How Should We Determine the Value of CAR T-Cell Therapy?

Silbert, Sara; Yanik, Gregory A.; Shuman, Andrew G.

doi:10.1001/amajethics.2019.844

Cited by 11 publications

(2 citation statements)

References 9 publications

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“…DAF considerations : With higher payer willingness to pay thresholds comes the possibility that developers will be motivated to inflate the price of GTs to meet the ICER threshold ( 52 , 53 ), in other words, the developers will charge the price as high as the payer are able to endure ( 26 ). It is unlikely that payers will accept innovative value-pricing methods, considering that they must be implemented with a radical modification of current policy.…”

Section: Economic Analysismentioning

confidence: 99%

Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment

Qiu

Pochopien²,

Liang

et al. 2022

Front. Public Health

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Gene therapies (GTs) are considered to be a paradigm-shifting class of treatments with the potential to treat previously incurable diseases or those with significant unmet treatment needs. However, considerable challenges remain in their health technology assessment (HTA), mainly stemming from the inability to perform robust clinical trials to convince decision-makers to pay the high prices for the potential long-term treatment benefits provided. This article aims to review the recommendations that have been published for evidence generation and economic analysis for GTs against the feasibility of their implementation within current HTA decision analysis frameworks. After reviewing the systematically identified literature, we found that questions remain on the appropriateness of GT evidence generation, considering that additional, broader values brought by GTs seem insufficiently incorporated within proposed analytic methods. In cases where innovative methods are proposed, HTA organizations remain highly conservative and resistant to change their reference case and decision analysis framework. Such resistances are largely attributed to the substantial evidence uncertainty, resource-consuming administration process, and the absence of consensus on the optimized methodology to balance all the advantages and potential pitfalls of GTs.

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Section: Economic Analysismentioning

confidence: 99%

Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment

Qiu

Pochopien²,

Liang

et al. 2022

Front. Public Health

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“…To date, scarce research has quantified real costs attributable to CAR T-cell therapy, which is crucial in drug reimbursement discussion between health players. Country-specific economic evaluations are necessary to determine whether and how to offer patients these highly personalized forms of immunotherapy [11,[20][21][22][23][24].…”

Section: Introductionmentioning

confidence: 99%

Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center

et al. 2022

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Real world effectiveness, toxicity and costs analyses from chimeric antigen receptor (CAR)-T cell therapy are of utmost relevance to determine whether and how to offer patients highly personalized immunotherapy. In this study, we aimed at describing CAR T-cells effectiveness, safety and costs in a Portuguese Comprehensive Cancer Center. We performed a retrospective descriptive study of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma and transformed follicular lymphoma referred to CAR T-cell therapy, between May 2019 and February 2021. Rates of treatment response, toxicity and survival (Kaplan-Meier method) were analyzed by intention-to-treat. Direct medical costs stratified by inpatient-care, outpatient-care, and diagnostic-therapeutic procedures (DTP) were derived based on resources used and their respective unit costs. In twenty patients (median age 49.5y; 55%male; 70%DLBCL; 50% with primary refractory disease), best overall and complete response rates were 65.0% and 45.0%, respectively. Median overall (OS) and progression-free survivals were 9.2 and 7.3 months; 12-month OS rate was 42.6% (95%CI:23.2–78.3). Grade≥3 cytokine release syndrome and neurotoxicity occurred in 5.6% and 11.1% of patients, respectively. CAR T-cell therapy expenditure, including adverse events costs, was 7 176 196€, or 286 238€ when excluding drug cost. Median cost for treated patient was 355 165€ with CAR T-cell drug cost accounting for 97.0% of the overall expense. Excluding CAR T-cell acquisition cost, inpatient-care and DTP accounted for 57% and 38% of total cost/patient, respectively. Our findings highlight the heavy economic burden of CAR T-cell therapy driven by drug acquisition costs.

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Dürfen Gentherapien so viel kosten? Ethische Bewertung der hohen Preise und des performanceorientierten Erstattungsmodells

Alex,

König

2023

Gen- Und Zelltherapie 2.023 - Forschung, Klinische Anwendung Und Gesellschaft

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ZusammenfassungIn diesem Kapitel wird untersucht, ob hohe Preise für Gentherapien gerechtfertigt sind und ob die Problematik, die mit hohen Preisen verbunden ist, durch das Erstattungsmodell „pay for performance“ (P4P) behoben werden kann. Dazu wird zunächst beschrieben, wie Preise für neue Arzneimittel, zu denen auch Gentherapien zählen, in Deutschland festgelegt werden (Abschn. 22.2). Danach wird P4P als Beispiel für ein Erstattungsmodell vorgestellt (Abschn. 22.3).

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How Should We Determine the Value of CAR T-Cell Therapy?

Cited by 11 publications

References 9 publications

Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment

Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment

Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center

Dürfen Gentherapien so viel kosten? Ethische Bewertung der hohen Preise und des performanceorientierten Erstattungsmodells

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