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 countr

SUBMITTER: Fedewa SA 

PROVIDER: S-EPMC11992419 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature