title: Drug development: EU paediatric legislation, the European Medicines Agency and its Paediatric Committee--adolescents' melanoma as a paradigm.
abstract: The European Medicines Agency (EMA) website lists all diseases that officially exist in adults only. The class waiver for juvenile melanoma was revoked in 2008 referring to US SEER statistics. This statistical justification is misleading. Melanoma in adolescents is much rarer than claimed by EMA/Paediatric Committee; < 1 ∕ 4 of adolescents with melanoma need systemic treatment; separate efficacy studies are neither medically justified nor feasible. The scarce adolescent patients should be allowed to participate in adult trials. To force companies to investigate them separately turns them into paediatric hostages, to adapt the term therapeutic orphans coined in 1968 by Shirkey. There are now five melanoma Paediatric Investigation Plans (PIPs). Probably none of the PIP-triggered clinical studies will ever be completed; we propose to call them ghost studies. An oncology research network considering a reasonable trial in melanoma, including adolescents, will compete for recruitment with the PIP-triggered trials designed by regulatory tunnel vision and sponsored by companies under EMA-imposed pressure. EMA/Paediatric Committee's territorial enthusiasm ("our patients") damages oncology research.
authors.affiliation: klausrose Consulting, Switzerland.
authors.forename: Klaus
authors.initials: K
authors.lastname: Rose
authors.forename: Stephen
authors.initials: S
authors.lastname: Senn
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002986:Clinical Trials as Topic; D017277:Drug Approval / Q000331:legislation & jurisprudence*; D005062:European Union; D006801:Humans; D008545:Melanoma / Q000188:drug therapy*; D018579:Patient Selection; D010372:Pediatrics / Q000331:legislation & jurisprudence*
keywords: Melanoma; Paediatric Drug Development; Paediatric Investigation Plan (PIP)