CAR T-Cell Therapies in Italy: Patient Access Barriers and Recommendations for Health System Solutions

Jommi, Claudio; Bramanti, Stéfania; Pani, Marcello; Ghirardini, Alessandro; Santoro, Armando

doi:10.3389/fphar.2022.915342

Cited by 12 publications

(11 citation statements)

References 21 publications

(34 reference statements)

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“…Bispecific antibodies share at least partly the mechanisms of action and some toxicity aspects of CAR T cell therapies, like cytokine release syndrome (CRS), neurotoxicity, and increased rates of severe infection, but are more readily accessible due to the absence of a long manufacturing process unlike CAR T cell therapies [63]. Interestingly, these results are similar to the finding of a recent Italian analysis, reporting that from a population of 638 potentially eligible patients, only 110 patients (17%) finally received CAR T therapy [64]. However, it highlights the existence of a clear data gap that needs to be addressed by further research (Fig.…”

Section: Analysis Of Car T Cell Therapy Access For Austrian Dlbcl Pat...supporting

confidence: 66%

Barriers to patient access of CAR T cell therapies in Austria

Hopfinger

Rupp

Greil

2023

memo

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SummaryChimeric antigen receptor (CAR) T cell therapies offer a promising new therapeutic option for treating B cell malignancies, for instance relapsed or refractory diffuse large B cell lymphoma (DLBCL). However, patient access to this type of cellular immunotherapy may be limited due to systemic barriers even in wealthy Western countries. In Austria, the CAR T eligible DLBCL population is estimated to encompass approximately 56 patients based on the criteria applied for CAR T registrational trials. However, less than 40% of these DLBCL patients eligible for commercial standard-of-care CAR T cell therapy were finally treated with CAR T cell therapy in 2021 based on our analysis. This report discusses potential barriers that may impede current patient access to CAR T cell therapy and provides recommendations for systemic solutions to address these barriers and improve the CAR T access situation in Austria.

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Section: Analysis Of Car T Cell Therapy Access For Austrian Dlbcl Pat...supporting

confidence: 66%

Barriers to patient access of CAR T cell therapies in Austria

Hopfinger

Rupp

Greil

2023

memo

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“…Currently, regions use different tariffs to cover CAR T-cell therapy procedures, for instance tariffs for allogeneic or autologous stem cell transplantation. There is no evidence whether these tariffs cover the relevant procedure costs ( 57 ). In Germany, hospitals may ask for funds for innovative and complex medicines/procedures through the NUB.…”

Section: Challenges Best Practices and Health System Focus Areas For ...mentioning

confidence: 99%

Comparative analysis of CAR T-cell therapy access for DLBCL patients: associated challenges and solutions in the four largest EU countries

et al. 2023

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IntroductionCAR T-cell therapy has emerged as a promising new immuno-oncology treatment that engages the patient’s immune system to fight certain hematological malignancies, including diffuse large B-cell lymphoma (DLBCL). In the European Union (EU), CAR T-cell therapies have been approved for relapsed/refractory (R/R) DLBCL patients since 2018, but patient access is often still limited or delayed. This paper is aimed at discussing challenges to access and possible solutions in the largest four EU countries.MethodsThe analysis relied on literature review, market data collection, since homogeneous data coming from registries were not available, and discussion with experts coming from all four countries.ResultsWe calculated that in 2020, between 58% and 83% of R/R DLBCL patients (EMA approved label population) or between 29% and 71% of the estimated medically eligible R/R DLBCL patients, were not treated with a licensed CAR T-cell therapy. Common challenges along the patient journey that may result in limited access or delays to CAR T-cell therapy were identified. These include timely identification and referral of eligible patients, pre-treatment funding approval by authorities and payers, and resource needs at CAR T-cell centers.DiscussionThese challenges, existing best practices and recommended focus areas for health systems are discussed here, with the aim to inform necessary actions for overcoming patient access challenges for current CAR T-cell therapies as well as for future cell and gene therapies.

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“…Anti‐CD19 CAR structure comprises an extracellular single‐chain antibody fragment, usually from a murine origin, directed against CD19 protein, and joined to a hinge, a transmembrane domain and an endodomain, which is ultimately responsible for signal transduction [2]. The first indication approved for anti‐CD19 CAR‐T cell therapy was B‐cell acute lymphoblastic leukemia (B‐ALL) in pediatric and young‐adult patients, and diffuse large B‐cell lymphoma or primary mediastinal large B‐cell lymphoma in adults [3]. In the last years, the number of pathologies that can be treated with this therapy has substantially increased [4].…”

Section: Introductionmentioning

confidence: 99%

The challenge of standardizing CAR‐T cell monitoring: A comparison of two flow‐cytometry methods and correlation with qPCR technique

Valdivieso‐Shephard,

Matas‐Pérez,

García‐Bujalance

et al. 2024

Cytometry Pt A

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Chimeric antigen receptor (CAR) T‐cell therapy is a breakthrough in hematologic malignancies, such as acute B lymphoblastic leukemia (B‐ALL). Monitoring this treatment is recommended, although standardized protocols have not been developed yet. This work compares two flow cytometry monitoring strategies and correlates this technique with qPCR method. CAR‐T cells were detected by two different flow‐cytometry protocols (A and B) in nine blood samples from one healthy donor and five B‐ALL patients treated with Tisagenlecleucel (Kymriah®, USA). HIV‐1 viral load allowed CAR detection by qPCR, using samples from seven healthy donors and nine B‐ALL patients. CAR detection by protocol A and B did not yield statistically significant differences (1.9% vs. 11.8% CD3 + CAR+, p = 0.07). However, protocol B showed a better discrimination of the CD3 + CAR+ population. A strong correlation was observed between protocol B and qPCR (r = 0.7, p < 0.0001). CD3 + CAR+ cells were detected by flow cytometry only when HIV‐1 viral load was above 104 copies/mL. In conclusion, protocol B was the most specific flow‐cytometry procedure for the identification of CAR‐T cells and showed a high correlation with qPCR. Further efforts are needed to achieve a standardized monitoring approach.

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CAR T-Cell Therapies in Italy: Patient Access Barriers and Recommendations for Health System Solutions

Cited by 12 publications

References 21 publications

Barriers to patient access of CAR T cell therapies in Austria

Barriers to patient access of CAR T cell therapies in Austria

Comparative analysis of CAR T-cell therapy access for DLBCL patients: associated challenges and solutions in the four largest EU countries

The challenge of standardizing CAR‐T cell monitoring: A comparison of two flow‐cytometry methods and correlation with qPCR technique

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