Title |
Expanding Patient Access to Investigational Drugs Single Patient Investigational New Drug and the “Right to Try”
|
---|---|
Published in |
JACC: Basic to Translational Science, May 2018
|
DOI | 10.1016/j.jacbts.2017.11.007 |
Pubmed ID | |
Authors |
Gail A. Van Norman |
Abstract |
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. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 4 | 44% |
Chile | 1 | 11% |
Netherlands | 1 | 11% |
Spain | 1 | 11% |
China | 1 | 11% |
United Kingdom | 1 | 11% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 5 | 56% |
Scientists | 2 | 22% |
Science communicators (journalists, bloggers, editors) | 2 | 22% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 37 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 12 | 32% |
Student > Ph. D. Student | 6 | 16% |
Researcher | 2 | 5% |
Student > Bachelor | 2 | 5% |
Lecturer > Senior Lecturer | 1 | 3% |
Other | 2 | 5% |
Unknown | 12 | 32% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 10 | 27% |
Biochemistry, Genetics and Molecular Biology | 4 | 11% |
Pharmacology, Toxicology and Pharmaceutical Science | 3 | 8% |
Agricultural and Biological Sciences | 2 | 5% |
Nursing and Health Professions | 1 | 3% |
Other | 4 | 11% |
Unknown | 13 | 35% |