Nav: Home

Nearly half of pediatric clinical trials go unfinished or unpublished

August 04, 2016

Recent legislation is encouraging clinical trials in children, including the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Yet clinical trials in children commonly go either uncompleted or unpublished, finds a comprehensive study conducted by researchers at Boston Children's Hospital. Results were published online August 4 by the journal Pediatrics.

In all, 19 percent of trials were discontinued early, and 30 percent of completed trials remained unpublished in the medical literature several years later. "We feel there is a lot of inefficiency and waste that could be addressed," says senior investigator Florence Bourgeois, MD, MPH, of Boston Children's Hospital.

Overall, trials sponsored by industry were more likely to be completed than trials sponsored by academic institutions, the investigators found. However, completed trials sponsored by industry were less likely to be published than trials sponsored by academia. These findings are similar to those seen for clinical trials in adults.

"Our findings are in line with previously published studies focusing on adult trials, which may speak to how commonplace discontinuation and non-publication are in medical research in general," says coauthor Natalie Pica, MD, PhD, a resident at Boston Children's. "We need to make sure that when children participate in clinical trials, their efforts are contributing to broader scientific knowledge."

Pica and Bourgeois tracked 559 randomized, controlled pediatric trials registered on ClinicalTrials.gov from 2008 to 2010 and whose final status (completed or discontinued) was confirmed by the end of 2012. They then searched for related peer-reviewed publications through September 1, 2015. When no publication could be found, they inquired with study investigators and sponsors via email.

Their findings:
  • Of the 559 trials, 104 (19 percent) were discontinued early. Two thirds of these had already enrolled participants.

  • Of the 455 completed trials, 136 (30 percent) remained unpublished after an average of 58 months post-completion. (Forty-two of these, or 31 percent, did post results to ClinicalTrials.gov.)

  • Of the 104 discontinued trials, 39 percent were sponsored by industry and 55 percent by academic institutions. (The rest were funded by other sources.)

  • Two years after trial completion, academia-sponsored trials accounted for 30 percent of unpublished trials, and industry-sponsored trials for 63 percent. Three years after trial completion, academia-sponsored trials accounted for 23 percent of unpublished trials, and industry-sponsored trials for 70 percent.

  • In a multivariate analysis, the likelihood of non-publication was more than doubled for industry-sponsored trials two years after completion (odds ratio, 2.21) and more than tripled three years after completion (OR, 3.12).

  • Overall, more than 8,000 children were enrolled in trials that were never completed, and more than 69,000 children were enrolled in completed trials that were never published.

"This is the first study to look systematically at discontinuation and nonpublication of interventional pediatric clinical trials," says Bourgeois. "A number of legislative initiatives have been implemented to increase the study of interventions in children. Now we need to make sure that the proper resources are in place to ensure that information gleaned from these studies reaches the scientific community."

One proposed initiative cited by the paper is RIAT (Restoring Invisible and Abandoned Trials), which is supported by some high-profile journals. RIAT invites researchers with unpublished trials to either commit to publish within a year or provide public access to their data, allowing independent investigators to become "restorative authors."

"It's hard to reanalyze others' data," says Pica, "but this may be a useful mechanism to make sure that findings from completed trials are disseminated in the medical literature."
-end-
The study was supported by the National Institute of Child Health and Human Development (1R21HD072382) of the National Institutes of Health and the Fred Lovejoy House-Staff Research and Education Fund at Boston Children's Hospital.

About Boston Children's Hospital

Boston Children's Hospital is home to the world's largest research enterprise based at a pediatric medical center, where its discoveries have benefited both children and adults since 1869. More than 1,100 scientists, including seven members of the National Academy of Sciences, 11 members of the Institute of Medicine and 10 members of the Howard Hughes Medical Institute comprise Boston Children's research community. Founded as a 20-bed hospital for children, Boston Children's today is a 404-bed comprehensive center for pediatric and adolescent health care and the primary pediatric teaching affiliate of Harvard Medical School. More on our Vector and Thriving blogs and social media channels: @BostonChildrens, @BCH_Innovation, Facebook and YouTube.

Boston Children's Hospital

Related Clinical Trials Articles:

Giving children a voice in clinical trials
Children as young as 8 years old with incurable cancer can reliably characterize the impact an experimental therapy has on their symptoms and quality of life -- even at the earliest stages of drug development -- making self-reported patient outcomes a potential new clinical trial endpoint.
Better health for women involved in clinical trials
Women who participate in obstetric and gynecology clinical trials experience improved health outcomes compared to those who are not involved in trials, according to research by Queen Mary University of London.
Final artificial pancreas clinical trials now open
Clinical trials are now enrolling to provide the final tests for a University of Virginia-developed artificial pancreas to automatically monitor and regulate blood-sugar levels in people with type 1 diabetes.
Why the bar needs to be raised for human clinical trials
Standards for authorizing first-time trials of drugs in humans are lax, and should be strengthened in several ways, McGill University researchers argue in a paper published today in Nature.
New drug formulary will help expedite use of agents in clinical trials
The National Cancer Institute (NCI) today launched a new drug formulary (the 'NCI Formulary') that will enable investigators at NCI-designated Cancer Centers to have quicker access to approved and investigational agents for use in preclinical studies and cancer clinical trials.
Review examines diversity in dermatology clinical trials
Racial and ethnic groups can be underrepresented in medical research.
Reshaping the future of global clinical trials practice
Researchers at the University of Liverpool have developed a new international guideline to help standardize how results from clinical trial studies are reported.
Fewer cardiovascular drugs being studied in clinical trials
The number of cardiovascular drugs in the research pipeline has declined across all phases of development in the last 20 years even as cardiovascular disease has become the No.
Sex hormones skew outcomes in clinical trials -- here's how
Clinical research often excludes females from their trials under the assumption that 'one size fits all,' that a painkiller or antidepressant will be equally effective in subjects of either sex, but a growing number of scientists are criticizing this approach.
Nearly half of pediatric clinical trials go unfinished or unpublished
Clinical trials in children commonly go either uncompleted or unpublished, finds a comprehensive study conducted by researchers at Boston Children's Hospital.

Related Clinical Trials Reading:

Best Science Podcasts 2019

We have hand picked the best science podcasts for 2019. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

Anthropomorphic
Do animals grieve? Do they have language or consciousness? For a long time, scientists resisted the urge to look for human qualities in animals. This hour, TED speakers explore how that is changing. Guests include biological anthropologist Barbara King, dolphin researcher Denise Herzing, primatologist Frans de Waal, and ecologist Carl Safina.
Now Playing: Science for the People

#SB2 2019 Science Birthday Minisode: Mary Golda Ross
Our second annual Science Birthday is here, and this year we celebrate the wonderful Mary Golda Ross, born 9 August 1908. She died in 2008 at age 99, but left a lasting mark on the science of rocketry and space exploration as an early woman in engineering, and one of the first Native Americans in engineering. Join Rachelle and Bethany for this very special birthday minisode celebrating Mary and her achievements. Thanks to our Patreons who make this show possible! Read more about Mary G. Ross: Interview with Mary Ross on Lash Publications International, by Laurel Sheppard Meet Mary Golda...