Implementing Good Clinical Practice in Two Noncommercial Phase II Studies in Children with Cancer

Abstract

In noncommercial clinical drug research the implementation of the principles of Good Clinical Practice (GCP) has been criticized for introducing unnecessary bureaucracy at the expense of scientific activity, especially when small populations such as children or patients with orphan diseases are concerned. Patients and Methods: From May 2003 to September 2005, we conducted two prospective open-label multicenter phase II studies in pediatric oncology. Aside from medical questions, these studies set out to explore the requirements according to the essential standards of the ICH-GCP Guideline in anticipation of the implementation of the EU Regulation in German Drug Law in August 2004; the latter, prospective investigation was initiated by the Coordinating Center for Clinical Trials (KKS). Results: The main GCP requirements were systematically reviewed and critically discussed by focusing mainly on the situation of noncommercial pediatric drug research. Conclusion: While putting GCP into practice in academic research increases costs, the challenge will be to apply these guidelines to good effect, for better quality and increased evidence. Implementing Good Clinical Practice in Two Noncommercial Phase II Studies in Children with Cancer Alexandra Wagner-Bohna Anita Ripkens-Reinharda Gerlinde Benninger-Döringa Joachim Boosb a Coordinating Centre for Clinical Trials, University Hospital of Münster, bDepartment of Pediatric Hematology and Oncology, University Children’s Hospital of Münster, Germany

Why do we need noncommercial, investigator-initiated clinical trials?

“…A recently reported noncommercial phase II IIT in the challenging area of pediatric oncology shows that, with commitment, an experienced, academically based clinical trials unit, with government and private (the Lilly Foundation) funding as well as an industry supply of the medicinal product under investigation (gemcitabine), can perform a GCP-standard trial. 8 Although the drug is useful in adults, the IIT in children was conclusively negative, thus avoiding unnecessary patient exposure in the future.…”