Global patterns of hemophilia drug trials, hemophilia care, and health care measures

Abstract

Background

Drug trials are vital to establish safe and effective treatments for congenital hemophilia, a bleeding disorder that affects about 800,000 males worldwide. The global distribution of hemophilia drug trials (HDTs) and their alignment with hemophilia care is unknown.

Objectives

This study aimed to evaluate the global distribution of HDTs and its association with hemophilia care.

Methods

In this cross-sectional study, HDTs conducted between 2007 and 2022 were selected from the clinicaltrials.gov database. The density of trials per 1000 expected males with hemophilia (eMwH) was assessed according to hemophilia care measures (factor VIII and IX utilization per 1000 eMwH) derived from World Federation of Hemophilia data.

Results

Among 124 trials, 55 countries were represented, with an average of 7.9 countries per trial. Most HDT sites were in high-income (74.4%) or upper middle (20.1%)–income countries. The number of sites in lower-middle–income countries doubled, from 12 in 2007-2011 to 30 in 2017-2022—a nonsignificant increase from 5.8% to 7.0% (P = .53). Factor utilization was substantially reduced in lower-middle (0.4 international units [IUs] per 1000 eMwH) and upper middle (2.8 IUs per 1000 eMwH) compared with high (6.8 IUs per 1000 eMwH) income countries. HDT density was moderately correlated with factor usage (r = 0.436; P ≤ .001).

Conclusion

Most HDT sites were located in high-income countries, although a substantial proportion were in upper middle–income countries. A small but increasing number of trials were conducted in lower-middle–income countries, where factor usage is relatively low. This study provides evidence on the global distribution of HDT and raises questions regarding the generalizability, barriers, opportunities, and ethics of trials for a rare bleeding disorder.