Jonathan Davis, chief of the Newborn Medicine Division at Tufts Medical Center and professor of pediatrics at the Tufts University School of Medicine, is conducting research and clinical trials to make newborn medication safer.
According to Davis, the body of research on the safety of neonatal drugs is currently limited. He explained that most organizations do not want to conduct clinical trials involving infants because of a fear of publicity disasters and malpractice lawsuits if infants are harmed by their drugs during the trials.
“People say, ‘We just don’t want to experiment on anybody that small and that sick.’ But what I say is: If you don’t know what you’re doing and you expose the child to risk that way, isn’t that worse?” Davis said.
According to Davis, currently the most widely-used method for administering drugs to newborns is diluting doses of adult drugs.
“Most of the time, we don’t have drugs specifically developed for newborns. Most of the drugs we use have never been adequately tested,” Davis said.
He emphasized that one of the difficulties with neonatal medicine is that doctors do not have access to the same patient input that is used to treat adults. According to Davis, it can be difficult to determine whether an infant is in pain, so doctors work with nurse practitioners to make decisions.
Unlike adults, Davis explained, neonates cannot participate in most types of clinical trials, like walking on a treadmill and describing how they feel afterward. This makes it difficult for doctors and scientists to determine the long term effects of drugs on neonates.
Lynn Hudson, chief science officer for the Critical Path Institute and executive director of the International Neonatal Consortium (INC), expanded on the limitations of current medications available for specific health problems in infants.
“If you or I had a seizure condition, there’s about a dozen different anti-seizure medications that we can use. Some people never respond to the first one that they’re given, and then they use some other one to see if [they] respond,” Hudson said. “Babies have exactly one drug that can be used, and when they don’t respond, there’s nothing that the doctors can do.”
Davis said that most past legislative efforts have not solved this problem, but some progress has been made to raise awareness about the issue. In particular, Davis said, the United States Congress passed a law in 2012 that encouraged further study on the effects of new medication on newborns and required the Food and Drug Administration (FDA) to hire a neonatologist.
According to Davis, one way of supporting research about neonatal medicine is through the research consortium model.
“What [organizations like the Critical Path Institute] do is set up research consortia, get people together [and] provide the infrastructure and support to get people together and to get them talking,” Davis said.
Hudson explained that the consortia run by the Critical Path Institute are focused on developing tools, methods and databases for the FDA to use to develop drugs and devices for therapies.
According to Davis, the Critical Path Institute received a large grant from the FDA to form INC in 2015. Davis, a co-director of INC, explained that the global consortium incorporates about 40 different countries, along with regulators from different countries, representatives from major pharmaceutical companies, family groups and nursing groups.
“We work with the regulators from the FDA, who are now working collaboratively with us to try to improve the outcomes for these babies,” Davis said. “All of these organizations come together and for the first time … we discuss neonatal problems, propose solutions and we have physicians [and] scientists from all over the globe.”
According to Hudson, because companies often do not have the resources to conduct large scale neonatal studies, organizations like the INC that collect a wide range of data on neonatal medicine are critical.
“Different drugs target different symptoms of a disease, so you want to hear from the patients as to what’s most important to them. For neonatal [medicine], it’s a little bit different because you have to rely on the families for input. And for neonates, it’s really hard too, because there’s very little knowledge about longer term outcomes of whatever the neonates are being treated with,” Hudson said.
For this reason, Hudson said that innovative research like that being conducted by Davis contributes significantly to improving the safety of neonatal clinical trials.
“[Davis] is playing an active role in bringing in the neonatal nursing community, and that’s not a community that’s often included in research consortia and research efforts, so again we’re trying to bring in everybody that has some sort of role in developing treatments for neonates,” Hudson said. “We are starting to think beyond the typical measures that we’ve used, and we especially have to do that for infants.”
The collaboration between different research consortia and places like the Tufts Medical Center is critical for developing new medical technologies for neonates, she said.
“One of the areas of technology that we’re already exploring and incorporating into our other consortia is the use of wearable devices and sensors to monitor all sorts of aspects of activity,” Hudson said. “This would include sleep behaviors, so this might be very important for babies, because you could really get a sense for whether something is improving their sleep, improving their ability to wiggle around.”
Hudson added that organizations like the FDA play an important role in furthering research on neonatal medicine and getting organizations like the Critical Path Institute to start initiatives in areas in need of development.
Christopher-Paul Milne, director of research and research associate professor at the Tufts Center for the Study of Drug Development, has collaborated with Davis on medicine development research in the pediatric field. Milne emphasized that the study of neonatal medicine is especially critical because of the shortage of neonatologists in the United States and worldwide.
“[Research on neonatal medicine] is important for public health, to help [the pharmaceutical] industry comply with regulations and to help academia,” Milne said.
According to Milne, although very few pediatric studies have been tested so far on the neonatal population, collaboration between the medical and regulatory communities is essential to better understanding how we can improve the safety of neonatal medication and therapies.
“We can connect people in the [pharmaceutical] industry with people conducting the studies so they can understand the obstacles and figure out how to resolve them,” Milne said.