The US Food and Drug Administration (FDA) has issued a revised draft guidance on developing drugs to treat inborn errors of metabolism (IEM) to offer clarification on how such treatments should be evaluated in conjunction with dietary management, considerations regarding trial subjects from different countries and on the use of control groups.
IEM are a group of disorders where the metabolic pathways fail to properly break down or store carbohydrates, fatty acids or proteins. The disorders can be inherited or result from spontaneous mutations.
To help sponsors developing drugs for such disorders, FDA published a draft guidance in 2018 outlining its thinking on topics such as clinical trial design, taking into consideration some of the challenges of running clinical trials in the IEM patient population, and other considerations, such as changes in dietary management. After reviewing the comments it received on the earlier draft guidance, FDA issued a revised draft guidance on 11 July to offer some additional clarifications. (RELATED: Diet in Clinical Trials: FDA Drafts Guidance, Regulatory Focus 23 July 2018)…
