Advancements in AI, machine learning, and computing have revolutionized rare disease drug development through in silico modeling. These computational models simulate clinical trials by generating synthetic patients, which is especially valuable for rare diseases with small patient populations. In silico trials, which integrate biological and physiological data, can accelerate novel therapies' development and approval. This approach offers an alternative to randomized controlled trials, which can be impractical or unethical for rare diseases. The white paper highlights real-world examples and the potential of in silico methods to transform drug development.

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