The growth of gene and cell therapies (CGTs) and specialty pharmaceuticals represents a radical shift in the treatment of rare diseases. By changing the parameters for care coordination, such innovations are improving patient outcomes and leading to abetter quality of life. Treatments for rare diseases have been shown to provide more significant health benefits on average than can drugs that are formulated for more common conditions. Regrettably, over 95% of rare-disease patients in the United States lack a US Food and Drug Administration (FDA)-approved treatment for their condition.
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