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Essential cancer medicines, resource-stratified guidelines, and cancer care implementation in low- and middle-income countries

Year: 2025
Type: editorial
Abstract: The purpose of the Essential Medicines List (EML) first conceived by the World Health Organization (WHO) in the 1970s is to provide guidance to Member States (countries) on which medicines are necessary to provide effective treatment for patients with common diseases. Dr Halfdan Mahler, the former WHO Director General, warned in 1975 at the World Health Assembly of the “urgent need to ensure that most essential drugs are available at a reasonable price,”1 leading to the publication of WHO’s first EML 2 years later. Several resulting medical success stories have included widespread access to affordable medicines for HIV, hepatitis C, and tuberculosis. By contrast, the selection of medicines to treat cancer guided by the EML has been more challenging due to the complexity of cancer treatment and high cost of the most efficacious therapies. Some cancer medicines are highly effective but are also extraordinarily expensive and threaten to “break the bank” for low- and middle-income countries (LMICs). First published in 2006 by the Breast Health Global Initiative (BHGI) for breast cancer management, resource-stratified guidelines (RSGs) provide an evidence-based framework to identify systematic gaps in breast cancer early detection, prompt diagnosis, and treatment to completion to guide investment for program development in LMICs based on optimal use of existing sustainable resources.2 Since 2006, other groups have established resource-stratification guidelines for other malignancies including the National Comprehensive Cancer Network (NCCN), the American Society of Clinical Oncology (ASCO), and WHO through the Global Breast Cancer Initiative (GBCI).3,4 A logical next step is to assess how WHO’s EML and other organizations’ RSGs align with each other to outline sustainable strategies for successfully treating all cancer in LMICs. In the current issue of the Journal, Wilson and colleagues provide an important and valuable analysis of the relationship between the WHO Essential Medicines List (EML) for cancer in comparison to two sets of resource-stratified guidelines (RSGs), one from the National Cancer Grid (NCG) of India5 and the other from the National Comprehensive Cancer Network (NCCN).6 This analysis examines how the 2023 EML, which includes 64 cancer medicines for 219 tumor-specific indications, can best be aligned with RSGs to guide the treatment of more cancer patients globally using effective drug regimens and providing guidance on how each may potentially be better applied in real-world limited resource settings. Although some common cancers (eg, hepatocellular carcinoma) have no EML-listed medicines due to low clinical benefit, Wilson and colleagues identified other cancers (eg, esophageal and gastric) with effective systemic therapies that should be considered for EML inclusion. Among the EML-listed cancer medicines, 42% (35/84) and 73% (62/84) are called for on the NCCN Basic and Core Guidelines,7 respectively. For comparison, 163/196 (83%) and 175/196 (89%) are called for on the NCG Essential and Optimal RSGs, respectively. Importantly, cancers with no listed medicines on the 2023 EML are responsible for 34% of cancer deaths globally. If the additional systemic therapies identified in the Wilson et al. study were added to the EML, the proportion of patients that could be treated effectively would rise from approximately 70% to 84% of patients with a cancer diagnosis. The number of deaths attributable to cancers with no recommended medicines on the EML would correspondingly fall from 3.0 million to 1.6 million. These data provide a strong impetus for future EML iterations to examine how overall cancer coverage can be improved in given resource-constrained settings. A limitation of WHO’s EML is that it lists medications as single agents rather than combination regimens. For infectious diseases and many noncommunicable diseases, single-agent therapies are common and useful. By contrast, few cancers are treated with single agents—the great majority of effective cancer treatments are based on combination drug regimens based on decades of randomized trials. The current EML does not provide guidance on how and when EML medicines should be used in practice, which itself requires a level of oncological expertise that may be out of scope for a UN agency. Wilson and colleagues opine that future iterations of the EML should consider if and how the recommended drugs are typically combined in treatment regimens. Noting that the EML is only updated every 2 years, it would be unrealistic for the EML to remain up-to-date on all cancer treatment regimens. However, it could be useful to Ministries of Health if the EML could provide more specific guidance on what resources are required to make specific EML medications applicable. For example, trastuzumab was added to the EML because of its extraordinary efficacy in improving outcomes for HER2/neu positive cancers. However, the utility of this targeted biological therapy hinges on it being combined with cytotoxic chemotherapy to be effective; without chemotherapy, the efficacy of trastuzumab is limited. Given that the toxicity of cancer medicines is a major barrier for patients completing their cancer treatments, the value of supportive care medicines such as antiemetics and other supportive services cannot be overemphasized.8 Drug availability is a critical topic, but equally important is how the medicines are used in practice. In sub-Saharan Africa, only 50% of patients who begin systemic treatment within 3 months of diagnosis complete it.9 Although the causes of treatment abandonment are varied and complex, failure to complete systemic treatment protocols is the worst of all worlds where the patient suffers treatment toxicity but fails to receive the predicted clinical benefit. Improved methods are needed globally to assess the degree to which patients complete, not just initiate, treatment. Benjamin O. Anderson (Conceptualization, Writing—original draft) None declared. Dr Anderson has received consulting fees and travel support from AstraZeneca. No new data were generated or analyzed for this Editorial.
DOI: 10.1093/jnci/djaf203
Language: English
Open Access status: bronze
OpenAlex ID: W4414003283
Cites: 5
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Related to: 10