Background

The WHO Essential Medicines List (EML) identifies priority medicines that are most important to public health. Over time, the EML has included an increasing number of cancer medicines. We aimed to investigate whether the cancer medicines in the EML are aligned with the priority medicines of frontline oncologists worldwide, and the extent to which these medicines are accessible in routine clinical practice.

Methods

This international, cross-sectional survey was developed by investigators from a range of clinical practice settings across low-income to high-income countries, including members of the WHO Essential Medicines Cancer Working Group. A 28-question electronic survey was developed and disseminated to a global network of oncologists in 89 countries and regions by use of a hierarchical snowball method; each primary contact distributed the survey through their national and regional oncology associations or personal networks. The survey was open from Oct 15 to Dec 7, 2020. Fully qualified physicians who prescribe systemic anticancer therapy to adults were eligible to participate in the survey. The primary question asked respondents to select the ten cancer medicines that would provide the greatest public health benefit to their country; subsequent questions explored availability and cost of cancer medicines. Descriptive statistics were used to compare access to medicines between low-income and lower-middle-income countries, upper-middle-income countries, and high-income countries.

Findings

87 country-level contacts and two regional networks were invited to participate in the survey; 46 (52%) accepted the invitation and distributed the survey. 1697 respondents opened the survey link; 423 were excluded as they did not answer the primary study question and 326 were excluded because of ineligibility. 948 eligible oncologists from 82 countries completed the survey (165 [17%] in low-income and lower-middle-income countries, 165 [17%] in upper-middle-income countries, and 618 [65%] in high-income countries). The most commonly selected medicines were doxorubicin (by 499 [53%] of 948 respondents), cisplatin (by 470 [50%]), paclitaxel (by 423 [45%]), pembrolizumab (by 414 [44%]), trastuzumab (by 402 [42%]), carboplatin (by 390 [41%]), and 5-fluorouracil (by 386 [41%]). Of the 20 most frequently selected high-priority cancer medicines, 19 (95%) are currently on the WHO EML; 12 (60%) were cytotoxic agents and 13 (65%) were granted US Food and Drug Administration regulatory approval before 2000. The proportion of respondents indicating universal availability of each top 20 medication was 9–54% in low-income and lower-middle-income countries, 13–90% in upper-middle-income countries, and 68–94% in high-income countries. The risk of catastrophic expenditure (spending >40% of total consumption net of spending on food) was more common in low-income and lower-middle-income countries, with 13–68% of respondents indicating a substantial risk of catastrophic expenditures for each of the top 20 medications in lower-middle-income countries versus 2–41% of respondents in upper-middle-income countries and 0–9% in high-income countries.

Interpretation

These data demonstrate major barriers in access to core cancer medicines worldwide. These findings challenge the feasibility of adding additional expensive cancer medicines to the EML. There is an urgent need for global and country-level policy action to ensure patients with cancer globally have access to high priority medicines.

Funding

None.

中文翻译：

---

82 个国家的肿瘤学家认为获得癌症药物至关重要：一项国际横断面调查

背景

世界卫生组织基本药物清单 (EML) 确定了对公共卫生最重要的优先药物。随着时间的推移，EML 纳入了越来越多的癌症药物。我们的目的是调查 EML 中的癌症药物是否与全球一线肿瘤学家的优先药物一致，以及这些药物在常规临床实践中的可获得程度。

方法

这项国际横断面调查是由来自低收入国家和高收入国家的一系列临床实践机构的研究人员开展的，其中包括世界卫生组织基本药物癌症工作组的成员。开发了一项包含 28 个问题的电子调查，并通过分层滚雪球法向 89 个国家和地区的全球肿瘤学家网络传播；每个主要联系人通过其国家和区域肿瘤学协会或个人网络分发调查问卷。该调查于2020年10月15日至12月7日开放。为成人进行全身抗癌治疗的完全合格的医生有资格参加该调查。主要问题要求受访者选择十种能够为其国家带来最大公共卫生效益的癌症药物；随后的问题探讨了癌症药物的可用性和成本。使用描述性统计数据来比较低收入和中低收入国家、中高收入国家和高收入国家的药品获取情况。

发现

87 个国家级联系人和两个区域网络受邀参加调查；46 人（52%）接受了邀请并分发了调查问卷。1697名受访者打开了调查链接；423 人因未回答主要研究问题而被排除，326 人因不符合资格而被排除。来自 82 个国家的 948 名符合资格的肿瘤学家完成了调查（低收入和中低收入国家有 165 名 [17%]，中高收入国家有 165 名 [17%]，高收入国家有 618 名 [65%]国家）。最常选择的药物是阿霉素（948 名受访者中的 499 名 [53%]）、顺铂（470 名 [50%]）、紫杉醇（423 名 [45%]）、派姆单抗（414 名 [44%]）、曲妥珠单抗（402 [42%]）、卡铂（390 [41%]）和 5-氟尿嘧啶（386 [41%]）。在 20 种最常选择的高优先级癌症药物中，有 19 种（95%）目前已列入 WHO EML；12 种 (60%) 是细胞毒性药物，13 种 (65%) 在 2000 年之前获得了美国食品和药物管理局监管批准。在低收入和低收入人群中，表示普遍可获得前 20 种药物的受访者比例为 9-54%。中等收入国家为 13-90%，中高收入国家为 13-90%，高收入国家为 68-94%。灾难性支出的风险（支出占总消费净额的 40% 以上，扣除食品支出）在低收入和中低收入国家更为常见，13-68% 的受访者表示每个国家都存在巨大的灾难性支出风险。中低收入国家中 20 种前 20 种药物的受访者比例为 2-41%，高收入国家中为 0-9%。

解释

这些数据表明全球范围内获取核心癌症药物的主要障碍。这些发现对在 EML 中添加额外昂贵的癌症药物的可行性提出了挑战。迫切需要采取全球和国家层面的政策行动，以确保全球癌症患者能够获得高度优先的药物。

资金

没有任何。