Melanoma and pediatric drug development: clinical progress vs. regulatory activism in minors - a narrative review

Introduction: Melanoma is the malignancy where, in recent years, drug treatment has massively improved prognosis and quality of life. Based on genetic analysis, we differentiate today melanomas caused by cumulative solar damage (CSD) from others. Areas covered: Conventional CSD-caused melanomas affect predominantly adults and occasionally adolescents. Spitz melanoma and melanoma arising in congenital nevi, the two other pediatric melanoma types, are not CSD-caused, are genetically different, have different prognoses, and need different treatment. In contrast to the improved situation in adult melanoma, regulatory demand for pediatric labels in minors has resulted in pointless and harmful studies and has obfuscated diagnosis and treatment. Modern communication facilitates a worldwide analysis of extremely rare diseases such as melanoma in minors. Regulatory demand for on-label treatment only is demonstrably wrong for pediatric melanomas. They are too rare for randomized controlled trials but nevertheless deserve effective treatment. Expert opinion: Adolescents with conventional, CSD-caused melanoma should be treated as adults. Their bodies are already mature. For other childhood melanomas, registries and consultations with clinical specialists are better options than dogmatically demanded regulatory studies.