Health disparities and health technology assessment: Current value assessment frameworks fall short

Approaches taken by health technology assessment bodies may not be inclusive of all patient populations. Health disparities play a pivotal role in health outcomes of marginalized patient populations. While many health technology assessment organizations understand the importance of addressing health disparities, they rarely incorporate health disparities into the value assessment process.

Introduction

A growing body of evidence suggests that health and healthcare disparities threaten many patient populations across a variety of different disease states. The terms health disparities and healthcare disparities are similar but there are nuances in each definition. Health disparities refer to differences among patient groups in outcomes and health measures in disease states, such as differences in incidence, prevalence, and mortality. Healthcare disparities refer to barriers in access to care across specific patient populations. Health disparities exist across a variety of different dimensions, including but not limited to racial/ethnic groups, gender, sexual orientation, age, disability status, socioeconomic class, and geographic location.

Historically, differences in infant mortality rates in the United States (US) have served as an example of health disparities. Native Americans and Alaska Natives have an infant mortality rate that is nearly 60% higher than the rate in White Americans. In 2013, Black Americans had the highest infant mortality rate of any ethnic group; the rate was 11.11 infant deaths per 1,000 births. In comparison, the rate in Asians/Pacific Islanders was 3.90 deaths per 1,000 births. Health disparities are also present in common diseases such as heart disease; in 2010, Black Americans were nearly 30% more likely than White Americans to die prematurely from heart disease and Black men were nearly twice as likely as White men to die prematurely from stroke. The recent COVID-19 pandemic brought these disparities to light once again, as there are clear differences in health outcomes across different patient populations. Recent analyses suggest that there are dramatic racial and ethnic variances in incidence and mortality related to COVID-19.

Health disparities are highly impactful for underserved patient populations, and therefore serve as an important contextual consideration when assessing the value of new health technologies. Historically, health disparities have not been considered a core component in health technology assessment (HTA) efforts. The following review describes the current state of health disparities in the context of value assessment in the US and 2 European HTA bodies.

ICER

The Institute for Clinical and Economic Review (ICER) is an HTA group in the US that provides an independent source of evidence review for more than a decade. ICER has undertaken several efforts recently to address health disparities in their work. ICER presents a voting question to the review panel during the public meeting of their value assessments to determine an intervention’s ability to reduce important health disparities as a “potential other benefit or disadvantage.” ICER has also stated that health disparities play a role in topic selection. ICER prioritizes potential topics based on a variety of factors, one of which is described as “topics that are particularly relevant to the public due to prevalence, severity, disparities, and cost.” ICER also states that it prioritizes topics that “involve vulnerable populations with the potential to reduce health disparities.” Additionally, ICER has described the importance of having a leadership team that understands and has experience with reducing health disparities. In a statement announcing the appointment of new governance board members, ICER highlighted the new members’ experience with closing health disparity gaps. Finally, ICER has explored the possibility of conducting scenario analyses to capture the impact of disparities on life expectancy across different subpopulations in reviews, if feasible. In addition to these initiatives, ICER’s mission is to provide high-value care to all patients at a fair price, indicating that health inequities should be central to their approach.

In reviewing the recent ICER evidence report on therapies for high cholesterol, it’s clear that an effort is made to incorporate health disparities. The background section notes differences in atherosclerotic cardiovascular disease burden by race and sex, and states that patient groups and clinical experts are concerned about longstanding health disparities in this disease state. In the report summary, authors note that the burden of the disease is disproportionately shared by vulnerable populations and it’s essential that payers allow access to novel lipid-lowering therapies in these populations at a cost-effective price. Despite these efforts, health disparities are not incorporated analytically in the modeling approach or comparative clinical effectiveness review.

Other US-based value assessment frameworks

There are several other US-based value assessment organizations that have varying levels of influence on key decision makers. This review examines the American Society of Clinical Oncology (ASCO), the National Comprehensive Cancer Network (NCCN), and the Innovation and Value Initiative (IVI). These 3 organizations each have unique value frameworks, with ASCO and NCCN specifically focused on value assessment of oncology therapeutics.

ASCO has announced several initiatives related to addressing health disparities. In 2009, ASCO released a policy statement on disparities in cancer care. In 2013, a Health Disparities Committee was established, and was later renamed to the Health Equity Committee. In 2020, ASCO released an updated policy statement on cancer care disparities and health equity. The policy statement includes 4 key recommendations related to health disparities: 1) ensure equitable access to high-quality care, 2) ensure equitable access to research, 3) address structural barriers, and 4) increase awareness and action. The statement aims to affirm ASCO’s commitment toward achievement of cancer health equity and encourages key cancer care stakeholders to move toward this shared goal. In 2021, ASCO published recommendations for a framework to address cancer care disparities in underserved populations. All of these initiatives demonstrate consistent effort in addressing health disparities; however, ASCO has not incorporated health disparities in its value assessment activities in a meaningful way. The ASCO Value Framework aims to facilitate shared decision making between providers and patients to guide the selection of high-value treatments, but the framework does not utilize inputs that are reflective of health disparities across different subpopulations.

NCCN’s Evidence Blocks are another US-based value framework that is used to inform cost-effective cancer care. The Evidence Blocks consist of 5 key measures about specific cancer therapies: 1) efficacy of a regimen, 2) safety of regimen, 3) quality of evidence, 4) consistency of evidence, and 5) affordability of regimen. These 5 key measures are rated on a 5-point Likert scale and are visually presented to provide a quick overview of the overall cost-effectiveness and clinical benefit. The intent is for providers and patients to be able to quickly scan across various regimens and get a general sense of the 5 key measures in relation to one another. The goal of NCCN’s Evidence Blocks is to provide healthcare providers and patients with the information that is needed to make informed choices when they select a cancer care regimen, with cost as a consideration along with clinical factors. While the Evidence Blocks may be useful in accomplishing that goal, health disparities are not incorporated in the framework. Because NCCN’s Evidence Blocks are a simplified value framework that doesn’t involve modeling, and there are complex issues related to health disparities, it would be difficult to incorporate health disparities in the present form of the Evidence Blocks. For example, adding health disparities as another key measure that is rated using a 5-point Likert scale would be difficult to consistently and reliably measure. Nevertheless, health disparities have been demonstrated to have a significant impact on health outcomes for patients and, thus, should be a fundamental factor considered in any sort of value assessment.

IVI is an additional US-based value assessment organization. IVI has published principles that support efforts to reduce health disparities and improve health equity. One of these principles is the goal of “cultivating modernized methods,” which includes a statement on the importance of reducing discrimination and disparities based on patient heterogeneity or disability. Another principle is the goal of “supporting health equity,” which states that value assessment should facilitate various subgroup and distributional analyses to reflect diverse communities of patients in pursuit of improving access to care and equity in care. Additionally, IVI has published press releases and conducted webinars highlighting the importance of addressing health disparities. During the COVID-19 pandemic, IVI published a press release on value assessment in the age of COVID-19, and included statements on the importance of incorporating a broader societal and public health perspective to reduce health disparities and increase equity. Additionally, IVI hosted a 3-part webinar series including sessions titled “Meaningful Action Toward Health Equity,” “Methods in Value Assessment That Support Equity,” and “Walking the Talk – How Should Policy Uphold Equity in Value Assessment?” that aimed to define equity in the context of value assessment and describe actions that must be taken to improve health equity. IVI’s open-source value models can be adapted to account for various novel value elements, which may include health disparities. Despite the adaptability in models, there appears to still be a lack of available data to drive meaningful inclusion of health disparities in the value assessment process. For example, in IVI’s recent assessment on major depressive disorder, the report mentions subgroups defined by age, race/ethnicity, and socioeconomic status as target subpopulations in the simulation but do not elaborate on how this subgroup analysis will be conducted in the context of the full analysis.

NICE

The National Institute for Health and Care Excellence (NICE) produces guidance on various health-related areas and assesses the use of technologies for the National Health Services of England and Wales. These assessments provide information on the clinical and cost-effectiveness of new and established medical technologies. The technology appraisal process is rigorous and standardized across various therapeutic areas, although health disparities are not considered as a key factor in this process. In NICE’s guide to the technology appraisal process, there are statements that subgroups of patients may be identified during the scoping process and national organizations representing specific ethnic groups may be identified as an interested party to provide feedback during the process. Additionally, in the evidence submission template for technology appraisal, NICE encourages manufacturers to provide information on special considerations including issues related to equity or equality.

IQWIG

The Institute for Quality and Efficiency in Health Care (IQWiG) is a research institute that is commissioned by the German Joint Federal Committee (G-BA) as a regulatory agency to provide both an assessment of the comparative clinical effectiveness of medical technologies and a recommendation on the added clinical benefit of a new technology. In their General Methods – Version 6.0, IQWiG notes that specific subgroup analyses may be performed as part of the assessment and may examine differences among subgroups that are either qualitative (such as a reversal of effect in one group but not another) or quantitative (such as differences in effect size). The goal of subgroup analyses is to identify particular groups of patients that may benefit more or less from the intervention being assessed. The General Methods guidance notes several limitations with subgroup analyses, including a potential lack of power, multiple testing bias, and comparability between treatment groups within subgroups. If subgroup analyses are not planned prior to the clinical trial and/or the statistical analysis plan, then they may not carry any characteristic of proof. Despite these limitations, the guidance notes that IQWiG is required to consider characteristics (eg, specific to age or gender) – suggesting that health disparities among these patient groups must be considered in a subgroup analysis when applicable. Nevertheless, when reviewing a recent assessment of nivolumab in esophageal cancer conducted by IQWiG, the subgroup analyses were often clinical subgroups (number of previous treatments, clinical biomarkers, etc) rather than subgroups related to health disparities.

Key takeaways

While HTA bodies in the US have led initiatives and expressed a commitment to reducing health disparities, they rarely incorporate health disparities in their value frameworks. Many bodies, such as ICER, have cited a lack of clinical trial diversity as a limiting factor in the incorporation of health disparities. The data simply are not available to meaningfully assess these patient populations. Even in European bodies such as NICE and IQWiG that are considered to be ahead of the US in terms of HTA, health disparities are not incorporated; however, health disparities in the United Kingdom and Germany may not be as pronounced as they are in the US. Health disparities and inequalities have been addressed from a policy perspective quite extensively in European countries over the last 30 years; while US policymakers have also attempted to address health disparities in recent decades, the focus on disparities due to underlying racial and socioeconomic differences is much more pronounced. Additionally, the lack of universal coverage and poor efficiency of the US healthcare system are limiting for progress in health disparities (Table 1).

Table 1. Summary of VAF/HTA body approaches to health disparities

HTA bodies must take action to improve upon how they address health disparities in their value assessment in order to align with their stated principles and commitments. To provide these bodies with adequate data, manufacturers should commit to including more diverse patient populations in their clinical trials. The European Medicines Agency (EMA) published a network strategy highlighting key strategies into 2025. In this document, the EMA highlights the importance of addressing inequalities in healthcare across the European Union and promote equity in clinical trial development. The US Food and Drug Administration (FDA) has published guidance to encourage manufacturers to aim for diverse participation in clinical trials. This guidance includes suggestions such as adopting inclusive trial practices, broadening eligibility criteria, and reducing the burden of clinical trials on patients, adopting enrollment and retention practices to enhance inclusiveness. These recommendations are all nonbinding to manufacturers and are treated as suggestions rather than standards. The Pharmaceutical Research and Manufacturers of America (PhRMA), a national organization representing manufacturers, also has published guidance on clinical trial diversity. The guidance focuses on building trust and acknowledging past failures in clinical trial design, especially with ethnic minorities, reducing barriers to clinical trial access, using real-world data to enhance information on diverse population, and inclusion in clinical trial participation. These principles mirror those of the FDA’s guidance, but both are suggestions to manufacturers rather than standards.

Some large pharmaceutical manufacturers have published principles on clinical trial diversity as well, suggesting that these guidances are being addressed by the industry to some degree. For example, Eli Lilly published a statement recognizing the failures of the industry to include diverse population with a commitment to include more minority participants in clinical trials. Eli Lilly has been able to achieve some progress; 39% of the most recent 12,000 US patients in their clinical trials at the time the statement was published were minorities. Pfizer has published a similar statement and states that their mission is to establish diversity in terms of race, ethnicity, age, and gender across their research portfolio.

The case for real-world evidence in lieu of clinical trial data is convincing for understanding specific patient subgroups. US HTA bodies currently favor clinical trial data for clinical effectiveness and cost-effectiveness analyses; however, as stated earlier, clinical trial data are not always available for certain patient populations. Manufacturers should invest in real-world evidence studies to fill the gaps in underrepresented patient populations for products that are already marketed and should commit to more diversity in clinical trial enrollment for products that are in clinical trial research. HTA bodies should work to adapt their current value assessment frameworks to incorporate real-world evidence in analyses for marginalized patient populations, even if they are scenario analyses that are supplementary to the primary findings. 

Awareness of health disparities is growing in the industry, but principles, mission statements, and guidances must be followed by action to be truly impactful. It is easy to recognize that health disparities exist in the US, but it is much more difficult to do something about it. There’s hope that awareness is slowly translating into actions that will provide the necessary evidence to address health disparities, but we’re not there quite yet. Efforts from multiple key stakeholders are necessary to ensure that marginalized patient subgroups have a seat at the table when value in health is being determined.

The article should be referenced as follows: 

Cook S, Tan R. Health disparities and health technology assessment: Current value assessment frameworks fall short. HTA Quarterly. Fall 2021. https://www.xcenda.com/insights/htaq-fall-2021-health-disparities-hta-vaf

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