Bridging Clinical and Access Gaps in Phenylketonuria: A Managed Care Perspective

Program Description

The lifelong and complex burden of phenylketonuria (PKU), a rare, inherited metabolic disorder, results from deficient phenylalanine hydroxylase (PAH) enzyme activity, which contributes to the build-up of the amino acid phenylalanine (Phe) in the brain. PKU is associated with substantial economic burden and unique challenges stemming from the cost of specialty medications and complex nutrition requirements. This webinar provides managed care professionals and pharmacists with information on PKU pathophysiology, and emphasis on the importance of early diagnosis via newborn screening, and the necessity of lifelong management to prevent irreversible neurocognitive and other long-term complications. Pharmacologic and lifestyle modifications will be reviewed, with a focus on nutritional requirements as well as current therapeutic options, expanding indications for adolescents, and new and emerging therapies. The discussion will also encompass considerations for managed care pharmacists, including high-priority issues, strategies to minimize delays in access to specialty medications, formulary decision-making, and ways to improve care coordination and provide patient support.

Target audience: Managed Care Pharmacist

Type of activity: Application

Release date: December 3, 2025

Expiration date: December 3, 2026

Time to complete activity: 1.0 hours

Learner level: Foundational

Fee: Free

Educational Objectives

At the completion of this activity, participants will be able to:

• Explain the underlying pathophysiology, clinical presentation, and lifelong burden of phenylketonuria (PKU)
• Ascertain knowledge of the PKU treatment landscape, including current standards of care and new therapeutic innovations
• Employ strategies to optimize access, coverage, and coordinated care for individuals with PKU to support improved patient outcomes