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Long-chain fatty acid oxidation disorders (LC-FAODs) represent a group of rare inborn errors of metabolism characterized by acute crises of energy metabolism and severe energy deficiency. Clinical manifestations include rhabdomyolysis, liver dysfunction, severe hypoglycemia, and cardiomyopathy. The symptoms associated with these genetic disorders may result in frequent hospitalizations and early death. Many patients with LC-FAODs experience major clinical events and significant morbidity and mortality that portends a substantial need for better therapeutic options for these disorders. Triheptanoin was recently approved for the treatment of LC-FAODs. As with any novel agent for rare diseases, the treatment adoption and coverage criteria for rare disease therapy such as this presents challenges for managed care decision makers. Both managed care decision makers and specialty pharmacists must ensure that healthcare professionals provide appropriate patient and caregiver education surrounding the management of LC-FAODs, including vital information on adopting emerging agents into the therapy armamentarium for these disorders.

Long-chain fatty acid oxidation disorders: managed care and specialty pharmacy implications