FDA subcommittee to examine regulatory science for neonatal studies

The US Food and Drug Administration (FDA) is beginning its new effort to stimulate research on treatments for neonates in the wake of last year’s legislation, the FDA Safety and Innovation Act.

An Institute of Medicine report last year said that from 1998 through 2010, only 23 (6%) of the 365 labeling changes that involved the submission of new pediatric studies “included the addition of information from studies with neonates.”

A recent commentary on the subject of federal legislation and the advancement of pediatric drug studies said the lack of clinical trials on neonates implies they are a “therapeutic orphan,” at potential risk for getting ineffective medications and invalidated doses, and for developing unanticipated complications including adverse drug reactions.

As part of the new work, in March the FDA held the first meeting of its neonatology subcommittee with 13 experts from government and clinical practice to discuss advancing the regulatory science.

Now the agency is moving toward bringing a senior level neonatologist into the FDA to help create a strategy, although it’s too early to be say whether that person can be on board soon, said Robert Nelson, MD, PhD, deputy director of FDA’s office of pediatric therapeutics, in an April interview.

Although Congress called for a neonatologist in that capacity, it did not provide funds for the position. However, the FDA says funding will be available through the Oak Ridge Institute for Science and Education for a fellowship for what will probably be part-time work for up to 2 years, “depending on the seniority of the candidate and the percent of effort dedicated to the fellowship.”

Nelson said there’s been interest expressed in the position. “We don’t think that hiring just one neonatologist within FDA is going to solve the broader problem of trying to facilitate product development for the neonatal population,” he said, so the agency also wants to leverage the expertise of the neonatal community.

That’s one reason for the neonatal subcommittee, he said. The hope is that the agency will be able to reach out to individual neonatologists to work on specific issues, with the internal neonatologist being the point of contact.

Nelson said he hopes the person will be mid or late career and well-placed within the neonatal community, to get the process up and running.

A goal will be to make some “early wins” by identifying products or product areas in which progress can be made quickly. Perhaps demonstrating the value of the process would help the agency make an argument for funds to complete the congressional mandate to have a neonatologist for at least 5 years, he noted.

One challenge, said Nelson, will be getting studies done on the many drugs that are no longer under patent and that are being used off-label in neonatal intensive care units. Another challenge is getting industry to see neonatology as an area in which to develop new products for problems without adequate treatment.

One possibility being discussed, he said, is something similar to a public-private consortium to study the challenges to that research, including such things as lengths of trials and endpoints.

Jonathan Davis, MD, acting chair of the FDA neonatology subcommittee and chief of the division of newborn medicine at Tufts Medical Center in Boston, also pointed out that the legislation gives the agency more leverage to get drug companies to conduct research on neonates, unless there is a specific reason not to.

“There have been many instances, even in recent history, where we have finally studied the drug in great detail and found that it doesn’t work [in newborns] or it may not be safe,” he said. “It is something that we feel very strongly about, that we have to help babies, because if we don’t, I am not sure who will,” said Davis.