iINDs and inborn errors of metabolism

Posted by & filed under Treatment.

In the recent edition of Molecular Genetics and Metabolism, P. Dickson and J. Tolar discuss the potential of individual investigational new drug applications (iINDs) for N-of-One therapeutic trials of experimental therapies in inborn errors of metabolism (Mol Gen Metab 116 (2015):1-3). They discuss “pros”: faster bench to bedside translation, treating life-threatening conditions with no other therapeutic options, and “cons”: introducing risk to drug approvals, diminish patient pools for larger, controlled studies.

In the end, my take-away from this commentary is that that iINDs should be approached with a cautious optimism in carefully selected therapeutics for patients for inborn errors of metabolism. I also feel strongly that results of these N-of-One therapeutic trials, whether effective or not, should be made available to the metabolic community to guide further therapeutic decision making.

Hilary Vernon, MD PhD

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