Sponsors of atopic dermatitis drugs for children don’t have to wait to test a drug’s safety or efficacy on adults before beginning pediatric trials, the FDA says.
The new guidance, released last week, reverses longstanding FDA recommendations that drugs trying to treat a disease by affecting the entire body start with adult trials.
Atopic dermatitis is the dry, itchy rash caused by eczema that affects nearly 18 million Americans, most of them children. It often clusters with asthma and hay fever.
The agency says its U-turn was prompted by recommendations from a 2015 meeting of its Dermatologic and Ophthalmic Drug Advisory Committee.
The FDA acknowledges that “some major safety questions” may be left open before trials can begin.
But it says it’s “not generally necessary to have an extensive safety database in adults before initiating pediatric studies” as long as the disease isn’t potentially fatal to pediatric populations and there’s a risk doctors may be prescribing therapies off-label before trials wrap up.
Trials will have to consider a drug’s effect on children of all ages, including toddlers and infants, the guidance adds.
Sponsors may have to start with older children first if specific data from an older subpopulation can help inform the study, if there’s an “age-related technical issue” or if there is some reason to worry about a drug’s safety in younger children.
Read the final guidance here: www.fdanews.com/10-02-18-AtopicDermatitis.pdf.
Re-Trials for Pediatrics Pay Off
The FDA’s pediatric exclusivity rules allowed drugmakers to reap a 680 percent return on their investments, a new analysis finds.
The Best Pharmaceuticals for Children Act of 2002 offered drugmakers six months of market exclusivity in return for running drugs already approved for adults through clinical trials again to test their safety and efficacy for children.
Congress passed the measure because it was concerned that doctors were prescribing adult meds to kids off-label and the pharmaceutical industry argued that limited exclusivity provided proper incentive to invest in fresh trials.
The incentive has proved lucratice, according to the analysis published in JAMA Internal Medicine.
Study author Michael S. Sinha and his colleagues at Boston’s Brigham and Women’s Hospital examined 54 drugs given exclusivity between 2007 and 2012.
They estimate pharma companies spent an average of $36.4 million to re-test their drugs in clinical trials and the median net return was $176 million—a ratio of about 680 percent.
“Meaningful knowledge of pediatric uses of pharmaceuticals has come from the pediatric exclusivity program, but at a high cost” in hefty drug prices passed along to consumers, Sinha said, noting that “other approaches … such as direct funding” for pediatric trials “may be more economically efficient.”