Rose Klaus, Senn Stephen
klausrose Consulting, Switzerland.
Pharm Stat. 2014 Jul-Aug;13(4):211-3. doi: 10.1002/pst.1623. Epub 2014 Jun 6.
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 The class waiver for juvenile melanoma was revoked in 2008 referring to US SEER statistics.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.
欧洲药品管理局（EMA）网站列出了所有仅在成人中正式存在的疾病。2008年，参考美国监测、流行病学和最终结果（SEER）统计数据，青少年黑色素瘤的类别豁免被撤销。这种统计依据具有误导性。青少年黑色素瘤比EMA/儿科委员会声称的要罕见得多；患有黑色素瘤的青少年中，不到四分之一需要进行全身治疗；单独进行疗效研究在医学上既不合理也不可行。稀少的青少年患者应被允许参加成人试验。用1968年 Shirkey 创造的“治疗孤儿”一词来说，迫使公司对他们进行单独研究，会使他们成为儿科的人质。目前有五项黑色素瘤儿科研究计划（PIPs）。可能由PIP引发的临床研究一项都不会完成；我们建议称它们为幽灵研究。一个考虑在黑色素瘤（包括青少年）方面进行合理试验的肿瘤学研究网络，将与由监管狭隘视野引发、在EMA施加的压力下由公司赞助的PIP引发的试验竞争招募患者。EMA/儿科委员会的地域热情（“我们的患者”）损害了肿瘤学研究。
