The regulatory push to develop pediatric versions of many new adult medicines has prompted some CROs to step up their clinical research activities this year by creating or expanding pediatric investigator networks and pursuing the rare disease arena for trials involving both adults and children.
Both the FDA and European Medicines Agency (EMA) made significant changes in their regulations and now require a pediatric component for most clinical research. In the U.S., pediatric versions of new medications often are completed about nine years after the FDA approves adult versions—a gap that many sponsors and CROs hope to narrow.
The growth of these trials in children also has led to the creation of Paidion Research, the first global CRO dedicated exclusively to pediatric clinical research with a site network across a range of pediatric providers. The nearly 2½-year-old company also is a niche provider, specializing in neonatal and pediatric intensive care.
“We’ve been operational for a year and a half, and our growth is robust to where we will have about four or five pediatric trials this year and expect to expand to eight to 10 pediatric trials in 2016,” said Barry Mangum, Pharm.D., founder and CEO of Paidion—a company name derived from the Greek word for child. “We are the only all-pediatric CRO.”
Currently, its trials include a number of neonatal indications and rare pediatric diseases. The young company also has created a sustainable network of 50 neonatal centers, with plans to expand to 100 centers in the coming months.
Several other CROs also are expanding their pediatric clinical trial endeavors. Pharmaceutical Product Development (PPD) recently heralded its eight-site global pediatric network that is designed to accelerate the process of faster trial startup, more predictable enrollment and higher-quality data. Among the pediatric investigator sites are Columbia University, UNC at Chapel Hill Department of Pediatrics, Seattle Children’s Research Institute, Manchester University Hospitals in the U.K., and Schneider Children’s Medical Center in Israel.
“Building formal relationships with strategically important centers that have significant pediatric clinical trial expertise, a proven track record of superior quality and the ability to recruit pediatric patients demonstrates our commitment to partnering with our clients to bring life-changing medications to market faster and at less cost,” Mark Sorrentino, M.D., PPD’s executive medical director and chair of its pediatric practice area, said in a prepared statement.
Also planning to expand its pediatric capabilities is WCCT Global, a Southern California CRO that will provide clinical trial services for children with diabetes, cardiovascular, GI and CNS issues, along with orphan drug clinical trials in both children and adults. While pediatric clinical trials are a small part of WCCT’s clinical trial portfolio, it has seen a greater acceptance of parents of pediatric patients with orphan diseases where there is no treatment available. The organization is willing, however, to consider trial participation.
“We are in a very populated area of California with potential patients within driving distance of us, and we are working with sponsors so we can do things to identify subjects—both adults and children—with rare diseases,” said Kenneth Kim, M.D., the founder and CEO of WCCT. “We’ve done trials in children with orphan diseases, some in the muscle-wasting space, and have been developing relationships with physicians and key opinion leaders. Still, the challenge is finding parents with those young patients.”
Indeed, identifying an adequate number of children suitable to be patients is far and away the single greatest challenge in conducting a pediatric clinical trial, according to several surveys including a study released last month from Premier Research. The study also revealed a lack of awareness among clinical trial sponsors that, as part of the FDA Safety and Innovation Act (FDASIA), the agency will award priority review vouchers to sponsors of rare pediatric disease product applications meeting specific criteria.
Medical products, such as biopharmaceuticals, that undergo priority review generally are given an approval decision—positive or negative—within six months of filing date, rather than the usual 10 months, according to Nachiket Dave, Premier Research’s director of regulatory affairs, who said very few survey respondents indicated they ever read the FDA guidance.
Still, difficulty in recruitment, which Premier Research cited in its 2012 survey on pediatric clinical trials challenges, continues today.
Finally, a Quintiles survey earlier this year found that while 73% of parents said they were interested in having their child participate in an appropriate clinical trial, 96% said they have never actually participated in a trial. The reason: They were never asked.
The survey, which was presented at the national conference of the American Academy of Pediatrics, concluded that while most parents preferred to learn about a pediatric trial at the doctor’s office or through friends and family, there needs to be better communication between parents and pediatricians. It also cited the need for physicians to make clinical trials part of the treatment conversation with parents.
In turn, the survey suggested biopharmaceutical companies should develop their own communication efforts to educate parents about the safety and value of participating in pediatric clinical trials to show them where they can learn more about trial opportunities.
“Patient recruitment and retention is imperative to the success of these efforts, and spending a little extra time and money up-front to engage this hard-to-reach population will deliver long-term strategic benefits down the line,” the survey concluded.
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