The cures conundrum: The importance of getting value assessment right for transformational therapies

To ensure meaningful patient access to innovative therapies, US healthcare decision-makers must embrace evidence-based value assessment methods. In this article, we consider whether ICER’s assessment framework is sufficiently rigorous to accurately capture the value of potentially transformational therapies.

The passage of the Inflation Reduction Act has intensified the spotlight on pharmaceutical pricing in the United States (US) and reinvigorated policy conversations about how to define and measure the value of pharmaceutical treatments. Although the context is new, discussions about the application of value assessment methodologies within the US health system have been happening for years. In the last decade, numerous value assessment frameworks or tools have emerged to help US stakeholders appraise the value of healthcare treatments and services. Each value assessment tool has its own methodological approach, purpose, and intended audience, such as guiding shared decision-making between patients and their care team or informing payers’ coverage and formulary decisions.

To gain a better understanding of payer perceptions and use of US value assessment tools, AmerisourceBergen conducted a survey of 51 individuals from healthcare payer organizations on their perceptions and use of the predominant US value assessment tools cited in the literature.  

Results from this survey point to increased payer interest in and use of US value assessment tools, particularly evidence reports by the Institute for Clinical and Economic Review (ICER). Growing reliance on ICER’s assessments to inform formulary and coverage decisions has important implications for patient access to healthcare treatments, as ICER’s determinations could influence a payer’s decision to exclude a treatment from its formulary or implement stringent utilization management strategies (eg, prior authorization or step therapy requirements). Therefore, it is important that value assessors, including ICER, rely on a strong foundation of evidence-based methods. This is particularly true when assessing the value of transformational therapies which hold the promise to deliver groundbreaking improvements in patient health outcomes. 

Assessing the value of curative therapies 

In 2019, ICER published methods adaptations for the evaluation of single- and short-term therapies (SSTs). In its SST framework, ICER defines SSTs as ’therapies that are delivered through a single intervention or a short-term course (less than one year) of treatment that offer a significant potential for substantial and sustained health benefits extending throughout patients’ lifetimes.’ Within this definition, ICER identifies 2 potential subcategories: 1) potential cures that can eradicate a disease or condition, and 2) high-impact therapies that can produce sustained major health gains or halt the progression of significant illnesses. 

A vast pipeline of cell and gene therapies is currently in development. Given the unique characteristics and curative potential of these therapies, it is crucial that US healthcare decision-makers apply evidence-based value assessment methods to support meaningful patient access whilst promoting health system sustainability. We examined ICER’s SST framework and identified important considerations related to its underlying methodology and application. 

ICER’s SST framework methodology

Our analysis of ICER’s SST framework identified 3 potential methodological limitations. First, ICER’s SST framework introduced the concept of economic surplus or ‘shared savings’ analyses, which encompass 2 scenarios: 

• 50/50 shared savings scenario: 50% of cost offsets are awarded to the treatment, and 50% are excluded from the cost-effectiveness analysis (CEA) 
• Cost-offset cap scenario: Cost offsets awarded to the treatment are capped at $150,000 per year, and the remainder is excluded from the CEA

A close examination of ICER’s shared savings scenarios reveals inconsistent logic and a dearth of scientific justification to support their application. For example, the allocation estimates (eg, 50% savings awarded to treatment and the $150,000 cost-offset cap) appear to have been chosen arbitrarily. ICER did not cite any sources to support its 50/50 shared savings allocation. Moreover, ICER selected the $150,000 cost-offset cap because it aligned with the top range of its cost-effectiveness threshold, despite recognizing that real-world cost offsets in some conditions would likely far exceed this figure. The lack of rigorous empirical evidence to support these scenarios has implications for ICER’s assessments, as the application of an artificially low cost-offset cap will result in the underestimation of a treatment’s value, which could, in turn, undermine incentives for future research and development for SSTs.

Second, ICER’s SST framework does not ensure the application of a societal perspective when assessing SSTs. This methodological decision is misaligned with expert recommendations for health technology assessment (HTA). The use of the societal perspective in the assessment of SSTs is critical, as it captures the unique overall cost impacts and public benefits generated by transformational medicines by accounting for surplus resources that could be used for other purposes in non-healthcare sectors. The use of the societal perspective also provides a more representative assessment of the value of SSTs by reflecting the potential for SSTs to deliver groundbreaking advancements in patient health outcomes and replace expensive and less effective standards of care.

Third, ICER’s SST framework does not incorporate many methodological adaptations needed to account for the unique considerations and value attributes of SSTs. For example, SSTs are associated with exceptionally long-term benefits, which has led other HTA bodies to apply differential discounting rates in their respective CEAs to account for the inherent uncertainty involved in forecasting long-term cost-effectiveness. Despite such practices by other HTA organizations, ICER uses a standardised 3% discounting rate, which could lead to the underestimation of the treatment’s real-world value. Additionally, ICER applies its standard $150,000 per quality-adjusted life-year (QALY) cost-effectiveness threshold in its SST assessments. However, other HTA bodies use higher thresholds in their assessments of highly specialised technologies to reflect the high cost of developing treatments for rare disease populations. For example, the National Institute for Health and Care Excellence in the United Kingdom applies a threshold of £100,000 per QALY gained for highly specialised technologies but applies a much lower threshold of £20,000 to £30,000 in its standard technology appraisals. The application of higher thresholds for treatments associated with a large societal impact is also widely cited in the literature.    

These methodological limitations are important for healthcare stakeholders to consider when using assessments to inform decision-making, as inaccurate value determinations can result in inappropriate access barriers for patients and weaken incentives for continued research and development of innovative treatments.  

ICER’s application of its SST framework 

As of July 2023, ICER applied its shared savings scenarios in 3 complete assessments. Although ICER is applying its SST framework in its 2023 assessments of treatments for sickle cell disease and metachromatic leukodystrophy, ICER’s final assessment reports will not be completed until August and October, respectively, and are unavailable for our analysis. Our examination of ICER’s application of its SST framework in its hemophilia A (2020), beta thalassemia (2022), and hemophilia A & B (2022) reviews reveals opportunities for improvement in the following areas: 

• Transparency: In December 2022, ICER posted an updated version of its SST framework without publicly announcing the changes or allowing for public input or discourse. Moreover, the updated framework was posted the day before ICER published its final evidence report on hemophilia A & B (2022) to justify the application of new methods in the assessment. Making updates in this way challenges ICER’s claims that their assessments are transparent and informed by multi-stakeholder engagement. 
• Application of rigorous methods: In the 2019 SST framework, ICER described its shared savings scenarios as hypothetical and exploratory, stating that they were not intended to provide normative guides to pricing. However, in the beta thalassemia and hemophilia A & B assessments, ICER used these scenarios as the key determinants of its pricing benchmarks and as the focal point of its report-at-a-glance summaries. Such actions run counter to ICER’s stated processes as outlined in its methodological framework and make untested methodologies the fulcrum of its assessments, resulting in less rigorous value determinations.   
• Inclusion of patient-focused value elements: ICER’s inclusion of the societal perspective and patient-centered value elements in its SST reviews is very limited. The SST framework outlines additional value dimensions that ICER committed to including in its assessments. However, in the limited number of SST assessments completed to date, ICER rarely and inconsistently incorporated these value attributes in the application of its framework.  

 
Looking ahead

In June 2023, ICER announced proposed updates to its value assessment framework but did not signal any changes to its SST methodology apart from codifying the application of its shared savings scenarios for SSTs or other treatments with relevant and substantial potential cost offsets. ICER is now in the process of reviewing stakeholder feedback and will release its updated framework in September 2023. 

Delivering on the promise of SSTs will require the application of rigorous value assessment methods that are fit for purpose. ICER’s development of specific assessment method adaptations for SSTs represents an organizational acknowledgment of the unique considerations associated with these treatments. To advance evidence-based healthcare decision-making, current limitations to ICER’s SST framework methodology should be addressed, and ICER should evolve its processes in a transparent manner, guided by multi-stakeholder input. 

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