A well-designed clinical trial program requires both toxicological and epidemiological evidence to provide a solid foundation for the clinical phase of drug testing and regulatory review, whereas ineffective or inadequate toxicology and epidemiology data could halt the progress of a potentially promising new drug. In particular, successful drug development requires a thorough understanding of the drug candidates’s pathophysiology and mechanism of action, as well as the targeted disease’s prevalence and natural history. Elucidating the epidemiology of a rare disease through real-world studies is particularly important, as requirements for nonclinical toxicology studies to support rare disease clinical programs may vary, depending on the impact of the rare disease on quality of life and mortality.

This white paper highlights important evidence attained from both toxicology and epidemiology studies for a successful orphan drug launch. ToxStrategies (www.toxstrategies.com) and EpidStrategies, a division of ToxStrategies (www.epidstrategies.com) have the necessary expertise to aid clients in their nonclinical and real-world data needs.