title: [New drugs in oncology--features of clinical trials for market authorisation and arguments for the rapid implementation of independent clinical trials following approval].
abstract: The market authorisation or extension of indication for all oncology drugs in Europe is now based on Regulation (EC) No. 726/2004, a centralised procedure of the European Medicines Agency (EMA). Studies in recent years have highlighted deficiencies in pivotal studies. For example, the requirements of the EMA are not always consistently followed and studies are stopped prematurely after only interim analysis that at this time point shows improved efficacy with regard to the comparator arm. Our current analysis of the European Assessment Reports (reporting period: 01/01/2009 to 08/13/2012) on 29 drugs for 39 oncology indications shows that the quality of the trials for market authorisation has improved in several respects. Primary endpoints recommended by the EMA and the Food and Drug Administration (FDA) such as overall survival and progression-free survival are used, and only one study was conducted as a phase II trial with no comparator arm. In contrast, oncology drugs that are approved for the treatment of rare diseases (orphan drugs) are based on small studies which are often carried out without blinding, are not randomised and investigate surrogate endpoints. To answer patient-relevant issues following market authorisation, it is necessary to conduct independent clinical studies. Increased public funding needs to be provided and bureaucratic hurdles have to be reduced. Only this will permit a more efficient use of limited health care resources and allow to improve the quality of care for cancer patients.
authors.affiliation: Arzneimittelkommission der deutschen Ärzteschaft (AkdÄ), Berlin, Deutschland. wolf-dieter.ludwig@akdae.de
authors.forename: Wolf-Dieter
authors.initials: WD
authors.lastname: Ludwig
authors.forename: Gisela
authors.initials: G
authors.lastname: Schott
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D017277:Drug Approval / Q000458:organization & administration*; D005060:Europe; D008389:Marketing of Health Services / Q000458:organization & administration*; D008495:Medical Oncology / Q000458:organization & administration*
chemical_list: D000970:Antineoplastic Agents