Dietary Management Critical in IEM Drug Trials, FDA Guidance Says

Optimizing dietary management during drug trials is critical to accurately assessing drug efficacy in clinical trials for inborn errors of metabolism (IEM), the FDA says in a new draft guidance.

Dietary management is the core avenue of treatment for a number of IEMs in which specific enzyme mutations prevent the breakdown of dietary components and subsequently lead to toxic metabolites and organ damage. 

The new eight-page draft guidance says trials that don’t adequately consider and brace for the impact of dietary changes may jeopardize the interpretability of their findings.

The FDA’s new guidance also provides advice on trial designs, dietary assessments (including challenges and limitations), the use of historical controls, and considerations for planned surgeries and other procedures, which IEM patients often undergo and must increase caloric intake for.

Comments are due on the guidance by Sept. 11.

Read the full draft guidance here.

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