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

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.
Competing Interests: Scott C. HowardConsulting or Advisory Role: Sanofi, BTG, ServierSpeakers' Bureau: Sanofi, Jazz Pharmaceuticals Dorothy LombeThis author is a member of the Journal of Global Oncology Editorial Board. Journal policy recused the author from having any role in the peer review of this manuscript. Bishal GyawaliThis author is a member of the Journal of Global Oncology Editorial Board. Journal policy recused the author from having any role in the peer review of this manuscript.Consulting or Advisory Role: Vivio Health Felipe RoitbergHonoraria: Boehringer Ingelheim, AstraZeneca, Oncologia BrasilConsulting or Advisory Role: MSD OncologyResearch Funding: Roche (Inst), Boehringer Ingelheim (Inst), MSD (Inst), Bayer (Inst), AstraZeneca (Inst), Takeda (Inst) Elisabeth G. E. De VriesConsulting or Advisory Role: Daiichi Sankyo (Inst), NSABP Foundation (Inst), Crescendo Biologics (Inst)Research Funding: Amgen (Inst), Synthon (Inst), CytomX Therapeutics (Inst), Regeneron (Inst), G1 Therapeutics (Inst), Bayer (Inst), Roche (Inst), Genentech (Inst), Servier (Inst), Crescendo Biologics (Inst)Uncompensated Relationships: ESMO (Inst)Uncompensated Relationships: World Health Organization (Inst)Uncompensated Relationships: RECIST (Inst) Richard SullivanThis author is a member of the Journal of Global Oncology Editorial Board. Journal policy recused the author from having any role in the peer review of this manuscript.Honoraria: PfizerConsulting or Advisory Role: Pfizer (Inst)No other potential conflicts of interest were reported.