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Published on: February 14, 2019
Association between pediatric clinical trials and global burden of disease
Florence T Bourgeois1, Karen L Olson, John P A Ioannidis
1Division of Emergency Medicine, Boston Children's Hospital, 300 Longwood Ave, Boston, MA 02115. florence.bourgeois@childrens.harvard.edu.
Insights
Pediatric clinical trials show a moderate link to disease burden, especially in low-income nations. This highlights neglected areas needing more research investment for child health.
Area of Science:
- Clinical research
- Pediatric health
- Global health disparities
Background:
- Research funding allocation should prioritize conditions with the highest disease burden.
- Pediatric populations are historically underrepresented in clinical research.
- Understanding research focus alignment with disease burden is critical for equitable resource allocation.
Purpose of the Study:
- To assess the association between pediatric clinical trial focus and disease burden.
- To identify clinical domains in pediatric research that are neglected relative to disease burden.
Main Methods:
- Cross-sectional study of clinical trials registered in ClinicalTrials.gov.
- Included patient-level intervention trials in pediatric populations initiated from 2006 onwards.
- Correlated the number of pediatric clinical trials with age-specific disease burden measures across 21 conditions in countries of varying income levels.
Main Results:
- Neuropsychiatric and infectious diseases led in trial numbers; injuries and maternal conditions had the fewest.
- A moderate correlation (r = 0.58) was found between global trial counts and disease burden.
- The correlation was weakest in low-income countries (r = 0.47), with injuries, nutritional deficiencies, and respiratory infections being significantly understudied relative to their burden.
Conclusions:
- Pediatric clinical trial activity is only moderately aligned with the burden of disease, with the weakest alignment in low-income countries.
- Significant discrepancies between research focus and disease burden highlight critical areas for targeted investment and research prioritization.
- Addressing these neglected areas is essential for improving global pediatric health outcomes.
Background:
The allocation of research resources should favor conditions responsible for the greatest disease burden. This is particularly important in pediatric populations, which have been underrepresented in clinical research. Our aim was to measure the association between the focus of pediatric clinical trials and burden of disease and to identify neglected clinical domains.
Methods:
We performed a cross-sectional study of clinical trials by using trial records in ClinicalTrials.gov. All trials started in 2006 or after and studying patient-level interventions in pediatric populations were included. Age-specific measures of disease burden were obtained for 21 separate conditions for high-, middle-, and low-income countries. We measured the correlation between number of pediatric clinical trials and disease burden for each condition.
Results:
Neuropsychiatric conditions and infectious diseases were the most studied conditions globally in terms of number of trials (874 and 847 trials, respectively), while intentional injuries (5 trials) and maternal conditions (4 trials) were the least studied. Clinical trials were only moderately correlated with global disease burden (r = 0.58, P = .006). Correlations were also moderate within each of the country income levels, but lowest in low-income countries (r = .47, P = .03). Globally, the conditions most understudied relative to disease burden were injuries (-260 trials for unintentional injuries and -160 trials for intentional injuries), nutritional deficiencies (-175 trials), and respiratory infections (-171 trials).
Conclusions:
Pediatric clinical trial activity is only moderately associated with pediatric burden of disease, and least associated in low-income countries. The mismatch between clinical trials and disease burden identifies key clinical areas for focus and investment.
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