Yesterday (11 July, 2023) the USFDA's Center for Drug Evaluation and Research (CDER) released updated guidance on "Inborn Errors of Metabolism That Use Dietary Management: Considerations for Optimizing and Standardizing Diet in Clinical Trials for Drug Product Development".
FDA has developed this draft guidance to describe its recommendations for optimizing and standardizing dietary management in clinical trials for the development of drug products intended to treat inborn errors of metabolism (IEM). Diet plays a key role in the metabolic control of patients.
An inborn metabolic disorder (IEM), also known as inborn metabolic disorders or congenital metabolic diseases, is a genetic disorder that impairs a person's ability to utilize and process nutrients. There are various metabolic pathways that are affected by defects in enzymes or transport proteins.
Nutritional modifications are aimed at improving or restoring biochemical and physiological homeostasis by restricting the precursor nutrients that produce intermediate metabolic products that accumulate and cause toxicity.
Before entering clinical trials, sponsors should consider the following recommendations mentioned in the guidance for optimizing, standardizing, and maintaining patient diets. The accuracy of an evaluation of a new drug product's efficacy relies on optimizing and standardizing dietary management in these patients prior to and during clinical trials.
To know more about Clinical Trial Design, Challenges, and Limitations of Diet Assessments etc. check out this LINK.