JACC. Basic to translational science
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JACC Basic Transl Sci · Apr 2018
Expanding Patient Access to Investigational Drugs: Single Patient Investigational New Drug and the "Right to Try".
With drug approval times taking an average of 8 years from entry into clinical trials to full U. S. ⋯ The FDA approves over 99% of all single-patient INDs, providing emergency approval within hours, and non-emergency approval within an average of 4 days. "Right-to-try" laws passed in 38 states would allow patients to bypass FDA processes altogether, but contain controversial provisions that some claim risk more harm than benefit to desperate and vulnerable patients. This review focuses on FDA EA to non-approved drugs through a special category of IND-the single-patient IND-and "right-to-try" (R2T) access outside of the FDA.