Defining Essential Childhood Cancer Medicines to Inform Prioritization and Access: Results From an International, Cross-Sectional Survey

Abstract

PURPOSE 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. METHODS 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). RESULTS 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%). CONCLUSION 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.

Evidence of clinical benefit of WHO essential anticancer medicines for children, 2011–2021

“…According to a recent survey, the majority of anticancer medicines deemed essential by paediatric oncologists have been included in the current WHO EMLc. 7 Nevertheless, despite the emphasis on clinical evidence in children, the WHO EMLc has had limited ability to list anticancer medicine indications with traditionally defined high-quality clinical evidence, reflecting the limited availability of clinical trials in children. 14 , 15 From 2015 to 2021, 14 of the anticancer medicine indications added in the EMLc had no improvement on either OS or surrogate measures according to their highest level of documented evidence in children cited in the WHO EMLc technical reports.…”

“…The long-term comparative clinical benefit in children of most modern targeted therapies with high costs remains uncertain, which are thus rarely regarded as high-priority medicines by paediatric oncologists. 7 Further deliberation is necessary when selecting targeted therapies in WHO and national essential medicine lists.…”

“… 4 , 5 As pharmaceutical therapy plays a critical role in childhood cancer treatment, access to essential anticancer medicines is a key determinant of childhood cancer survival. 6 , 7 …”

Alignment in the registration, selection, procurement and reimbursement of essential medicines for childhood cancers in South Africa

“…While our findings also indicate alignment with international processes, the few gaps in comparison to the WHO EMLc may have a big impact. In fact, in a 2022 cross-sectional survey to determine priority essential childhood cancer medicines, dactinomycin was in the top 10 of most frequently selected drugs by paediatric oncologists when asked what medicines would achieve greatest benefit in children 28. Thus, the lack of market authorisation for dactinomycin in SA indicates that deficiencies in therapeutic care exist.…”

“…Additionally, even if some products were eventually successfully awarded in an additional tender for 2020–2022, this supplementary round brings a considerable delay of about 4 months based on the tender documents. These delays also affected core chemotherapeutics such as doxorubicin and vincristine 28. Noteworthy, DoH’s price expectations were unchanged for the additional tender round.…”