Drug development is an expensive and risky endeavor with an overall failure rate of ~85 % from the time drug candidates first enter the clinic to the time they reach the market. Add to this the complexity of a systemic disease with multiple proposed pathogenic mechanisms, no proven “gold standard” therapy against which to benchmark and no validated measures of disease activity, progression, or remission, and you have described a disease that one might deliberately steer away from for the purpose of developing new therapies. These diseases are often considered “intractable," and, in the absence of a major breakthrough on disease mechanism or clinical outcome assessment, they remain the territory of post-approval exploratory and investigator-initiated trials. It is in this context that this Chapter will provide a framework for understanding the preclinical, clinical, and regulatory challenges of drug development and provide insights to guide research and development strategies to advance novel drug discovery for SSc.
CITATION STYLE
Wright, T. M. (2016). Drug development and regulatory considerations for ssc therapies. In Scleroderma: From Pathogenesis to Comprehensive Management (pp. 595–601). Springer International Publishing. https://doi.org/10.1007/978-3-319-31407-5_43
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