Most clinical trials involving American children that violated FDAAA legal reporting requirements had not published outcomes in the scientific literature

Abstract

Non-publication, incomplete publication and excessively slow publication of clinical trial outcomes contribute to research waste and can harm patients. In this cohort study, we used public ClinicalTrials.gov registry records to identify 81 paediatric clinical trials in the United States that appear to have violated the FDA Amendments Act 2007 (FDAAA) reporting requirements. We then searched the literature for the outcomes of these 81 trials and contacted trial sponsors about the status of results. We found that only 22/81 trials (27.2%) had made their results public in a full-length peer-reviewed publication, and that only 8/81 (9.9%) trials had done so within one year of their primary completion date. Our findings highlight the need for US Food and Drug Administration and the National Institutes of Health to systematically monitor FDAAA compliance and enforce reporting requirements.

Regulatory gaps and research waste in clinical trials involving women with metastatic breast cancer in Germany

“…Our use of a methodology standardised across multiple projects and cohorts of clinical trials 24 enabled us to increase the efficiency of protocol development, project management and results reporting. We urge other researchers in this field to integrate outreach to trial sponsors into their methodologies, both to improve the quality of their own data 25 and to drive improvements in actual reporting performance.…”

“…16 Under German law, sponsors that fail to do so risk a fine of up to 25,000 Euros. 17 Depending on the type of CTIMP, either BfArM or Paul-Ehrlich-Institut (PEI) is the responsible German regulator, with BfArM regulating the large majority of CTIMPs. Thanks to strong engagement by both regulators, future compliance with CTIMP reporting requirements is likely to be very strong.…”

“…18 This provision has been integrated into German law. 17 However, the European Commission has repeatedly delayed the full launch of Eudamed, leaving sponsors in Germany and elsewhere in Europe confused about regulatory expectations. German device studies falling within the scope of the European regulation are overseen by BfArM, which seems likely to face an uphill struggle in securing compliance in coming years.…”

“…There is no legal requirement to make public the results of such trials under European law or German federal law. Those trials are instead regulated by provisions set out individually by Germany's 16 federal states, none of which appears to mandate or monitor results reporting 17. Therefore, it is currently completely legal in Germany for sponsors of such trials to recruit and treat patients and later not make study results public.…”

Global research landscape and burden of disease in pediatrics: Identifying the research gaps to set directions for a new general pediatric journal

“…Nineteen articles presented different tools or methods for evaluation, prediction, or knowledge translation. Two articles on research methodology, one about Bayesian methods for adapting trial data from adults to pediatric populations 10 and one about the violations of requirements in clinical trials, 11 were also among the latest preprints.…”