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. 2025 Feb 27;9(2):102714.
doi: 10.1016/j.rpth.2025.102714. eCollection 2025 Feb.

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

Stacey A Fedewa  1   2 , Leonard A Valentino  3 , Andee Koo  1 , Lorraine Cafuir  1   2 , Theresa W Gillespie  1 , Tyler W Buckner  4 , Duc Q Tran  5 , Ana Antun  1   2 , Christine L Kempton  1   2
Affiliations

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

Stacey A Fedewa et al. Res Pract Thromb Haemost. .

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.

Keywords: clinical trials; global health; health equity; hemophilia.

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Figures

None
Graphical abstract
Figure 1
Figure 1
The proportion of hemophilia clinical trial sites according to World Bank Income Categories (2007-2022). HIC, higher-income country; LMIC, lower-middle–income country; UMIC, upper middle–income country. There were no trials conducted in low-income countries.
Figure 2
Figure 2
Proportion of hemophilia clinical trial sites according to World Health Organization global regions (2007-2022). EMR, Eastern Mediterranean Region; SEAR, South East Asian Region; WPR, Western Pacific Region.
Figure 3
Figure 3
Randomized clinical trial density and factor utilization among 44 countries with trial and factor data.
See this image and copyright information in PMC

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