The Pediatric Research Equity Act (PREA)—requiring drug makers to test all drugs for safety and efficacy in pediatric populations as well as in adults—was passed in 2003 and updated in 2007. And in Europe, a similar requirement has been in effect since 2007.
Nevertheless, many sponsors ignore both requirements, testing their drugs only in adults. But this could change soon if PREA is renewed and set in stone this September, said Charlene Sanders, a pediatrician and vice president of regulatory affairs and pediatric consulting for CRO Premier Research, which specializes in pediatric trials.
Both the House and Senate have passed the FDA Safety and Innovation Act (FDASIA), reauthorizing PDUFA for another five years. The user-fee package now awaits President Obama’s signature, after which it would go into effect Oct. 1, making permanent both PREA and the Best Pharmaceuticals for Children Act.
Premier recently sought to learn the industry’s views on PREA, sending out the first of what will be a series of surveys to sponsors. The CRO received responses from 55 companies conducting trials in the U.S. and abroad, of which only 52% said complying with PREA was a high priority, 36% saying it was a moderately high priority and 10% admitting it was a relatively low priority.
Why do so many drug makers brush aside the requirement? Sanders said, “A lot of sponsors thought, ‘Okay, I’ll just deal with this later,’ and still others have been watching to see if it’s renewed in September, hedging their bets on it not getting renewed.”
But, she added, it’s likely this fall sponsors will begin to accept that testing drugs in pediatric populations is what they must do to be in compliance. And then there will be a mad rush for pediatric patients, as well as for expertise on how to recruit for and set up pediatric trials.
The survey also revealed that sponsors did not know companies can, in some circumstances, work with the FDA to tweak the PREA requirements that apply to their trials. About one-third (34%) either did not know how to conduct a PREA compliance discussion with the FDA or were not aware there may be room to amend or revise their PREA obligation, said Sanders.
But she explained that in some cases—particularly when recruiting children would greatly slow the trial and delay the availability of the drug to adults, and also when recruiting children with a certain condition would be exceedingly difficult—the FDA will make exceptions.
Recruiting pediatric patients is a big concern for sponsors. More than seven in 10 (73%) said they felt strongly that finding enough children to participate in trials is a problem. This, said Sanders, is likely to drive a great deal of work to CROs with expertise in pediatric trials. Nearly six in 10 (56%) identified pediatric patient recruitment as the number one area in which they needed help from a CRO in satisfying regulatory requirements.
Sanders said in some cases this is founded; there are many conditions and diseases for which recruiting children is very difficult. But for many other conditions and diseases it’s not as much of a conundrum; much of the worry comes from making assumptions about recruiting for pediatric trials. “Many of the concerns are founded,” said Sanders. “But also, many people haven’t yet experienced recruiting for pediatric trials and have a lot of fears that are unfounded.”
For eight years, Premier served as the administrator and clinical trial manager on pediatric trials for the National Institute of Child Health and Human Development (NICHD).
CenterWatch Weekly, July 2, 2012