Questionable International Pediatric Studies in the United States and Russia Triggered by Regulatory Authorities

Background: The concept of children as "therapeutic orphans" claims that children were/are denied the use of many modern drugs. Both the United States (US) and the European Union (EU) enacted laws based on this concept. Their regulatory authorities promote industry-sponsored pediatric studies. These studies recruit worldwide. We challenge their medical rationale.

Methods: We analyzed exemplarily international industry-sponsored pediatric studies in cancer and rheumatology listed in www.clinicaltrials.gov with at least one center in the US and Russia, respectively, for their medical value.

Findings: Most studies were/are pharmacokinetic (PK) and efficacy studies in young patients with limited or no medical value. Adolescents are physiologically (vis-à-vis drug metabolism) comparable to adults; for children only PK- and dose finding studies are necessary. Only newborns'/babies' organs are physiologically so different that separate proof of efficacy is needed for drugs with a therapeutic potential in this population. The identified studies were/are justified formally, regulatorily, but are medically unnecessary and therefore unethical. Parts of pediatric academia are misled by industry funds channeled by regulatory decisions into medically questionable studies. There are resulting substantial conflicts of interest; a blind spot in today's societal perception of drug development prevents us from recognizing them.

Interpretation: Pediatric studies triggered by regulatory demands constitute a worldwide systematic abuse of young patients. They are medically redundant at best, deter patients with lethal diseases participating in these studies from getting access to known effective innovative therapy, and have the potential to jeopardize public trust in science, research and authorities. Institutional Review Boards (IRBs)/ ethics committees (ECs) should become alerted. IRBs/ECs worldwide should suspend questionable pediatric studies and reject newly submitted ones. US and EU pediatric laws need revision.