By: Margaret A. Hamburg, M.D.
It has been 11 years since Congress passed the Best Pharmaceuticals for Children Act mandating the creation of the Office of Pediatric Therapeutics (OPT) at FDA. But it has been light years in terms of the progress we have made to ensure that children have access to innovative, safe and effective medical products.
Increasingly, parents can rest assured that the medications they give their children have been tested—in children—in scientifically necessary and ethical clinical trials. OPT’s Pediatric Advisory Committee has reviewed over 200 products for their safety when used by children. Today, nearly 500 drug and biologic products have been improved by including, in their labeling, information describing safety, effectiveness and, where appropriate, dosing relating to use of the product in children.
We’ve come a long way. And as director of OPT since 2003, pediatrician Dianne Murphy, M.D., has led the charge.
It is because of her indefatigable work on behalf of children that the American Academy of Pediatrics (AAP) has bestowed on Dr. Murphy its Excellence in Public Service Award (EPSA) “representing the highest honor awarded…to a public servant for distinguished service to the nation’s children, adolescents and young adults.” In the letter informing Dr. Murphy of this honor, AAP says her work at FDA “has profoundly improved the lives of children in the U.S. and around the world through increased access to needed therapeutics.”
I couldn’t agree more.
For many years before Dr. Murphy began her tenure at FDA, the playing field badly needed leveling when it came to safety in children’s drugs. Very few drugs—even those meant solely for children’s use— were actually tested on children. For one thing, some drug companies, eager to get their new products out while they still held exclusivity (that is, the period of time during which generic drugs cannot be developed), were reluctant to take the time or go to the expense. Many people, too, felt it was unethical to use children as so-called “guinea pigs.”
Without the information gained from testing children in clinical trials, health care professionals and parents alike could only guess when trying to gauge the correct dosages for children.
This was an enormous problem. Children are vulnerable. Their organs are developing, and they are experiencing physical changes that affect how a drug is metabolized. As Dr. Murphy has often said, guessing at the dose just doesn’t work. Children must be studied if we are to give them safe and effective treatments.
The sea change began ten years before her arrival at FDA, when AIDS was entering the public consciousness. Dr. Murphy has told me that at the time, she was treating children in her practice who were dying of AIDS because there were no drugs available specifically to address their unique needs.
Based on that hard lesson, a major priority when she came to FDA was to ensure that drugs for children first be tested in children in clinical trials. Dr. Murphy has worked with FDA scientists and reviewers to ensure that pediatric studies are rigorously designed and conducted in accord with current scientific understanding of the characteristics that make children unique. And she has championed FDA’s involvement in the international arena, where FDA has emerged as a leader in regulatory thinking.
In achieving this honor, Dr. Murphy is in very good company. She shares it with such distinguished former honorees as First Lady Michelle Obama, the late Sen. Edward M. Kennedy, Rep. Henry Waxman, former FDA Commissioner David A. Kessler, and National Institutes of Health Director Francis S. Collins.
At FDA, we offer our congratulations, and our thanks.
Margaret A. Hamburg, M.D., is Commissioner of the Food and Drug Administration