Author + information
- Gail A. Van Norman, MD∗ ()
- ↵∗Address for correspondence:
Dr. Gail A. Van Norman, Department of Anesthesiology and Pain Medicine, University of Washington, 2141 8th Avenue West, Seattle, Washington 98119.
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.
Dr. Van Norman has received financial support from the Journal of the American College of Cardiology.
The author attests he is in compliance with human studies committees and animal welfare regulations of the authors' institutions and Food and Drug Administration guidelines, including patient consent where appropriate. For more information, visit the JACC: Basic to Translational Science author instructions page.
- Received November 14, 2017.
- Accepted November 14, 2017.
- 2018 The Author
- Investigational New Drug INDs
- FDA Expanded Access INDs for Nonapproved Drugs
- The Single Patient IND
- Applying for an Individual Patient IND
- Accessing Investigational Drugs Without the FDA: “Right-To-Try” Legislation
- Obtaining Access to Drugs Under R2T
- Concerns About R2T
- Comparing Single Patient IND Versus R2T Legislation