Real-World Treatment Patterns of Trofinetide in Rett Syndrome: Analysis of Medical Pharmacy-Linked Claims Database in the United States
Author(s)
Nazia Rashid, PharmD, MS1, Vinod Yakkala, PhD2, Mirko Sikirica, PharmD3, Daksha Gopal, PhD2, Kitty Rajagopalan, BS, MS, PhD4.
1Medical Affaits, ACADIA Pharmaceuticals, San Diego, CA, USA, 2Anlitiks, Inc., WINDERMERE, FL, USA, 3ACADIA Pharmaceuticals, San Diego, CA, USA, 4Anlitiks, WINDERMERE, FL, USA.
1Medical Affaits, ACADIA Pharmaceuticals, San Diego, CA, USA, 2Anlitiks, Inc., WINDERMERE, FL, USA, 3ACADIA Pharmaceuticals, San Diego, CA, USA, 4Anlitiks, WINDERMERE, FL, USA.
OBJECTIVES: Trofinetide (TROF) remains the first and only US FDA approved treatment for Rett syndrome (RTT). This study described the real-world TROF treatment and dosing patterns.
METHODS: A retrospective claims analysis of individuals with RTT diagnosis (ICD-10-CM: F84.2) using linked data from the IQVIA Anonymized Patient Level Data and TROF pharmacy (Rx) database during 01/01/2021 to 09/30/2024 was performed. RTT individuals initiating TROF (1st Rx=index date) between 04/01/23-03/31/24 with ≥6 months of post-index follow-up were evaluated for treatment patterns: persistent group (with allowable treatment gap of ≤90 days) and non-persistent group. Additionally, the % of restarts among non-persistent group were examined. Median (IQR) time on treatment among persistent and non-persistent groups were analyzed; among non-persistent group, time to restart was analyzed. Dosing patterns among the study groups were evaluated (initial dose, last dose, dose escalation).
RESULTS: Of the 1175 RTT individuals initiating TROF: 55% (n=645) were persistent, 45% (n=530) were non-persistent, and 8% (n=40) from the non-persistent group restarted TROF. The median (IQR) days on TROF for these groups was: 429 (137), 90 (113), and 121 (52) days. Mean (SD) age among persistent vs. non-persistent group was 14.7 (10.8) vs. 16.5 (11.6) years. The mean BID initial dose (mL) was 37.1 vs. 39.3 and the last dose was (mL) 37.7 vs. 40.9, respectively, among persistent vs. non-persistent groups. Mean BID dose titration (i.e., dose escalation) between initial and second TROF dose among persistent and non-persistent groups was 6.6mL vs. 10.2mL.
CONCLUSIONS: TROF individuals who were persistent were on TROF for ≥14 months. Mean initial dose was lower in the persistent vs. non-persistent group. The rate of dose escalation between initial and second TROF dose was lower in the persistent vs. non-persistent group. While titration occurred in both groups, the speed of titration in the non-persistent group warrants further investigation.
METHODS: A retrospective claims analysis of individuals with RTT diagnosis (ICD-10-CM: F84.2) using linked data from the IQVIA Anonymized Patient Level Data and TROF pharmacy (Rx) database during 01/01/2021 to 09/30/2024 was performed. RTT individuals initiating TROF (1st Rx=index date) between 04/01/23-03/31/24 with ≥6 months of post-index follow-up were evaluated for treatment patterns: persistent group (with allowable treatment gap of ≤90 days) and non-persistent group. Additionally, the % of restarts among non-persistent group were examined. Median (IQR) time on treatment among persistent and non-persistent groups were analyzed; among non-persistent group, time to restart was analyzed. Dosing patterns among the study groups were evaluated (initial dose, last dose, dose escalation).
RESULTS: Of the 1175 RTT individuals initiating TROF: 55% (n=645) were persistent, 45% (n=530) were non-persistent, and 8% (n=40) from the non-persistent group restarted TROF. The median (IQR) days on TROF for these groups was: 429 (137), 90 (113), and 121 (52) days. Mean (SD) age among persistent vs. non-persistent group was 14.7 (10.8) vs. 16.5 (11.6) years. The mean BID initial dose (mL) was 37.1 vs. 39.3 and the last dose was (mL) 37.7 vs. 40.9, respectively, among persistent vs. non-persistent groups. Mean BID dose titration (i.e., dose escalation) between initial and second TROF dose among persistent and non-persistent groups was 6.6mL vs. 10.2mL.
CONCLUSIONS: TROF individuals who were persistent were on TROF for ≥14 months. Mean initial dose was lower in the persistent vs. non-persistent group. The rate of dose escalation between initial and second TROF dose was lower in the persistent vs. non-persistent group. While titration occurred in both groups, the speed of titration in the non-persistent group warrants further investigation.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EPH205
Topic
Epidemiology & Public Health
Disease
Rare & Orphan Diseases