Pediatric Pharmaceutical Legislation in the USA and EU and Their Impact on Adult and Pediatric Drug Development

Abstract

Drug development is a complex undertaking, not an academic exercise. The key players, mainly pharmaceutical industry, regulatory authorities and academia, have different logics and interests. Industry consists of large, medium, and small companies who compete (or cooperate), win or fail. For some decades large companies seemed to set the tone in drug development, but that paradigm may be changing. Pediatric legislation has imposed the logic of public health over this already complex process. The intention is certainly laudable. The key question is if it works, and to what degree the US and EU legislation are comparable. The breakthrough improvements in pediatric oncology in the last decades happened without direct contribution from regulatory authorities. The successful treatment schemes for children with cancer are off-label and will remain so. Breakthrough innovations in rare pediatric diseases such as cystic fibrosis or enzyme deficiencies were not triggered by pediatric legislation. The number of label changes, of submitted pediatric investigation plans (PIPs), or of clinical trials that companies must commit to have in themselves limited significance. Do all label changes improve child treatment? Do trials in rare diseases make sense if there are not enough patients on this planet? Does the interference of the European Medicines Agency (EMA) and its pediatric committee (PDCO) in worldwide research in rare pediatric diseases promote child health, or does it harm? At the end, the reader will have to answer these questions for himself. A framework is offered for guidance through the maze of dimensions that need to be taken into consideration.