Defining Essential Childhood Cancer Medicines to Inform Prioritization and Access: Results From an International, Cross-Sectional Survey.
Defining Essential Childhood Cancer Medicines to Inform Prioritization and Access: Results From an International, Cross-Sectional Survey.
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DOI：
10.1200/go.22.00034
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发表时间：
2022-06
影响因子：
4.5
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中科院分区：
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获得基本癌症药物是全球儿童癌症结局的一个主要决定因素。儿科肿瘤学家在多大程度上认为世卫组织《儿童基本药物标准清单》（EMLc）中列出的药物是必不可少的尚不清楚，在临床护理的第一线获得这些药物的程度也不得而知。一项电子调查通过国际儿科肿瘤学会的邮件列表分发给来自87个国家的成员。受访者被要求选择10种癌症药物，在他们的情况下为患者提供最大的好处；随后的问题探讨了药物的可获得性和成本。描述性和双变量统计比较了低收入和中低收入国家（LMICs）、中高收入国家（UMICs）和高收入国家（HICs）之间的药品可及性。在来自44个国家的159名受访者中，43名（27%）来自中低收入国家，79名（50%）来自中低收入国家，37名（23%）来自高收入国家。排在前五位的药物分别是甲氨蝶呤（75%）、长春新碱（74%）、阿霉素（74%）、环磷酰胺（69%）和阿糖胞苷（65%）。在确定的重点药物中，87%（31个中的27个）在2021年EMLc中有代表，77%（31个中的24个）在LMIC、UMIC和HIC受访者生成的清单中常见。表示普遍可获得每种顶级药物的答复者比例在低收入和中等收入国家为9%至46%，低收入和中等收入国家为25%至89%，高收入和中等收入国家为67%至100%。灾难性支出的风险在低收入和中等收入国家中更为常见（8%-20%），而中低收入和中等收入国家（0%-28%）和高收入国家（0%）则不同。大多数肿瘤学家认为对儿童癌症治疗至关重要的药物目前都包括在EMLc中。在获得这些药物方面仍然存在障碍，其特点是可得性方面存在差距，以及家庭灾难性支出的风险，这在低收入环境中最为明显，但在所有收入环境中都很明显。
Access to essential cancer medicines is a major determinant of childhood cancer outcomes globally. The degree to which pediatric oncologists deem medicines listed on WHO's Model List of Essential Medicines for Children (EMLc) essential is unknown, as is the extent to which such medicines are accessible on the front lines of clinical care. An electronic survey developed was distributed through the International Society of Pediatric Oncology mailing list to members from 87 countries. Respondents were asked to select 10 cancer medicines that would provide the greatest benefit to patients in their context; subsequent questions explored medicine availability and cost. Descriptive and bivariate statistics compared access to medicines between low- and lower-middle–income countries (LMICs), upper-middle–income countries (UMICs), and high-income countries (HICs). Among 159 respondents from 44 countries, 43 (27%) were from LMICs, 79 (50%) from UMICs, and 37 (23%) from HICs. The top five medicines were methotrexate (75%), vincristine (74%), doxorubicin (74%), cyclophosphamide (69%), and cytarabine (65%). Of the priority medicines identified, 87% (27 of 31) are represented on the 2021 EMLc and 77% (24 of 31) were common to the lists generated by LMIC, UMIC, and HIC respondents. The proportion of respondents indicating universal availability for each of the top medicines ranged from 9% to 46% for LMIC, 25% to 89% for UMIC, and 67% to 100% for HIC. Risk of catastrophic expenditure was more common in LMIC (8%-20%), compared with UMIC (0%-28%) and HIC (0%). Most medicines that oncologists deem essential for childhood cancer treatment are currently included on the EMLc. Barriers remain in access to these medicines, characterized by gaps in availability and risks of catastrophic expenditure for families that are most pronounced in low-income settings but evident across all income contexts.