Abstract
Chimeric antigen receptor T-cell (CAR T) therapy has revolutionized treatment outcomes for hematologic malignancies, notably relapsed or refractory B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma, offering significant survival and remission benefits. Brazil remains the sole country in Latin America with access to CAR T therapy, yet widespread implementation faces substantial barriers, including complex manufacturing, specialized infrastructure requirements, and prohibitive costs of approximately USD 500,000 per patient. In July 2025, the Americas Health Foundation convened a multidisciplinary panel of Brazilian experts in oncology, regulatory science, public health, patient advocacy, and healthcare technology to evaluate these challenges and identify strategies for sustainable access. The analysis highlights marked disparities between the publicly funded Unified Health System and the private insurance market, with many patients resorting to litigation to secure treatment. Emerging initiatives—such as domestic manufacturing collaborations and innovative reimbursement models—are discussed as potential solutions to cost and supply obstacles. The report integrates clinical, economic, regulatory, and infrastructural perspectives to guide policymakers, health administrators, and industry stakeholders. Priority recommendations include fostering local production capacity, streamlining regulatory pathways, expanding specialized treatment centers, and implementing equitable funding strategies. These measures aim to strengthen CAR T therapy accessibility in Brazil and provide a replicable framework for other resource-limited settings confronting similar barriers.
1 Introduction
Chimeric antigen receptor T-cell (CAR T) therapy represents a transformative field advancement in oncology treatment, offering unprecedented improvements in survival and remission rates, and has dramatically changed the treatment landscape for hematologic malignancies worldwide. This innovative cellular therapy genetically modifies a patient’s T cells to express synthetic chimeric antigen receptors (CARs), enabling the immune system to selectively recognize, bind to, and eradicate tumor-associated malignant cells (). Currently, Brazil is the only country in Latin America with access to CAR T therapy. In Brazil, patients undergoing therapy for relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) and diffuse large B-cell lymphoma (DLBCL) have been among the first to be offered access to CAR T therapy. This access addresses a critical unmet need, as relapsed or refractory patients have limited effective treatment options and poor survival outcomes. By offering CAR T therapy, Brazil provides a potentially curative intervention for patients who would otherwise face palliative care or rapid disease progression.
The regulatory approval landscape governing patient access to CAR T therapies across Brazil has undergone rapid evolution, marked by a growing number of product approvals from international healthcare authorities. Although Brazil has begun to achieve early success in developing regulatory frameworks for CAR T therapy to address unmet patient needs, broader implementation remains challenging. The therapy’s highly complex manufacturing process, which demands specialized infrastructure, highly trained personnel, and rigorous quality control, poses substantial barriers to scalability, particularly in resource-constrained settings such as Brazil (). Manufacturing costs alone can reach an estimated USD 500,000 per patient, excluding significant ancillary expenses for treatment administration, hospitalization, and management of therapy-associated adverse events (). Lessons from international contexts underscore the importance of collaborative governance models in navigating the regulatory, logistical, and financial complexities inherent in innovations such as CAR T (). This report aims to provide a comprehensive analysis of the outstanding value of CAR T therapy for patients with hematologic diseases in Brazil, incorporating diverse stakeholder perspectives and evidence-based recommendations for successful implementation. By examining clinical evidence, economic considerations, regulatory requirements, and infrastructure needs, the authors aim to inform policy decisions and guide the development of sustainable pathways for this innovative cancer treatment in Brazil and across other resource-limited countries.
2 Materials and methods
The Americas Health Foundation convened an expert panel meeting in July 2025 for a 3-day meeting to explore the barriers to CAR T-cell therapy in Brazil. The panel included six Brazilian panelists representing diverse areas of expertise, including hematology–oncology, regulatory science, public health policy, patient rights, and healthcare technology. Experts were chosen based on their leadership roles in their respective areas.
Before the meeting, a literature search was conducted in PubMed to inform the agenda and ensure an evidence-based discussion framework. The search terms were as follows:
Broad CAR-T in Brazil (“chimeric antigen receptor” OR “CAR-T” OR “CAR-T-cell” OR “CAR-T cell”) AND (Brazil OR Brazilian)
Access and availability (“chimeric antigen receptor” OR “CAR-T”) AND (Brazil OR Brazilian) AND (access OR availability OR “health care access”)
Reimbursement and economics (“chimeric antigen receptor” OR “CAR-T”) AND (Brazil OR Brazilian) AND (reimbursement OR coverage OR “health insurance” OR “cost-effectiveness” OR “economic evaluation” OR “health technology assessment” OR HTA)
Regulatory, legal, and policy gaps (“chimeric antigen receptor” OR “CAR-T”) AND (Brazil OR Brazilian) AND (regulation OR regulatory OR “health policy” OR legal OR legislation OR “approval” OR “ANVISA”)
Institutional readiness and manufacturing (“chimeric antigen receptor” OR “CAR-T”) AND (Brazil OR Brazilian) AND (manufacturing OR “cell processing” OR “GMP” OR “infrastructure” OR “hospital readiness” OR “treatment center”)
Patient impact and outcomes (“chimeric antigen receptor” OR “CAR-T”) AND (Brazil OR Brazilian) AND (“patient outcomes” OR survival OR “quality of life” OR toxicity OR “cytokine release syndrome” OR neurotoxicity)
Implementation and stakeholder perspectives (“chimeric antigen receptor” OR “CAR-T”) AND (Brazil OR Brazilian) AND (implementation OR “stakeholder” OR “policy recommendation” OR “scale-up” OR “barrier” OR facilitator)
The meeting was structured into six thematic sessions, each guided by a set of precirculated questions focusing on:
The current status of CAR T-cell therapy and distinguishing CAR T technologies.
The landscape of CAR T-cell therapy in Brazil: availability and access.
The landscape of CAR T-cell therapy in Brazil: reimbursement.
Evidence of patient impact.
Institutional, legal, and regulatory gaps preventing the introduction or expansion of CAR T-cell therapy in Brazil.
Actionable recommendations to improve access to CAR T-cell therapy in Brazil at the right clinical moment, including proposed stakeholders and adaptations.
Before the meeting, each panelist was assigned a theme and was required to write a short paper. All papers were distributed before the meeting. The panel met for 3 days to discuss all papers, imparting their personal experience and additional evidence, and develop the paper. Panelists drew on empirical evidence and personal knowledge to review all papers. This manuscript is the result of a collaboration. Discussions were moderated by staff from the Americas Health Foundation. All authors agreed on the content of the manuscript.
3 Results
3.1 CAR T-cell therapy: evidence on efficacy, durability, and patient wellbeing
Approximately 23,000 new cases of non-Hodgkin lymphomas (NHLs) and leukemias were estimated in Brazil for 2023, with most originating from B lymphocytes (). Since CD19 surface marker expression is unique and restricted to B-lineage cells, this antigen represents a desirable target for immunotherapy (). Treatment with anti-CD19 CAR T cells represents a breakthrough in treating relapsed/refractory (R/R) B-cell malignancies, such as NHL and ALL, non-Hodgkin lymphomas and chronic lymphocytic leukemia, and refractory B-cell ALL (–). In the US, these results led to the FDA (Food and Drug Administration) approval of the first cellular products in 2017. Currently, three anti-CD19 CAR T products are approved for commercial use in Brazil ().
CAR T cells are artificially modified by inserting CARs into the T-cell surface, redirecting them against neoplastic cells (). CAR recognition of target cell antigens is independent of human leukocyte antigen (HLA) restriction (). CARs contain extracellular antibody-derived recognition domains and intracellular signaling domains, which combine receptor and costimulatory signals (, ). Second-generation CARs incorporate costimulatory domains like CD28 or 4-1BB (, ), enhancing T-cell persistence and clinical efficacy (, ).
In the context of ALL, tisagenlecleucel (tisa-cel) received FDA approval for R/R B-ALL in children and young adults in 2017, based on the pivotal ELIANA study (Table 1). This study demonstrated efficacy, with a complete remission rate of 81% within 3 months of infusion in patients aged 25 years or younger with R/R B-ALL. The 12-month disease-free survival rate was 59%, and the 12-month overall survival rate was 76% (). Long-term follow-up data showed that 55% of patients who achieved a complete response remained in remission at 24 months (). Real-world studies validated its efficacy and safety profile in this population (). The FDA approved brexucabtagene autoleucel (brexu-cel) for adult patients with R/R B-ALL in 2021; however, its regulatory pricing framework has been challenging for physicians and patients. The approval for B-ALL was based on the ZUMA-3 study, which showed a complete remission rate of 83% in adult patients with R/R B-ALL (). This therapy demonstrated efficacy in patients who failed previous treatments, including those who relapsed after allogeneic hematopoietic stem cell transplantation.
Table 1
| Active ingredient | Brand name | Approved indications | Approval date | Manufacturer |
|---|---|---|---|---|
| Tisagenlecleucel | Kymriah® |
| 2022 | Novartis Biociências S.A. |
| Axicabtagene ciloleucel | Yescarta® | Adults with LBCL after ≥2 prior treatments (primary refractory and relapse within 12 months from first line of therapy) | 2023 | Gilead Sciences Farmacêutica do Brasil Ltda. |
| Brexucabtagene autoleucel | Tecartus® | Adults with R/R MCL after ≥2 prior treatments | 2023 | |
| Adults with R/R precursor B-ALL | ||||
| Ciltacabtagene autoleucel | Carvykti® | Adults with R/R MM who have received a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody | 2022 | Janssen-Cilag Farmacêutica Ltda. |
Current ANVISA-approved CAR T therapies, indications, and line of therapy in Brazil.
R/R, relapse/refractory; B-ALL, B-cell acute lymphoblastic leukemia; DLBCL, diffuse large B-cell lymphoma; LBCL, large B-cell lymphoma; MCL, mantle cell lymphoma; MM, multiple myeloma; CD, cluster of differentiation.
In the setting of NHL, particularly R/R DLBCL, axi-cel demonstrated remarkable efficacy. The pivotal ZUMA-1 study reported overall response and complete response rates of 82% and 58%, respectively (). Long-term data updates revealed 5-year OS of 42.6% and a median OS of 25.8 months, highlighting the durability of responses (). Real-world data corroborated these results by confirming high response rates and durable remissions. However, axi-cel has been associated with a higher incidence of adverse effects, such as CRS and ICANS, compared to other approved products (tisa-cel, liso-cel) (). Tisa-cel also showed promising results in the NHL setting in the JULIET study, with overall response and CR rates of 52% and 40%, respectively, in R/R DLBCL. Long-term data revealed a 5-year OS of 43% and a median of 11.1 months (). Similar to axi-cel, real-world data corroborated the pivotal studies’ results for tisa-cel (). Liso-cel, the most recently approved product for NHL and not yet available in Brazil, demonstrated significant results in the pivotal TRANSCEND NHL 001 study and its long-term update. At a median follow-up of 19.9 months, the objective response rate was 73%, with a complete remission rate of 53%. The median (95% confidence interval) durations of response (DOR), progression-free survival (PFS), and overall survival (OS) were 23.1 months (8.6–not reached), 6.8 months (3.3–12.7), and 27.3 months (16.2–45.6), respectively. Estimated 2-year rates for DOR, PFS, and OS were 49.5%, 40.6%, and 50.5%, respectively (, ).
A direct comparison between axi-cel and tisa-cel in R/R DLBCL was conducted in the real-world data study from the French DESCAR-T registry. It revealed superior efficacy for axi-cel, with overall response and CR rates (80.4%/60.3% vs. 66.0%/42.1%), 1-year PFS (46.6% vs. 33.2%), and OS (63.5% vs. 48.8%) superior to those with tisa-cel. However, axi-cel was associated with a higher occurrence of low-grade CRS and grade ≥3 acute neurotoxicity (). These results were endorsed in meta-analyses that also demonstrated greater efficacy but with higher toxicity for axi-cel compared to tisa-cel (, ).
Beyond DLBCL and ALL, anti-CD19 CAR T cells have received approval for use in other settings. In mantle cell lymphoma (MCL), brexucel received FDA approval in 2020 and demonstrated high response rates in patients with R/R MCL (). For follicular lymphoma, axi-cel was approved following demonstration of efficacy in R/R cases (). Axi-cel also demonstrated relevant data in primary mediastinal B-cell NHL and high-grade B-cell NHL, including double-hit and triple-hit lymphomas, with responses to this therapy, offering hope for patients with these aggressive subtypes of NHL with traditionally poor prognoses (, ).
The accumulation of clinical trial results and real-world evidence led to changes in management guidelines for patients with R/R DLBCL. The National Comprehensive Cancer Network (NCCN, 2024) now recommends considering the best relationship between efficacy and toxicity when choosing between axi-cel and tisa-cel. Similarly, the Center for International Blood and Marrow Transplant Research (CIBMTR) emphasizes the importance of center experience and patient-specific factors in selecting the CAR T-cell product. Key considerations in selecting CAR T-cell therapy include the efficacy–toxicity balance, with axi-cel generally showing greater efficacy in R/R DLBCL but higher toxicity compared to tisa-cel. However, advances in knowledge and management of CRS and ICANS, plus the emergence of adequate safety data for patients with comorbidities or older age treated with axi-cel, have made axi-cel the most prescribed CAR T-cell product globally (, , , , ).
Brazil’s ANVISA granted marketing authorization for Yescarta (axicabtagene ciloleucel) for adult patients with R/R large B-cell lymphoma in 2022. All four products have demonstrated significant efficacy in their respective indications but are also associated with relevant toxicities, primarily cytokine release syndrome (CRS) and acute neurotoxicity (ICANS). Management of these toxicities has improved over time, with the development of standardized grading systems and treatment algorithms ().
3.2 Accessing CAR T therapies in Brazil’s public and private health systems
The approval of CAR T therapies by Brazil’s National Health Surveillance Agency (ANVISA) demonstrates adherence to rigorous safety and efficacy standards, creating the possibility for their integration into the national healthcare system. However, the reimbursement environment remains fragmented, with significant disparities between the publicly funded Unified Health System (Sistema Único de Saúde, SUS) and the private insurance market. Between 2020 and 2024, 104 patients in Brazil received CAR T-cell therapy, with a median age of 56 years (range 4–83), primarily for non-Hodgkin lymphoma (72%), ALL (22%), and multiple myeloma (4%) (). A 2024 report highlighted the early implementation of CAR T therapy in Brazil and underscores the role of the SBTMO/CIBMTR registry in monitoring activity, evaluating outcomes, and supporting regulatory compliance ().
Under Brazil’s current regulatory framework, any institution that meets the required safety and compliance standards is eligible for certification to provide CAR T therapy, reinforcing the principle of equitable access. However, this framework does not currently lead to improved access due to several challenges. Treatment timelines still average 100 to 150 days nationwide due to challenges in accessing CAR T therapy. In the private sector, access is further complicated by the lack of incorporation of CAR T therapies from the mandatory coverage list maintained by the National Regulatory Agency for Private Health Insurance and Plans (ANS) (). Consequently, costs are often shared between insurers and patients through higher premiums, co-payments, and deductibles. This financial burden frequently leads insurers to deny coverage, forcing patients to seek legal action to secure treatment. It is anticipated that these innovative treatments may gradually be integrated into insurance plans, likely with specific authorization processes and potentially innovative reimbursement models (, ).
A pivotal reform in the scope of the “Rol de Procedimentos e Eventos em Saúde” (List of Covered Procedures) maintained by Brazil’s National Supplementary Health Agency (ANS) has allowed the inclusion of therapies with demonstrated clinical benefit, contingent upon recommendations from Brazil’s Health Technology Assessment (HTA) agency, Brazil’s National Committee for Health Technology Incorporation in the Unified Health System (CONITEC), or at least one recognized international HTA body (Figure 1). This policy shift underscores the growing role of evidence-based medicine and HTA in determining eligibility for coverage of advanced therapies. Aligning national HTA processes with global benchmarks through initiatives promoting regulatory harmonization and integrated assessment frameworks aims to accelerate the market entry of gene and cell therapies while maintaining rigor in clinical and economic evaluation. Under Federal Law 9.656/98, private health insurers in Brazil are legally required to provide coverage for treatments included in the ANS-mandated benefits package (“rol da ANS”). Yet, Wang et al. found that courts ruled in favor of patients 96% of the time when insurers denied treatment because it was “not on the list,” demonstrating that such denials often conflict with the statutory coverage framework and prevailing jurisprudence (). Moreover, Law 14.454/2022 further reinforced this position by allowing coverage for non-listed technologies under specific evidentiary criteria, indicating that continued refusals to authorize CAR T therapy likely constitute non-compliance with existing legal and regulatory obligations ().
Figure 1
While Brazil’s regulatory framework emphasizes equitable access by stating that any institution meeting these standards should be eligible to provide therapy, in practice, operational limitations restrict the number of centers able to deliver treatment. Currently, only a limited number of institutions in Brazil possess the necessary certification and infrastructure to administer CAR T therapy safely. The manufacturer must verify each of these institutions to ensure compliance with global safety protocols.
3.3 Improving CAR T accessibility in Brazil: from national initiatives to judicial leverage
A combination of judicial activism, regulatory reforms, and investment in local biomanufacturing is driving efforts to expand access to CAR T therapy in Brazil. Under Brazil’s Constitution, health is recognized as a fundamental right, and litigation is a key tool for patients and advocacy groups in their efforts toward securing access to expensive therapies. The Supreme Federal Court (STF) has frequently ruled in favor of patients, requiring the provision of treatments that would otherwise be unavailable. This strong judicial precedent is especially relevant for CAR T therapies, where patients and civil society organizations are increasingly turning to the courts to demand timely and equitable access to these life-saving interventions.
At the same time, new reimbursement models are being considered to make CAR T therapy financially sustainable. Outcome-based agreements, which link payment to measurable results such as survival or long-term response, reflect a broader shift toward value-based healthcare financing. While these agreements promote efficiency and risk-sharing in theory, their adoption in Brazil has been hindered by administrative hurdles, fragmented health data systems, and the absence of mechanisms for retroactive payment adjustments. To overcome these barriers, simplified approaches including bundled payments tied to explicit, verifiable outcomes are emerging as more practical options. In parallel, investments in digital infrastructure to track patient outcomes could help generate real-world evidence, strengthening future HTAs, price negotiations, and coverage policies ().
Beyond legal and regulatory innovation, Brazil is actively cultivating domestic manufacturing capabilities to address the cost of commercially available CAR T products (Figure 2). One of the most significant efforts is the collaboration between Fundação Oswaldo Cruz (Fiocruz) and the U.S.-based non-profit Caring Cross, aimed at establishing national capacity for the production of CAR T-cell and gene therapies. The initial focus is on therapies for leukemia and lymphoma, with the ambitious goal of offering treatments free of charge to patients and reimbursed by the public healthcare system (Sistema Único de Saúde, SUS) at an estimated USD 35,000 per dose ().
Figure 2
Complementing this initiative is a strategic partnership between the Children’s Hospital of Philadelphia (CHOP) () and the Brazilian National Cancer Institute (INCA), supported by the Brazilian Ministry of Health. This collaboration seeks to develop an automated CAR T manufacturing facility dedicated to expanding access for pediatric oncology patients. By leveraging cost-efficient production technologies, the project aims to deliver high-quality cell therapies at a fraction of the global market cost. In addition to these international partnerships, Brazil is advancing its domestic research agenda through leading institutions such as the University of São Paulo’s Center for Cell-Based Therapy (CTC) in Ribeirão Preto and the Butantan Institute (Figure 2). These institutions have initiated clinical trials for locally developed CAR T constructs, supported by public research funding. In 2024, Brazil launched its first publicly funded phase 1/2 clinical trial for a national CAR T product targeting refractory DLBCL, with plans to enroll up to 81 participants across three sites within the Hospital das Clínicas network. Another approach is research initiatives that reduce costs by locally manufacturing in Brazil. Such initiatives could reduce production costs and enable eventual nationwide incorporation, improving affordability, scalability, and equitable access across the oncology care continuum (). These investments signal a strategic commitment to establishing Brazil as a regional hub for affordable, scalable CAR T production aligned with global safety standards and national health priorities.
3.4 Lessons from global CAR T integration for Brazil’s landscape
The global integration of CAR T therapies has exposed common regulatory, infrastructural, and financial constraints. Countries have adopted diverse strategies to bridge the translational gap between research and clinical application. In the United States, the FDA initially imposed Risk Evaluation and Mitigation Strategies (REMS) for CAR T due to severe toxicities such as CRS but recently lifted these requirements to reduce access barriers (). Germany employs a decentralized HTA system with flexible budgeting and strong coordination between payers and providers. France’s Temporary Access for Use (TAU) program enables conditional use before formal market authorization, offering a potentially adaptable model for Brazil. Spain and Japan have centralized CAR T administration in accredited centers and invested in national registries for longitudinal outcome tracking. The United Kingdom integrates CAR T into the National Health Service via Managed Access Agreements, linking reimbursement to real-world outcomes and applying flexible cost-effectiveness thresholds (Table 2) ().
Table 2
| Country | Regulatory pathway | Coverage strategy | Centers of excellence | Payment/HTA model | Brazil: adaptation potential |
|---|---|---|---|---|---|
| United States | FDA approval with initial REMS requirement (recently removed) () | Private and public insurers with CMS coverage | Certified centers with REMS training | Outcome-based reimbursement through CMS and private payers | Streamlined ANVISA process with risk management and certification requirements |
| Germany | Decentralized assessment, fast integration | Coverage via SHI with regional flexibility | Strong pharma-provider coordination | Negotiated price models, early benefit assessment | Leverage decentralized HTA and budgeting flexibility |
| France | TAU before market authorization | National health insurance under TAU | Centers of excellence participate in TAU studies | TAU-linked data collection and pricing model | Pre-approval access mechanisms with data reporting |
| Spain | Centralized approval with registry tracking | Public coverage for CAR T in selected hospitals | Specialized accredited centers | Longitudinal national registry and negotiated prices | Build a national CAR T registry and accredit centers |
| Japan | Conditional early approval with post-market surveillance | National health insurance with outcome monitoring | Designated hospitals | Reimbursement linked to outcomes | Conditional approval linked to pharmacovigilance |
| UK | NICE appraisal and conditional reimbursement via Managed Access Agreements | Public NHS coverage | NHS centers with specialized infrastructure | Value-based pricing with real-world data integration | Explore MAA for high-cost therapies |
International implementation models and lessons for CAR T therapy in Brazil.
ANVISA, Health Regulatory Agency; CAR, chimeric antigen receptor; CMS, Centers for Medicare & Medicaid Services; FDA, Food and Drug Administration; HTA, health technology assessment; MAA, Managed Access Agreements; NHS, National Health Service; NICE, National Institute for Health and Care Excellence; REMS, Risk Evaluation and Mitigation Strategy; SHI, statutory health insurance; TAU, temporary access for use.
For Brazil, adapting elements of these international models is essential. Regulatory modernization through ANVISA is underway, and Brazil’s Medicines Market Regulation Chamber (CMED) has also launched a public consultation period to establish a new procedure or revise the rules that produce the price for CAR T products in Brazil. Civil society groups and patient organizations have called for a comprehensive and transparent regulatory framework that includes lifecycle management from clinical development through post-infusion monitoring to reduce judicialization and ensure equitable access ().
A national clinical guideline is also necessary, particularly to standardize the management of treatment-related toxicities such as CRS and ICANS, which remain leading causes of morbidity (). Despite select centers of excellence, Brazil’s delivery infrastructure remains insufficient. CAR T requires not only trained multidisciplinary teams and specialized units but also coordinated referral systems.
To maximize clinical and economic value, Brazil must enforce national protocols focused on diagnostic accuracy and patient selection. Requiring accredited centers to report outcomes such as survival and toxicity would create a results-based feedback loop to drive quality improvement and cost efficiency. Performance-based reimbursement models, such as outcome-based contracts piloted for spinal muscular atrophy therapies in Latin America, offer a promising path for improving access and, potentially, integrating CAR T into Brazil’s public health system. Finally, the inclusion of patient advocacy organizations in formal governance, as exemplified by the recent legislative mandate to incorporate civil society into CONITEC, enhances legitimacy and aligns health technology policy with patient priorities (). The private market can serve as a catalyst for innovation and capacity-building by piloting new payment and delivery models before integrating them into the public system. Its flexibility allows faster adoption of outcome-based contracts and advanced data infrastructure, generating real-world evidence that can inform national policy and reimbursement standards. By leveraging private investment and operational efficiency, Brazil can accelerate the scaling of CAR T therapies while reducing the financial risk to the public sector.
4 Discussion
The limited and unequal access to CAR T therapy in Brazil has resulted in significant clinical and ethical consequences, particularly for patients with rapidly progressive hematologic malignancies. The only published national dataset (Journal Bone Marrow Transplant Cell, 2023) reports outcomes for patients who received CAR T, omitting eligible individuals who never received treatment. This exclusion masks a critical gap in care delivery: many patients deteriorate clinically or die while awaiting access to CAR T therapy, events not captured in conventional outcome metrics. These omissions represent a systemic failure in care equity and timely intervention. Global real-world evidence further underscores the severity of these delays. Rejeski et al. reported that more than 20% of referred patients never reach infusion, primarily due to disease progression during the waiting period for CAR T treatment. In Brazil, the impact of such delays is amplified by regional disparities in healthcare infrastructure, inconsistent referral pathways, and the absence of integrated clinical care delivery mechanisms. Collectively, these structural barriers limit early intervention, which is essential, particularly when administered at low tumor burden and preserved performance status.
The national expansion of CAR T therapy is further constrained by regulatory and institutional fragmentation. Although recent policies, including Law No. 14.874/2024 and ANVISA’s RDCs 505/2021, 836/2023, and IN 270/2023, provide a legal basis for clinical trials, product registration, and manufacturing, Brazil lacks a comprehensive legislative framework governing patient access, pricing, reimbursement, and public coverage for advanced therapies. This regulatory vacuum impedes long-term planning and discourages investment in the sector. Moreover, judicial disputes such as the Sindusfarma challenge to ANS Technical Note No. 3/2023 highlight the escalating judicialization of health policy in Brazil in response to insurers’ efforts to evade payment for treatments that constitute a guaranteed patient right.
From a systems-level perspective, despite patient need and clinician support, Brazil’s readiness to implement CAR T therapy at scale remains low. While centers of excellence exist nationwide, the broader health system lacks the infrastructure, financing models, and cross-sectoral coordination required for national deployment. There is also a historically low referring rate from community hematologists to CAR T treatment centers. The absence of a coordinated, multisectoral implementation strategy encompassing federal and state health authorities, regulatory bodies, industry stakeholders, payers, and patient advocacy groups further hampers progress.
CAR T’s complex supply chain, regulatory dependencies, and need for real-time patient monitoring are particularly relevant in Brazil, where institutional fragmentation and disparities in implementation infrastructure require more nuanced assessments of systemic capacity and policy alignment. Brazil faces a convergence of challenges that hinder equitable access to CAR T therapy, spanning from legal and regulatory ambiguities to infrastructural, economic, medical education, and governance constraints. Addressing these gaps is essential not only to reduce preventable mortality but also to ensure the transformative potential of CAR T is realized within a framework of justice, sustainability, and universal health coverage.
5 Conclusion
CAR T-cell therapy represents a paradigm shift in the treatment of hematologic malignancies, with compelling clinical efficacy and durable responses. However, its successful integration into Brazil’s health system requires overcoming substantial barriers related to regulation, infrastructure, financing, and equitable access. While Brazil has made meaningful progress through regulatory approvals, judicial activism, and early steps toward local manufacturing, systemic gaps remain in national protocols, reimbursement mechanisms, and clinical infrastructure. Drawing on global implementation models, Brazil must now develop a coordinated, multisectoral strategy that includes patient-centered governance, adaptive regulation, and value-based reimbursement to ensure that CAR T therapy fulfills its transformative potential within a framework of universal and equitable care (Table 3).
Table 3
| Recommendation | Stakeholder(s) responsible | Anticipated impact | Timeline |
|---|---|---|---|
| Maintain an iterative national clinical guideline for CAR T therapy (with updates as evidence evolves) | Medical societies, public and private payers | Detail the indication criteria, contraindications, treatment regimen, management of toxicities, and follow-up. A temporary national guideline will allow flexibility for adjustments as national experience accumulates and new evidence emerges | Short term |
| Implement continuous technical training programs for multidisciplinary teams (e.g., medical, nursing, pharmacy) | Training institutions, hospitals, industry | Invest in intensive training for medical, nursing, pharmaceutical, and other specialist teams involved in CAR T therapy, in line with best clinical practice | Medium term |
| Establish and maintain a national database of eligible and treated patients | Ministry of Health, academic institutions, payers, medical societies | This database will be essential for monitoring long-term efficacy and collecting data for cost-effectiveness analyses and to assess safety and efficacy over 15 years of treatment in the Brazilian context, including real-world evidence | Medium term |
| Form a task force with civil society representation | Government, research agencies, patient groups | Coordinate efforts, develop guidelines, and meet patients’ needs | Shor term |
| Simplify and clarify reimbursement models (both public and private systems) | Ministry of Health, ANS, payers | Reduces delays in treatment access; promotes financial sustainability | Long term |
| Foster public–private partnerships to scale manufacturing capacity | Government, biotech industry, development | Expand local production capability; reduce vein-to-vein time; increase autonomy and cost control | Long term |
| Integrate CAR T therapy into national cancer plans and rare disease strategies | Government, regulatory agencies (CONITEC, ANS) | Anchor CAR T as part of a broader national policy framework; improves strategic resource allocation | Long term |
Priority recommendations and “quick wins” for stakeholders in providing CAR T therapy to patients in Brazil.
ANS, National Regulatory Agency for private health insurance plans; CONITEC, National Committee for Health Technology Incorporation.
Statements
Author contributions
RG-C: Formal analysis, Investigation, Validation, Writing – original draft. AB: Formal analysis, Validation, Writing – original draft, Investigation. AJ: Conceptualization, Methodology, Project administration, Visualization, Writing – review & editing. MO: Formal analysis, Investigation, Validation, Writing – original draft. GS: Conceptualization, Data curation, Methodology, Project administration, Writing – review & editing. RS: Formal analysis, Investigation, Validation, Writing – original draft. JS-F: Formal analysis, Investigation, Validation, Writing – original draft. SS: Formal analysis, Investigation, Validation, Writing – original draft.
Funding
The author(s) declared that financial support was received for this work and/or its publication. The organization and implementation of the conference were carried out by Americas Health Foundation, a 501(c)3 non-profit organization dedicated to improving healthcare throughout the Latin American Region and was supported by an unrestricted grant from Kite. The funder had no influence on the design, implementation, and content of this manuscript.
Conflict of interest
Authors RG-C and SS were employed by company Oncoclínicas & Co. Author SS was employed by Unimed Central RS.
The remaining author(s) declared that this work was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
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Summary
Keywords
ANVISA, Brazil Unified Health System (SUS), Brazilian health access barriers, Brazilian medicine domestic manufacturing, judicial litigation
Citation
Guerino-Cunha RL, Bessa A, Jansen AM, Oliveira M, Sanku G, Scarabel R, Schmidt-Filho J and Stefani S (2026) Bridging innovation and equity: the evolving landscape of CAR T-cell therapy access in Brazil. Front. Hematol. 5:1813861. doi: 10.3389/frhem.2026.1813861
Received
19 February 2026
Revised
13 April 2026
Accepted
20 April 2026
Published
11 May 2026
Volume
5 - 2026
Edited by
Duygu Aydemir, Istanbul University, Türkiye
Reviewed by
Lilia Carolina León-Moreno, University of Guadalajara, Mexico
Hugo Antonio Romo Rubio, Civil Hospital of Guadalajara, Mexico
Updates
Copyright
© 2026 Guerino-Cunha, Bessa, Jansen, Oliveira, Sanku, Scarabel, Schmidt-Filho and Stefani.
This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
*Correspondence: Renato Luiz Guerino-Cunha, dr.renato.cunha@outlook.com
Disclaimer
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.