Expanding Patient Access to Investigational Drugs

• Published in: JACC. Basic to translational science Vol. 3; no. 2; pp. 280 - 293
• Main Author: Van Norman, Gail A., MD
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 01.04.2018; Elsevier
• Subjects: Cardiovascular; compassionate use; EIND; emergency IND; expanded access; right-to-try; single-patient IND; investigational new drug (filing); FDA; letter of authorization; expanded access; emergency IND; U.S. Food and Drug Administration; TIND; compassionate use; institutional review board; EAP; R2T; IRB; EIND; right-to-try; single-patient IND; emergency investigational new drug; IND; expanded access program; LOA; treatment investigational new drug
• ISSN: 2452-302X; 2452-302X
• DOI: 10.1016/j.jacbts.2017.11.007

Summary With drug approval times taking an average of 8 years from entry into clinical trials to full U.S. Food and Drug Administration (FDA) approval, patients with life-threatening and severely debilitating disease and no reasonable therapeutic options are advocating for expanded access (EA) to investigational drugs prior to approval. Special investigational new drug (IND) application categories allow patients who meet specific criteria to receive treatment with non-approved drugs. 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.