The results of dozens of clinical trials involving thousands of American children have never been made public in violation of the law.
The benefits and harms detected during those trials, which involved 3,627 children, remain completely unknown, leaving parents, doctors and medical researchers in the dark about the benefits and harms of potentially life-saving treatments.
UPDATE 24 January: FDA response added further below.
A report published today by the National Center for Health Research, Universities Allied for Essential Medicines and TranspariMED found that the results of 43 paediatric clinical trials subject to a U.S. federal disclosure law have never been made public, neither on a registry nor in the scientific literature.
The children who participated in these invisible trials include:
166 infants recovering from heart surgery
130 boys with incurable Duchenne Muscular Dystrophy
110 prematurely born babies
974 children with ADHD
An additional 38 trials had published results only partially or with delays, also in violation of the law.
Only last week, a Congressional committee had demanded that FDA and NIH take action to ensure that clinical trial results are made public in line with the law.
Which sponsors broke the law?
Law-breaking sponsors identified in the report include Phoenix Children's Hospital, Wayne State University, Astria Therapeutics, and Rhodes Pharmaceuticals; the latter is a wholly-owned subsidiary of Purdue Pharma.
By law, the FDA could impose a fine of over $13,000 for each day that a trial result is not made public, but to date has never done so, ignoring a total of over 3,000 trial results that verifiably remain unreported. (The true number of missing trial results probably exceeds 10,000.)
Asked what measures it had taken to ensure that missing results of pediatric trials were made public, the FDA declined to comment.
Calls for NIH to cut off funding
Similarly, the National Institutes of Health (NIH) have the power to cut off public funding to law-breaking institutions.
Dr Diana Zuckerman, President of the National Center for Health Research, said:
“When any adult or child participates in a clinical trial, they are taking a risk for the greater good, but if the results are not made public, those studies are of no help to anyone.”
“NIH is supposed to cut off funding if study results are not made public, but they haven't done that even once. Our next step is to work with Congress to fix this situation.”
Navya Dasari, a member of the student group Universities Allied for Essential Medicines, said:
“The parents of children who participate in these trials should be able to access the results, and doctors and parents of children who are not trial subjects should be able to access the latest science that could be relevant to their care.”
“The findings of this report highlight the urgent need for FDA and NIH to finally enforce the law.”
FDA has only sent 92 notices to sponsors
While the FDA did not respond to TranspariMED's initial outreach, STAT News managed to coax a reply from the agency:
An FDA spokesperson wrote us to say that, “in all areas the FDA regulates, the agency’s goal is to achieve voluntary compliance with these legal requirements. The FDA’s compliance activities related to the ClinicalTrials.gov requirements provide the opportunity for responsible parties to take voluntary corrective actions before the agency proceedswith a civil or criminal enforcement action.”
She added that the agency has also sent 92 preliminary notices — including 14 that were issued as part of a pilot program a decade ago — to drug companies and universities that failed to report trial results. “We will continue to monitor compliance with the ClinicalTrials.gov requirementsand will take appropriate action as warranted,” she continued.
American patients harmed
Past failures to make clinical trial results public have led to immense human and financial costs.
These include the deaths of tens of thousands of Americans in the 1980s because the dangers of a class of drugs went unrecognised (antiarrhythmic drugs), the misallocation of over $18 billion of largely public funds on a drug whose effectiveness had been overstated (Tamiflu), and investor losses of $37 billion following the withdrawal of a drug whose dangers had not been fully reported (Vioxx).
Such harms to patients and public health of unreported clinical trials have been exhaustively documented in the scientific literature. The World Health Organisation has repeatedly called for clinical trial results to be made public on trial registries within one year of trial completion.
Congress needs to wake up
Till Bruckner, founder of TranspariMED, said:
“Sick American children and their parents deserve better than this. With every passing day, more public money is being wasted and more patients are being harmed."
"Hopefully Congress will keep focused on this issue until FDA and NIH finally enforce the law.”
The full report can be downloaded as PDF below. See also the separate academic preprint and the full dataset listing all law-breaking paediatric clinical trials together with the companies and institutions responsible for making their results public.
What are you going to do about this?
TranspariMED currently receives zero core funding. Producing this report was only possible due to many hours of unpaid research performed by a team of TranspariMED volunteers. A big thank you to: Okan Basegmez, Ronak Borana, Belén Chavarría, Nick DeVito, Mayra Velarde, and Samruddhi Yerunkar.
Please make a donation today and join our fight to get all clinical trial results made public.