Retinoblastoma, a rare but aggressive eye cancer in infants and young children, accounts for 2% to 4% of pediatric malignancies globally, with an incidence rate ranging from 1 in 20 000 to 1 in 15 000 children. Approximately 8000 new cases are reported annually across the globe, of which more than 1000 are reported in China. Notably, regions with the highest prevalence, such as Asia and Africa, have the highest mortality rates, ranging from 40% to 70%, in stark contrast with the 3% to 5% mortality rate in high-income countries. According to the definition of rare cancers from the European RARECARE project (<6/100 000 people), retinoblastoma is considered a rare cancer with significant unmet clinical needs owing to the limited information available regarding its diagnosis and treatment. In 2018, the National Health Commission of China announced the “catalog for the first batch of rare diseases”; retinoblastoma appeared in this list, indicating its priority for policymakers in promoting the treatment of retinoblastoma. As a rare cancer, the number of clinical trials conducted for retinoblastoma is limited, particularly in low- and middle-income countries. Most of the existing research originates from high-income countries. However, high-income countries account for less than 10% of the global retinoblastoma cases, while low- and middle-income countries in Asia, Africa, and Latin America represent more than 80% of the cases.
