When the children-are-not-small-adults and the children-are-therapeutic-orphans mantras became US law in 1997, there was broad social consensus that in medical treatment everything is different in children compared to adults. The key catch in this concept is that newborn babies are indeed different from adults, but they grow. During adolescence, the body matures long before the young person comes of age legally. On a formal level, the mentioned mantras blur the different legal and physical meanings of the term “child”. We associate with children sweet, vulnerable babies and infants, not tall adolescents. But legally, adolescents remain children until they come of age. For drug approval, everybody under 18 is officially a child. The sad key to understand the perpetuating of this confusion is the material interest of pediatric researchers who, via pediatric laws, got the affluent pharmaceutical industry to pay for “pediatric” studies, even if they were pointless. The result are “pediatric” careers in academia, regulatory authorities, industry, and commercial clinical research.
The Food and Drug Advministration (FDA) rewarded countless “pediatric” cancer studies in all age groups. But not all malignancies among minors are completely different from adults. Two key examples are leukemia and melanoma, which occasionally affects people under 18. Two international “pediatric” melanoma studies in adolescents aged 12-17 years had to be terminated. They offered treatment below standard-of-care. Doctors refused to refer further patients. Industry has developed new drugs against melanoma. State-of-the-art treatment is two drugs; the “pediatric” melanoma studies offered only one. These studies were driven by a conflict of interest. There is no “pediatric” conventional melanoma. It’s the same disease before (very rare) and after the 18th birthday (more frequent). Google “pediatric melanoma conflict of interest” and read the publication that pops up first.
Acute lymphoblastic leukemia (ALL) is the most common malignancy in minors. Most young patients can be treated by chemotherapy. But when chemo failed, the prospects were dire until recently. Then, a six-year-old girl was healed in a cutting edge study. Google “Emily Whitehead”. The treatment that saved her (tisagenlecleucel/ Kymriah) is now approved in up to 25 years.
Since 2019, the FDA recommends that industry should include adolescent patients with cancer into adult studies. Google “considerations inclusion adolescents adult oncology studies” and read the first hit, an FDA guidance document. With this, the FDA indirectly admits that all “pediatric” cancer studies rewarded between 1997 and 2019 were pointless, at least in adolescents.
The FDA has relented in adolescents because the scientific evidence of “pediatric” cancer has grown thinner and thinner. But in minors under 12, it still demands pediatric cancer studies. This approach follows the European Union (EU) that since 2007 enforced many such studies. None had positive results. Many pediatric cancer research institutions support the FDA, claiming this will advance cancer treatment. It will not. But this is another story.
When your child gets cancer, don’t trust blindly. Emily survived in a good study. In an EU-demanded study she would probably have died. Click https://www.book2look.com/book/mMqwf6ap9C to learn more. The book will come out January 2022.