Glucocorticoid-prescribing trends in congenital adrenal hyperplasia, 2017 to 2023

This study investigates the utilisation of modern glucocorticoid medications (Acecort®, Alkindi®, Efmody®, and Plenadren®) for congenital adrenal hyperplasia due to 21-hydroxylase deficiency, examining prescribing patterns, barriers to adoption, and geographical and temporal trends.

A two-part study was conducted: a retrospective analysis of treatment regimens from the International Congenital Adrenal Hyperplasia Registry across 46 centres in 20 countries (2017-2023) and a qualitative survey of 39 centres regarding barriers to prescribing modern medications. Patients included both paediatric and adult populations. Data analysed included regional prescription trends, timing of modern glucocorticoid adoption, and identified barriers.

From 2017 to 2023, 44 of 790 (5%) patients transitioned from traditional to modern glucocorticoid therapy, with the highest adoption in high-income Western European countries. Alkindi® was exclusively prescribed to patients under 8 years, while 97% of Efmody® users were 7 years or older. By 2023, modern glucocorticoid availability varied among centres: Alkindi® (54%), Efmody® (46%), Plenadren® (33%), and Acecort® (15%).

Adoption of modern glucocorticoid medications for congenital adrenal hyperplasia remains limited, with only approximately 5% of patients transitioning from traditional therapies. Significant barriers include legislative approval, supply chain challenges, and elevated costs.

This international study looked at how new medications for congenital adrenal hyperplasia are used globally. We found that despite increasing availability of new medications during the study time period, only a small number of patients (5%) switched to these newer treatments. This limited use is mainly due to high costs, problems with getting legal approval, and supply issues, highlighting unequal access to care worldwide.