Under the United States Food and Drug Administration's Expanded Access program, a physician may treat a single patient with an experimental medication under an individual investigational new drug application (iIND). Metabolic geneticists may be among the specialists most likely to be asked to obtain an iIND, because there are many experimental treatments for inborn errors ofmetabolismwhich work in animals but suffer delays in translation to the bedside. The iIND has the potential to help bridge that gap, by gathering initial evidence in support of the use of an experimental drug in humans. If donewith experimental rigor, the datawill be useful, despite being limited to a single patient. However, iINDsmay pose risks to drug development if patients are not carefully selected. Whatever their advantages and disadvantages, iIND studies are here to stay. Metabolic specialists should cautiously consider the iIND as a tremendous opportunity for therapeutic experimentation.
Bibliographical noteFunding Information:
Supported in part by NIH / NINDS R01NS085381 to P.I.D. and NIH / NCATS (UCLA CTSI) UL1TR000124 . J.T. is supported in part by grants from the National Institutes of Health ( R01 AR063070 and R01AR059947 ), US Department of Defense ( W81XWH-12-1-0609 ), Epidermolysis Bullosa Research Fund , Jackson Gabriel Silver Foundation , and DebRA .
The iIND is not designed for research, and phase I clinical trials are the standard in any drug development program. The National Institutes of Health and the Food and Drug Administration offer several funding mechanisms, including NeuroNEXT, Rare Disease Clinical Research Networks, and the Orphan Products Development grant program. These programs support clinical trials for novel therapies in patients with inborn errors of metabolism. Additional funding mechanisms for small clinical trials, including n = 1 studies for extremely rare diseases or patient-tailored therapeutics, are needed in order to bring the latest therapeutic options to patients.
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