In their commentary, Kruijshaar et al. (2025) express concerns about the fragmentation of real-world data on Pompe disease due to industry-driven registries. These registries often focus on specific products from one company, limiting the ability to compare treatments across different companies. The authors argue that this fragmentation hinders a comprehensive understanding of treatment effects for Pompe disease.
They advocate for independent registries, like the International Pompe Survey, which has been collecting patient-reported outcomes since 2002, free from industry influence. This approach is seen as essential for generating unbiased real-world evidence (RWE) that can inform post-marketing surveillance and improve understanding of various treatment options. The letter calls for a shift towards such independent data collection to overcome the issue of siloed information.
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