title: The contributions of the European Medicines Agency and its pediatric committee to the fight against childhood leukemia.
abstract: BACKGROUND
  Although the diagnosis of childhood leukemia is no longer a death sentence, too many patients still die, more with acute myeloid leukemia than with acute lymphoblastic leukemia. The European Union pediatric legislation was introduced to improve pharmaceutical treatment of children, but some question whether the European Medicines Agency (EMA) approach is helping children with leukemia. Some have even suggested that the decisions of EMA pediatric committee (PDCO) are counterproductive. This study was designed to investigate the impact of PDCO-issued pediatric investigation plans (PIPs) for leukemia drugs.
  
  
  METHODS
  All PIPs listed under "oncology" were downloaded from the EMA website. Non-leukemia decisions including misclassifications, waivers (no PIP), and solid tumors were discarded. The leukemia decisions were analyzed, compared to pediatric leukemia trials in the database http://www.clinicaltrials.gov, and discussed in the light of current literature.
  
  
  RESULTS
  The PDCO leukemia decisions demand clinical trials in pediatric leukemia for all new adult drugs without prioritization. However, because leukemia in children is different and much rarer than in adults, these decisions have resulted in proposed studies that are scientifically and ethically questionable. They are also unnecessary, since once promising new compounds are approved for adults, more appropriate, prioritized pediatric leukemia trials are initiated worldwide without PDCO involvement.
  
  
  CONCLUSIONS
  EMA/PDCO leukemia PIPs do little to advance the treatment of childhood leukemia. The unintended negative effects of the flawed EMA/PDCO's standardized requesting of non-prioritized testing of every new adult leukemia drug in children with relapsed or refractory disease expose these children to questionable trials, and could undermine public trust in pediatric clinical research. Institutions, investigators, and ethics committees/institutional review boards need to be skeptical of trials triggered by PDCO. New, better ways to facilitate drug development for pediatric leukemia are needed.
authors.affiliation: klausrose Consulting, Pediatric Drug Development and More, Riehen, Switzerland.
authors.forename: Klaus
authors.initials: K
authors.lastname: Rose
authors.affiliation: Department of Clinical Pharmacology, University Medical School, Goettingen, Germany.
authors.forename: Philip D
authors.initials: PD
authors.lastname: Walson
keywords: better medicines for children; childhood leukemia; ghost studies; pediatric drug development; pediatric investigation plan; therapeutic hostages; therapeutic orphans