Pediatric versus adult drug trials for conditions with high pediatric disease burden

Abstract

Background and objective: Optimal treatment decisions in children require sufficient evidence on the safety and efficacy of pharmaceuticals in pediatric patients. However, there is concern that not enough trials are conducted in children and that pediatric trials differ from those performed in adults. Our objective was to measure the prevalence of pediatric studies among clinical drug trials and compare trial characteristics and quality indicators between pediatric and adult drug trials.

Methods: For conditions representing a high burden of pediatric disease, we identified all drug trials registered in ClinicalTrials.gov with start dates between 2006 and 2011 and tracked the resulting publications. We measured the proportion of pediatric trials and subjects for each condition and compared pediatric and adult trial characteristics and quality indicators.

Results: For the conditions selected, 59.9% of the disease burden was attributable to children, but only 12.0% (292/2440) of trials were pediatric (P < .001). Among pediatric trials, 58.6% were conducted without industry funding compared with 35.0% of adult trials (P < .001). Fewer pediatric compared with adult randomized trials examined safety outcomes (10.1% vs 16.9%, P = .008). Pediatric randomized trials were slightly more likely to be appropriately registered before study start (46.9% vs 39.3%, P = .04) and had a modestly higher probability of publication in the examined time frame (32.8% vs 23.2%, P = .04).

Conclusions: There is substantial discrepancy between pediatric burden of disease and the amount of clinical trial research devoted to pediatric populations. This may be related in part to trial funding, with pediatric trials relying primarily on government and nonprofit organizations.

FIGURE 1

Kaplan-Meier curves for the cumulative probability of publication for randomized controlled trials. A, Probability of publication for the period beginning with the study start date. The time at which 25% of trials were published was 3.9 years (95% CI: 3.2–4.2) for pediatric trials and 4.4 years (95% CI: 4.1–4.7) for adult trials. B, Probability of publication for the period beginning with the study completion date. The time at which 25% of trials were published was 2.0 years (95% CI: 1.7–2.3) for pediatric trials and 2.4 years (95% CI: 2.1–2.7) for adult trials.