Summary for newly updated draft guidance USFDA - Inborn errors of metabolism that use dietary management considerations for optimising and standardizing diet in clinical trials for drug product development

Introduction:

The newly updated guidance published by food and drug administrations current recommendations for optimising and standardizing dietary management in clinical trials for drug products intended treat inborn errors of metabolism when dietary management is important for metabolic control.

The guidance does not address scenarios in which directory optimisation and standard digestion maybe invisible like diseases with prominent neuro psychiatric symptoms.

The updated guidance (July 2023) revises the draft guidance of the same name issued on July 24, 2018 this revision clarified that:

Drug products should be studied in conjunction with dietary management for conditions where dietary management is the current standard of clinical care.

The most informative design is a randomized double-blind clinical trial that includes a concurrent control group (approved drug or Placebo).

Dietary optimization should be based on dietary standards for the relevant population and account for severity of patients metabolic defect and the patients age, growth and general health status.

Background:

Dietary management is an important treatment modality and standard of care for several inborn errors of metabolism where specific in enzymatic defects result in reduced or absent metabolism of a variety of dietary components, with subsequent accommulation of toxic metabolites and organ damage. 

Dietary management involves restricting particular food components for example: 

🔖Restricting protein for patients diagnosed with urea cycle disorder organic acidemias

🔖Limiting food containing certain fats in patients diagnosed with some fatty acid oxidation defects

🔖Limiting carbohydrates in patients diagnose with certain primary mitochondrial diseases

🔖Removing dietary galactose in patients diagnose with classical galactosemia

🔖Limiting fennel Allen in patients diagnosed with phenylketonuria

Key Recommendations:

👍Some of the key recommendations provided by USFDA 

👍Optimising, standardizing and maintaining diet stability in clinical trials.

👍Protocols should specify or standardized approach to dietary management for anticipated intercurrent procedures, illnesses, and metabolic events that may occur during the trial.

👍Considering various aspects of dietary management in Design of clinical trial.

👍Sponsor should encourage families and patients to document there diet frequently during the trial to support the routine documentation obtained during the three days before clinical site visit

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