Clinical studies evolved with modern drug development. When penicillin was produced industrially a first time during World War II, complicated studies were unnecessary. Patients with bacterial infections were literally snatched from their deathbeds by penicillin. Drug development accelerated, and the range of treatable diseases expanded. New antibiotics and drugs for pain, insomnia, diabetes, birth control, & more emerged. A German company developed and marketed thalidomide for all sorts of ailments, including insomnia, nervousness, and nausea in pregnancy. In the late 1950s, it was discovered that it had caused thousands of severe birth defects worldwide. Never US-approved (but in Canada), it became a turning point in drug development. From 1962 on, US law demanded studies for new drugs. Back in the 1950s, toxicities had been reported in babies treated with antibiotics. To avoid damage lawsuits in the litigious US, companies now inserted “pediatric warnings” into drug labels. This, in turn, led to a concept that claimed all children were “therapeutic orphans”.
Legitimate concerns for babies became the narrative that all drugs are dangerous in all children if not tested separately. The children-are-not-small-adults and children-are-therapeutic-orphans mantras invaded medicine, science, and politics. Confidence in science was high. US lawmakers decided to reward pediatric studies by patent extensions. Companies could sell drugs longer at a higher price. And here the big challenge began.
No need to be a doctor to know that 15-year-old teenagers who are bigger and stronger than mom or dad are bodily mature. No need to be a shrink to know that the mind is usually lagging behind. The FDA defined “children” first by below 17, today below 18 years. But drugs treat the body, not the legal status. “Pediatric” studies in minors, including bodily mature adolescents, became big business for researchers. All scientists complain about a lack of funds. For “pediatric” research, a bonanza started. It became worse when the European Union (EU) expanded the obligation for “pediatric” studies. Today, studies are performed worldwide. Also American children are threatened by EU-demanded studies. Industry paid pediatric researchers for studies. They were futile in adolescents, massively exaggerated in young children. Minors don’t remain as vulnerable as newborns until they come of age. Thousands of questionable “pediatric” studies are today performed worldwide.
The opinion that most pediatric studies are excessive is not shared by most medical and pharmaceutical professionals worldwide. In fact, I am one of very few. But I know what I am talking about. I am a medical doctor, was global head pediatrics in two large international pharma companies, am independent now, and publish & write books about this challenge.
The definition of children as under 18 is a legal, not a medical one. There are no drugs that work in adults and don’t work in children. Kids are not another species.
In my blogs, I will address two sides. I will warn parents that are asked to let their child participate in a study. Studies in minors can safe lives, or can harm. And I offer online seminars for pharmaceutical companies where I explain how to push back against potentially harmful studies demanded by the FDA and the European Medicines Agency (EMA).