. EE164: Equity Implications of Including Environmental Impacts of Health Technologies in Economic Evaluations Informing Pricing and Reimbursement Decisions. Presented at ISPOR Europe Congress, Barcelona, Spain. 2024.
  17. Issue Panel 146. Broader Value Elements: Methods to Quantify Each and Their Relevance for European Markets. Presented at ISPOR Europe Congress, Barcelona, Spain. 2024.

If you would like any further information on the themes presented above, please do not hesitate to contact Elizabeth Parke, Senior Analyst (LinkedIn), Naman Kochar, Senior Analyst (LinkedIn), Isabelle Newell, UK Deputy Head of Rare Diseases (LinkedIn) or Matt Griffiths, Global Head of HTA (LinkedIn). Elizabeth Parke, Naman Kochar, Isabelle Newell and Matt Griffiths are employees at Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners.

Loading