Why the study?
The recent surge in expensive novel drugs for rare cardiovascular diseases, especially cardiomyopathies and rare systemic diseases with cardiovascular involvement, raises concerns about global access equity. This study investigated international sales patterns of these medications across countries between 2015 and 2024.
Observational
Yes
Global consumption of novel rare cardiovascular disease medications, particularly for cardiomyopathies, has grown rapidly over the past decade but remains heavily skewed toward high-income nations rather than correlating with epidemiological disease burden.
Background In recent years, there has been a surge in novel cardiovascular drugs for rare cardiovascular disease (RCD), especially for cardiomyopathies and rare systemic diseases with cardiovascular involvement. However, given their high price, it raises concerns about global access equity. Purpose This study aims to investigate international patterns in the sales of medications for cardiomyopathies and rare systemic diseases with cardiovascular involvement across countries between 2015 and 2024. Methods Data from the IQVIA MIDAS database were used to characterise changes in the sales patterns of RCD medications across 66 countries. The sales data categorised by disease class were evaluated against national income levels and disease burden. The growth rate of medication sales was calculated. Lorenz curves and concentration indices were utilised to assess inequality in drug sales. Panel regression was conducted to examine the factors related to drug sales. Results Between 2015 and 2024, 66 of 83 (79.52%) countries and regions offered treatment for RCD, with 41 (62.12%) being high-income countries. Cardiomyopathy treatment sales demonstrated rapid annual growth (64.3%) compared to systemic disease treatments (7.9%) in recent years. Despite a rapid surge in sales of RCD medications, especially in lower-middle-income countries, the Lorenz curve showed that sales were heavily concentrated in regions with higher GDP per capita. For instance, the concentration index for cardiomyopathy treatments increased from -0.20 in 2015 to 0.55 in 2024, while systemic disease treatments demonstrated slightly improved distribution. Conclusion Despite increasing global volume of sales of RCD medications, there were existing significant socioeconomic disparities regarding access to RCD medications, particularly for cardiomyopathy treatments. Access to novel cardiomyopathy therapies is increasingly skewed toward high-income nations, and consumption is currently driven by national wealth rather than epidemiological burden, suggesting a gap between disease burden and treatment accessibility in less wealthy regions.
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Chou et al. (2026) conducted an observational in Rare cardiovascular diseases (cardiomyopathies and rare systemic diseases with cardiovascular involvement). Medications for rare cardiovascular diseases (cardiomyopathy treatments) vs. Systemic disease treatments / across national income levels was evaluated on Annual sales growth rate and inequality in drug sales (concentration index).
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