How to promote new therapies for neonates through education and extended exclusivity

By Melissa Fassbender

- Last updated on GMT

(Image: Getty/mmpile)
(Image: Getty/mmpile)
It has been nearly 20 years since a currently available drug was tested and approved specifically for the newborn population, say industry experts who are calling for education and policy changes to address the needs of this special patient population.

About 4m babies are born each year in the US, yet 90% of the medications they take have never been tested for children and neonates, according to the Chairman for the Alliance for Patient Access David Charles, MD.

As the industry is well aware, a significant portion of clinical trials fail to meet enrollment goals. Yet, “when you consider typical trials for children, infants, and neonates, the problem is much, much worse,”​ said Charles.

“Everyone agrees that we need to do clinical trials and develop medicine and understand if they are safe and effective in children, infants, and neonates,”​ he told us.

“It’s not medically acceptable to say, ‘we are going to test in adults and lessen the dose,’”​ Charles said. However, that is often the situation. While at a policy level, everyone agrees studies need to be conducted in children, when put forward, concerns surface.

“If you’re exposing children, infants, or neonates to risk, there’s just a higher level of concern,”​ said Charles.

Another challenge is the issue of informed consent, as for children, or anyone not of legal age, the decision to participate in a clinical trial is made by a parent or legal guardian.

Charles said these challenges must first be addressed through education, not just of the public, but physicians, scientists, and those sitting on investigation review boards.

“It is through clinical trials that we get all of our new medications, medical devices, and many diagnostic tests,”​ he added. “It’s a required essential step to show that something is safe and effective.”

The next steps: Policy changes and financial incentives

Conducting clinical trials is “extraordinarily”​ expensive, with a recent Tufts study estimating​ the average cost to develop and gain marketing approval for a new drug at $2.558bn.

Still, the cost to conduct a clinical trial in children, infants, and neonates is even higher because of the challenges in enrollment, consent, as well as finding investigators and centers that are willing to host clinical trials, Charles explained.

From an industry perspective, he noted, “there’s no way around the fact that clinical trials are expensive, and then in this population, it’s very expensive.”

To create incentives, legislation has been put forward proposing extended exclusivity periods, “such that manufacturers could have the opportunity, if the trial is successful, to recoup the costs”​ Charles explained.

The Promoting Life-Saving New Therapies for Neonates Act of 2017 ​seeks to amend the Federal Food, Drug, and Cosmetic Act​. If passed, the act would require the US Food and Drug Administration (FDA) to award sponsors a neonatal drug exclusivity voucher following the approval of a new drug or biological product for the treatment of newborns.

Susan Hepworth from Alliance for Patient Access explained that the act “would stimulate investment in the development of new drugs for neonates by rewarding drug sponsors who successfully develop new products for neonates with a one-year extension of their exclusivity period on another drug.”

“It would also ensure that new drugs would address the most critical needs of this population,” ​she told us.

The bill was introduced in May 2017 and has garnered the bipartisan support of 37 co-sponsors.

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