Tag: synthetic control arms

Use of Historical Controls in Rare Disease Regulatory Submissions

August 27, 2025

Use of Historical Controls in Rare Disease Regulatory Submissions Leveraging Historical Controls in Orphan Drug Trial Designs Introduction: Why Historical Controls Matter in Rare Disease Trials Rare disease clinical trials frequently face recruitment challenges due to small patient populations, ethical concerns with placebo groups, or urgency in life-threatening conditions. In such contexts, historical controls—data from…

Rare and Orphan Disease Trials, Trial Design & Methodology

Selecting Appropriate Control Groups in Rare Disease Studies

August 25, 2025

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Selecting Appropriate Control Groups in Rare Disease Studies How to Choose Effective Control Groups for Rare Disease Trials Introduction: Why Control Group Selection is Crucial in Rare Disease Research In clinical research, the control group serves as a critical comparator to evaluate the safety and efficacy of a new treatment. In the context of rare…

Rare and Orphan Disease Trials, Trial Design & Methodology

Using Real-World Data to Inform Disease Progression in Rare Conditions

August 13, 2025

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Using Real-World Data to Inform Disease Progression in Rare Conditions Leveraging Real-World Data to Understand and Model Disease Progression in Rare Diseases Introduction: The Value of Real-World Data in Rare Disease Trials Understanding disease progression is one of the foundational steps in rare disease clinical research. However, the scarcity of patients, heterogeneity in symptoms, and…

Natural History Studies, Rare and Orphan Disease Trials