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

Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.0 contact hours (0.10 CEU) under the ACPE universal activity number 0290-0000-25-407-H01-P. The activity is available for CE credit through December 3, 2026.

Instructions for Completing the Activity and Receiving CPE Credit

To receive CPE credit, participants must complete the pretest, review the activity in its entirety, complete the posttest, and complete the online activity evaluation. After successful completion of the online activity evaluation, you can submit your credit to CPE Monitor. You may view your credit within 48 hours at www.mycpemonitor.net. All participants must request credit before the activity expiration date. CE credit will not be issued after this date.

Disclosures

The following contributors have no relevant financial relationships with commercial interests to disclose:

Faculty

Sofia Shrestha, PharmD, CSP

Pharmacy Times Continuing Education^™ Planning Staff—Jim Palatine, RPh, MBA; Adam C. Welch, PharmD, MBA, FAPhA; Macara Desai; Susan Pordon; Brianna Winters; and Chloe Taccetta have no relevant financial relationships with commercial interests to disclose.

At least one anonymous peer reviewer was used as part of content validation and conflict resolution. The peer reviewer has no relevant financial relationships with commercial interests to disclose.

Educational Disclaimer

CPE activities are offered solely for educational purposes and do not constitute any form of professional advice or referral. Discussions concerning drugs, dosages, and procedures may reflect the clinical experience of the author(s) or they may be derived from the professional literature or other sources and may suggest uses that are investigational in nature and not approved labeling or indications. Participants are encouraged to refer to primary references or full prescribing information resources.

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