Recognizing Rett Syndrome: Managed Care Insights on Disease Burden

Recognizing Rett Syndrome: Managed Care Insights on Disease Burden is organized by Pharmacy Times Continuing Education (PTCE).

Release date: November 1, 2022
Expiration date: November 1, 2023

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 the 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 the 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 healthcare 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 the availability and accessibility of these future therapies.

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.