PC3002: Analysis of the Number of Research Experienced Pediatric Clinical Trial Investigators in the United States: A Critical Need and a Lot of Room for Improvement

Background: Off-label drug use in children is extremely high in both inpatient and ambulatory settings, with over 90% of drugs in some settings being prescribed off-label (Hoon et al, 2019). Physicians often prescribe drugs to children off-label due to a lack of approved medicines. Efforts to encourage development of more evidence based drugs for children, such as the Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA), have yielded some benefit, but progress is still slow. Trial conduct in children is uniquely challenging for a variety of reasons, including low patient numbers, unique physiology, and ethical considerations. Another limitation is the availability of sufficient industry experienced pediatric clinical trial investigators. Here, we analyze pediatric research experienced and naïve investigators in the United States to determine the potential for new pediatric clinical trial investigators.

Methods: Using clinical trial data from Citeline (https://citeline.informa.com/) and clinicaltrials.gov, we analyzed the number of recent (since 2017) industry sponsored trials conducted in the United States, and how many of those involved children ( < 18 years old) and how many investigators were required for those trials. Using claims data, we obtained the total number of physicians in the United States with specialization in pediatrics. We then analyzed the number of investigators with research experience in the subspecialties with the greatest numbers: hematology/oncology, neurology, cardiology, gastroenterology and psychiatry, as well as general pediatricians.

Results: There have been over 13,000 industry sponsored trials in the United States since 2017 and of those over 1,500 (11%) have included pediatric participants. Over 66,500 investigators are listed in Citeline associated with these trials, with just over 5,000 (7.5%) specializing in pediatrics.
Using claims data since March 2017, Table 1 shows that the pediatric subspecialties with the greatest proportion of research experienced investigators are, respectively: Hematology/Oncology (51.6%), Neurology (29.7%), Gastroenterology (27.1%), Cardiology (15.3%) and psychiatry (6.5%). In addition, there are over 96,000 pediatricians with no subspecialty, with 4,118 (4.3%) being research experienced.

Conclusion: Overall, a small proportion of physicians specializing in pediatrics have clinical trial research experience, and most of those with research experience have a subspecialty. As expected, the greatest proportion of pediatric clinical trial research experienced investigators specialized in hematology/oncology (51.6%), as clinical trials are typically the first consideration for treating children with cancer. The proportion of research experienced general pediatricians was much lower (4.3%). Given the large number of medicines that are used in community and ambulatory care settings, having clinical trial research experienced general pediatricians is vital to accelerate access to evidence based medicines for children. Next, we will identify the key barriers that physicians encounter when trying to enter clinical trial research.