FDA Commissioner Speaks to Children's National Clinicians During REW
Research scientists at Children's National, an "anchor institution in a community that has many strengths," can help to advance pediatric drug and device development
“Zika virus is raising this issue about studying pregnant women in a really major way. It’s pretty amazing and worrying that a single mosquito bite with an asymptomatic infection can cause severe fetal malformations,” Dr. Califf tells Children’s National staffers. “Also, we’re now learning that it looks like it’s likely that the maternal duration of the virus being active is longer than it is in non-pregnant women. Here we have a situation where we don’t have a treatment. We don’t have a vaccine. We’re going to develop a vaccine. The most important patient group is obviously the pregnant women. How are we going to do the studies, given all the concerns?”
Dr. Califf’s remarks were delivered as part of Research and Education Week, an annual celebration of the cutting-edge research and innovation happening every day at Children's National.
Indeed, six Children’s research scientists were among the international team that reported the surprising finding that the Zika virus lingered weeks longer than expected in the blood of a pregnant woman, results that were published last month in The New England Journal of Medicine.Dr. Califf told Children’s staffers that the FDA regulates products that represent one-fifth of the American economy, including cosmetics, food, veterinary products, tobacco, medicines, and devices. Federal legislation and researchers’ perseverance has led to a myriad of medicines and devices designed for the special needs of children.
- The FDA updated 51 medical product labels last year as a result of research studies conducted in children and has made 615 pediatric label changes from 1997 to 2016.
- As part of its efforts to encourage development of pediatric devices, the FDA has provided $21.4 million in grants to consortia that offer advice throughout the continuum of device development, including forming the concept, conducting preclinical work, manufacturing, and marketing. Since Children’s and partners launched the National Capital Consortium for Pediatric Device Innovation in 2013, the group has advised and assisted more than 50 device innovators.
“I think it’s critical to the FDA that you all figure out how to take advantage of this new era to do the phenotyping that is needed,” he says. During adulthood, ”things are relatively the same for decades. But you are dealing with human beings who are changing on a daily or weekly basis in ways that could radically change things like metabolism and distribution of a drug or how a device might actually be employed.”
During a question-and-answer session led by Surgeon-in-Chief Anthony Sandler, MD, Dr. Califf encouraged research scientists developing products that ultimately need FDA approval to involve patients early. In projects that have followed this route, patients and patient advocates have helped to improve the study design, the form used to obtain patient consent, and have helped to disseminate study results.
“The main thing, if you’re the inventor: Don’t come to them at the last minute,” he says. “Come to them right at the beginning. Make them partners in the development of your drug or device.”