Recognizing Rett Syndrome: Managed Care Insights on Disease Burden

This activity was originally broadcast live as a symposium at AJMC® Day of Education 2022.

Program Description

Rett syndrome (RTT) is a neurodevelopmental disorder that primarily affects females, causing physical regression and loss of motor control throughout the body, resulting in numerous comorbidities and complications. Mutations in the X-linked MECP2 gene are present in nearly 99% of girls and women with a confirmed diagnosis of RTT. Though there are no currently approved treatments for RTT, the development of therapeutics has focused primarily on addressing downstream effects of the MECP2 mutation or targeting MECP2 directly. As treatments approach potential approval, early identification of RTT characteristics and appropriate diagnosis will increase efficiency of care, improve outcomes, and reduce unnecessary costs for caregivers of girls and women with RTT. Managed care professionals must reflect on the individual costs associated with comorbidities and complications of RTT and plan for treatment protocols using evidence-based medicine to reduce health care resource utilization. This program will present available safety and efficacy data on emerging therapies for RTT, considerations for determining their potential place in the treatment of RTT symptoms and disease management, and guidance to ensure availability and accessibility of these future therapies.

PTCE aims to present the most updated information available. Since the launch of this program, trofinetide was approved in March 2023 by the FDA for the treatment of Rett syndrome in adults and children aged 2 years and older. Please refer to the prescribing information for additional information and join us in person or virtually at the Asembia Specialty Pharmacy Summit in May 2023 for additional updates on the treatment landscape in Rett syndrome.

Target audience: Specialty and managed care pharmacists

Type of activity: Application
Release date: November 1, 2022
Expiration date: November 1, 2023

Time to complete activity: 1.5 hours
Fee: Free

Educational Objectives

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

• Identify signs, symptoms, and diagnostic criteria for Rett syndrome (RTT) and disease complications associated with specific phenotypes.
• Analyze clinical efficacy and safety data of emerging treatments for RTT and the role of MECP2 as a therapeutic target.
• Examine the economic and quality of life burden of RTT for patients and caregivers as well as potential interventions managed care pharmacists can assume responsibility for to reduce health care resource utilization.

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 1.5 contact hours (0.15 CEU) under the ACPE universal activity number 0290-0000-22-541-H01-P. The activity is available for CE credit through November 1, 2023.

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

Alan K Percy, MD, has the following financial relationships with commercial interests to disclose:

• Grant/Research support: Acadia Pharmaceuticals
• Consultant on medical advisory boards: Acadia Pharmaceuticals, Neurogene

Kavita V. Nair, PhD, FAAN, has the following financial relationships with commercial interests to disclose:

• Consultant: Bristol Myers Squibb, Novartis, TG Therapeutics, PhRMA Foundation
• Speakers Bureau: Sanofi-Genzyme, Alexion
• Research grants: Genentech, PhRMA Foundation, Bristol Myers Squibb, Novartis

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

Pharmacy Times Continuing Education™ Planning Staff—Jim Palatine, RPh, MBA; Maryjo Dixon, RPh; Michelle C. Carlson, PharmD; Kylie Ferrentino; Susan Pordon; Brianna Winters; and Chloe Taccetta.

Anonymous peer reviewers were used as part of content validation and conflict resolution. The peer reviewers have 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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