Glucocorticoid Prescribing Trends in Congenital Adrenal Hyperplasia, 2017 to 2023.
This study investigates the utilization of modern glucocorticoid medications (Acecort®, Alkindi®, Efmody®, 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 2017-2023, 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.
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 2017-2023, 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.
Authors
Roxas Roxas, Gihawi Gihawi, Makarchuk Makarchuk, Bryce Bryce, Chen Chen, Ahmed Ahmed, Ali Ali, Drake Drake, Casipe Casipe, Groves Groves, Idkowiak Idkowiak, Krone Krone, Flueck Flueck, Nordenström Nordenström, Reisch Reisch, Claahsen-van der Grinten Claahsen-van der Grinten, Adriaansen Adriaansen, Birkebaek Birkebaek, Hannema Hannema, Reilly Reilly, Cussen Cussen, Zaric Zaric, Neumann Neumann, Baronio Baronio, Vieites Vieites, Alonso Alonso, Elsedfy Elsedfy, Mazen Mazen, Thankamony Thankamony, Witczak Witczak, Rees Rees, Atapattu Atapattu, Seneviratne Seneviratne, Cools Cools, El Kaddouri El Kaddouri, Perez Perez, Guven Guven, Poyrazoglu Poyrazoglu, Fu Fu, Janus Janus, Globa Globa, Shenoy Shenoy, de Bruin de Bruin, Korbonits Korbonits, Adam Adam, Wasniewska Wasniewska, Russo Russo, Phan-Hug Phan-Hug, Bonfig Bonfig, Salerno Salerno, Tomlinson Tomlinson, Leka-Emiri Leka-Emiri, de Vries de Vries, Yarhere Yarhere, Guaranga-Filho Guaranga-Filho, van Eck van Eck, Bachega Bachega, Krone Krone, De Bono De Bono, Davies Davies, Segev-Becker Segev-Becker, Iotova Iotova, Lenherr-Taube Lenherr-Taube, German German, Giordano Giordano, De Sanctis De Sanctis, Probst Probst, Markosyan Markosyan, Brewer Brewer, Costa Costa, Webb Webb
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