Chimeric antigen receptor (CAR) T-cell therapies, classified as advanced therapy medicine products, have revolutionized the treatment landscape for certain hematological cancers, providing new hope to patients who previously had limited options. Since the U.S. FDA approved tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta) in 2017 for relapsed or refractory B-cell precursor acute lymphoblastic leukemia and large B-cell lymphoma, respectively, evidence has suggested that CAR T-cell therapies could offer a potentially curative approach in a range of other hematological conditions.1,2,3,4

However, despite their potential to improve patient outcomes, access to CAR T-cell therapies remains inconsistent due to cost, delivery complexity, and manufacturing challenges. Additionally, disparities in access related to social determinants of health (SDOH) further limit equitable benefits, disproportionately impacting marginalized populations (such as those living in rural areas, individuals with no family or social networks, and older people).

Health technology assessment (HTA) has traditionally focused on clinical outcomes and cost-effectiveness. Although health equity has been recognized as a distinct value element in HTA, and relevant frameworks and guidelines exist, it is not routinely integrated into decision-making. As such, CAR T-cell therapies represent a valuable case study for better understanding and advancing equity considerations in HTA.

What are CAR T-cell therapies?

CAR T-cell therapies are a type of immunotherapy that modify a patient’s T-cells to target and attack cancer cells, offering effective options for relapsed or refractory hematological cancers. This process involves extracting, modifying, and reinfusing the cells, followed by close monitoring for severe adverse events. Beyond their current approved indications, CAR T-cell therapies are also being investigated for several other hematological malignancies, as well as in solid tumors and non-cancer indications such as autoimmune conditions.4

Delivering CAR T-cell therapies presents significant challenges for healthcare systems due to their complexity, high cost, and the need for specialized infrastructure and expertise. The treatment requires apheresis, cell manufacturing, conditioning therapy, and intensive post-infusion monitoring, all conducted at accredited centers, often located in major urban areas.5 Successful delivery also requires coordination among a multidisciplinary team of physicians, nurses, and pharmacists, along with investment in treatment center infrastructure, including intensive care unit capacity and specialized training to manage severe adverse events (e.g., cytokine release syndrome and neurotoxicity).5

CAR T-cell therapies: Highlighting equity concerns in access to innovative treatments

Ensuring that equitable access to healthcare is considered in the HTA decision-making, particularly for high-cost, innovative treatments like CAR T-cell therapy, has become a growing concern. Despite advancements in science, therapeutic applications, and complication management, access to CAR T-cell therapy remains limited, with only a small percentage of eligible patients receiving treatment.6,7 This restricted access stems from challenges specific to CAR T-cell therapy, such as high costs, complex logistics, and manufacturing constraints, which are compounded by factors related to SDOH and equity.

Equity gaps are evident in disease incidence and prevalence, treatment patterns, and outcomes of patients eligible for CAR T-cell therapies. For example, racial and ethnic minorities, particularly Black and Hispanic populations, experience higher rates of certain hematological malignancies, yet are underrepresented in clinical trials that inform CAR T-cell therapy approvals.8,9 This leads to gaps in effectiveness and safety data across populations. Furthermore, differences in diagnosis and referral patterns contribute to inequities, with marginalized groups less likely to be referred to specialized centers due to limited provider awareness or implicit biases. Older adults, who could benefit from CAR T-cell therapies, are often excluded from trials, limiting evidence for their use in this population.10 SDOH, such as geographic remoteness and socioeconomic status, exacerbate inequities in access to CAR T-cell therapies once they are approved. Patients living in rural areas face logistical and financial barriers to reaching treatment centers, while individuals from lower socioeconomic backgrounds struggle with transportation, caregiving responsibilities, and lost wages.11,12 These overlapping disparities create a cumulative burden, limiting equitable access and worsening outcomes for historically underserved groups.

Exploring equity factors in HTAs of CAR T-cell therapies and the journey toward inclusive access

Traditional HTA frameworks have historically overlooked equity considerations, prioritizing clinical efficacy and cost-effectiveness while neglecting how SDOH and equity factors affect patient access and outcomes. This gap not only exacerbates disparities but also fails to incentivize health technology developers to commit to systematic evidence gathering and addressing these issues in their evidence submissions. While several modified economic modeling approaches that account for equity considerations exist (e.g., distributional cost-effectiveness analyses, equity-based weighting, multi-criteria decision analysis), there is a lack of consensus on which approach is best and how these methods can systematically be incorporated into HTA.13,14 As a result, HTAs often do not account for the unique burdens faced by underserved populations, such as indirect costs related to travel, caregiving, and lost income, further exacerbating existing inequities.

Recent commitments to equity from HTA bodies present valuable opportunities to ensure fair access to novel, high-cost therapies.15,16 CAR T-cell therapies, with their complex delivery and high cost, serve as a compelling case study for examining how HTA bodies incorporate equity considerations into their assessments. To explore this further, we conducted a review of 18 HTAs from Canada’s Drug Agency and the National Institute for Health and Care Excellence, focusing on six CAR T-cell therapies. Our review found that most submissions acknowledged disparities in disease incidence, treatment, and outcomes based on race, socioeconomic status, diagnosis and referral patterns, and age. These disparities were often linked to financial and geographical barriers that disproportionately affect marginalized groups. However, there were limited and inconsistent efforts to quantify these factors in the economic modeling or in the analysis of the clinical evidence submitted. This likely reflects the fact that HTA bodies do not routinely require sponsors to quantify equity concerns within their submissions, leading both decision-makers and companies to potentially overlook these issues.

Cytel will present the results of this review at the 2025 ISPOR conference in Montreal, Canada, where we will explore how gaps in HTA evaluations can inadvertently perpetuate inequities in access to CAR T-cell therapies. Join us at our podium session to learn more about how incorporating equity considerations into HTA processes can promote more equitable outcomes and ensure that all patients, regardless of their background, can benefit from CAR T-cell therapies. Do not miss this opportunity to engage in the discussion on advancing inclusive access to high-cost, innovative therapies.

Addressing equity concerns in CAR T-cell therapies: Strategies for inclusive access

Cytel can support pharmaceutical clients in addressing equity concerns through the following offerings:

• Innovative trial designs that consider elements of health equity
• Generation of real-world evidence to supplement trial programs
• Lifecycle evidence generation to support value in diverse groups of patients
• Advanced analytics, such as transportability analyses, to maximize the use of evidence generated in other settings
• Quantifying the impact of inequalities in the value proposition of new health technologies.

Share Via