Watch this video for more insights into pediatric medicine from Dianne Murphy.
As Director of the Office of Pediatric Therapeutics at the Food and Drug Administration (FDA) since 2003, Dianne Murphy has had a front-row seat to the world of pediatric therapeutics. Dr. Murphy, who in 2013 received the Excellence in Public Service Award from the American Academy of Pediatrics (AAP), will be retiring from FDA at the end of October. Here are some of her insights.
Q: I understand you’ve worked for the Food and Drug Administration (FDA) two different times. When?
A: The first time was in 1990, when I was working as a pediatric infectious disease specialist at the University of Tennessee in Knoxville and was seeing kids with hemophilia who were dying of HIV. There were no drugs available to meet their unique needs. So when I was invited by a colleague to work at FDA, a major priority was to ensure that drugs for children first be tested in children in clinical trials. It was an amazing time—lots of change. We worked closely with NIH, as well as with various activist groups to change how we approached getting products that were being used by children, studied in children.
Back then, most products for children hadn’t been studied in children—who were instead receiving therapies tested only in adults. This time period was the beginning of an enormous push by FDA, the AAP, and Congress to get products used and studied in children. Eventually the effort was backed by several pieces of legislation that served as the foundation for pediatric studies as we know them today.
Q: Have you seen a change in the scientific information now available about the safety and effectiveness of products used in children?
A: Definitely. We’ve come a long way in that regard from roughly 25% of approved products having been studied in children at that time, to about 50% today. We now have over 630 products that have been studied and labeled for use in children.
We still have a long way to go, and the other 50% is going to be a harder slog, because we haven’t tackled the really hard stuff yet, such as therapies for neonates. It’s very hard to get parents to volunteer to put their neonates in a study, and of course it’s very difficult to work with such a young, fragile population of subjects.
Q: What have you liked most about working here?
A: That’s easy. It’s the people. And the issues.
You know, when I was growing up and thinking about medical school, I never said to myself, “Oh, I want to go work for the FDA.” But you come here and see the scope of the science and the work that we do, and you realize the impact it has on everyone’s daily life—from the food we eat, to the drugs we take, to the devices that are part of our everyday routines.
It’s an enormously interesting place to be from a science perspective. You get to deal with some of the best scientists in the world because you’re trying to solve problems like, how do you prove that a product works? And how do you set up a trial to better examine what sub-population would be the best target group to work for a certain therapy?
Why, we’re just now getting into the area of genomics and why a certain therapy works for some people but not others. I can only imagine the advances we’ll make in the coming years. More and more science is coming to light that helps us do a better job at identifying and examining newer therapies to be sure they are both safe and effective.
Q: What was it like speaking with parents and kids?
A: You get out there and you see what people’s concerns are. You can’t just answer with a rote, “we’re working on it.” You have to find a way to answer that makes sense to them. You have to be accountable.
We can’t always speak plainly about the work we do because some of it is proprietary, but we do have to find a way to hear what people are thinking about and respond to them in a way that says more than “oh, we’re publishing a guidance.”
Q: Did having been a parent yourself make any difference in your role here?
A: Absolutely. You feel that way as a practicing physician, too. When talking to parents, you can’t help but empathize with their concerns. You have a lot more empathy, even when it comes to a basic task such as trying to get an uncooperative child to take medicine, how difficult that can be. Sometimes it’s just as important to be a parent as it is a scientist when it comes to developing therapies for children.
Q: What advances in pediatric medicine have you seen during your tenure here?
A: I think the biggest accomplishment has been the fact that now pediatrics is not an afterthought in terms of product development, it’s become part of the process of developing therapeutic products.. That’s been a huge change for not just the FDA, but also for doctors, parents, nurses, and pharmaceutical companies.
I see a lot of movement in developing pediatric research networks that are national and international in scope. We need to have relationships with other countries, regulatory groups, and academic institutions.
Q: Where are you going to go when you leave us here?
A: Back in time!
I’m already a docent and a board member at a historic house in Alexandria, Virginia. I love history; it would have been my second career choice after medicine. I’ll probably go back to school to get a degree in historic preservation, as well. We live surrounded in the state of Virginia by enormous history. I sort of grew up with it, and it’s my secret passion.