Nature published an editorial supporting Kids vs. Cancer’s bill to increase support for pediatric clinical trials.
The bill, also known as the RACE for Children Act, aims to amend the Pediatric Research Equity Act (The Cancer Letter, July 15).
Under PREA, entities researching drugs through clinical trials enrolling adults must also test their drugs in pediatric populations for the same indication. The law grants FDA the discretion to issue waivers to companies if their drugs, biologics or devices treat or target an indication not present in pediatric populations.
According to an FDA guidance to industry: “A drug or biological product for an indication that has extremely limited applicability to pediatric patients because the pathophysiology of these diseases occur for the most part in the adult population. FDA would be likely to grant a waiver for studies on products developed for the treatment of these conditions without requiring applicants to provide additional evidence of impossibility or impracticality.”
The exception to the law makes it difficult for children to receive the newest and often most affective experimental treatments for their cancers, the Nature editorial states.
“Because they make up only 1 percent of U.S. patients with cancer, children are a low priority for pharmaceutical companies that want to launch an effective drug quickly. The hassle of a pediatric clinical trial may not seem worth it until after the drug has proved to be safe and effective in adults. This process can take decades, leaving children with therapies that are sometimes almost obsolete.”
If passed, the RACE for Children Act (S.3239, H.R.5858) would require drug developers to conduct pediatric studies under PREA when the indications of their study were molecularly relevant to childhood cancer.
The editorial supports the bill, but calls on lawmakers to consider not only mending the loophole in PREA, but finding other ways to incentivize companies to provide experimental drugs to children.
“When lawmakers pick it [the RACE for Children Act] up, they should also address problems with compassionate-use programmes—and ensure a transparent and useful process for people to gain access to unapproved drugs. They should also encourage companies to make more drugs available through market incentives, and provide increased protection should something go wrong,” Nature concluded.
