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The Price of Survival: Global Inequities in Anti‐GD2 Immunotherapy for Neuroblastoma

医学 神经母细胞瘤 免疫疗法 谈判 肿瘤科 梅德林 全球战略 免疫学 重症监护医学 全球卫生 抗体疗法 药品 药品审批 接种疫苗 血癌 内科学
作者
Ruzanna Papyan,Julieta Hoveyan,Martin Guscott,Jennifer Lowe,Gabriella Villanueva,Keri A. Streby,Emma Tadevosyan,Ester Demirtshyan,Jennifer Hylton,G. Tamamyan,Catherine G. Lam,Katherine K. Matthay,Scott C. Howard
出处
期刊:Pediatric Blood & Cancer [Wiley]
卷期号:73 (4): e70152-e70152
标识
DOI:10.1002/1545-5017.70152
摘要

BACKGROUND: Long-term survival for high-risk neuroblastoma has increased from 40% to 60% by optimizing chemotherapy, surgery, radiation therapy, and the addition of anti-GD2 antibody therapy. However, the high cost of this antibody presents access issues globally. This study evaluates the availability, financial coverage, and barriers to accessing these therapies across diverse healthcare settings. METHODS: An online survey collected data on demographics, treatment practices, financial coverage, regulatory status, and barriers to anti-GD2 therapy. Fisher's exact test was used to compare categorical variables between high-income countries (HICs) and low- and middle-income countries (LMICs). A retrospective cost analysis estimated the financial burden of anti-GD2 therapies using standardized dosing for a representative 3-year-old patient. RESULTS: Responses came from 100 facilities in 80 cities and 59 countries. Anti-GD2 therapy for frontline maintenance was available in 93% of HIC centers, but only 21% of LMIC centers; 65% of LMIC centers reported no access, while the remainder had limited or irregular availability (p < 0.0001, Fisher's exact test). When unavailable, isotretinoin was most often used alone. Financial coverage differed significantly: HICs relied on government or insurance funding, while LMICs depended on out-of-pocket or non-profit support. Cost analysis showed a full treatment course would cost approximately $192,750 for dinutuximab, $142,695 for dinutuximab beta, and $610,800 for naxitamab, highlighting the substantial financial burden these therapies impose. CONCLUSION: Global access to anti-GD2 therapy is highly unequal. Strategic measures, including negotiations for reduced drug costs, research-based access, and WHO Essential Medicines List inclusion, could help address these disparities.
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