Evaluating the Impact of Luxturna Gene Therapy on Vision Improvement and Disease Progression in Patients With RPE65 Mutation-Associated Inherited Vision Loss
Author(s)
Ina Kukreja, MBA, PT1, Arunima Sachdev, MA Economics2, Kulbhushan Tanwar, B.Tech3, Abhinav Nayyar, MBBA, MBA1, Shailaja Daral, MBA, MD1, Shakilur Rahman, PT3, Mohammad Talat Zaheer, MSc3, ARCHANA PRASAD, MBA3, Simran Pattanaik, MHA, BDS1, Rajat Rakesh Dewan, MBA, MBBS1, Vikash Kumar Verma, MBA, PharmD1, Abhimanyu Roy, MBA1, Rahul Goyal, BS Tech4, Louis Brooks Jr, MA5, Marissa Seligman, BS Pharma2.
1Optum, Gurgaon, India, 2Optum, Boston, MA, USA, 3Optum, Noida, India, 4Optum, Phoenix, AZ, USA, 5Optum, Bloomsbury, NJ, USA.
1Optum, Gurgaon, India, 2Optum, Boston, MA, USA, 3Optum, Noida, India, 4Optum, Phoenix, AZ, USA, 5Optum, Bloomsbury, NJ, USA.
Presentation Documents
OBJECTIVES: This study aims to investigate the impact of Luxturna, a novel gene therapy drug, on patients with inherited vision loss caused by mutations in both copies of the RPE65 gene. The research aims to provide insights into the potential effects of Luxturna on vision improvement, preservation and slowing down disease progression.
METHODS: A retrospective study was conducted using the Optum® Market Clarity Dataset, focusing on patients with claims or EHR records for Hereditary Retinal Dystrophy (HRD) between January 1, 2017, and March 31, 2024, identified using ICD-10 H35.5*. Identified patients were divided into two mutually exclusive groups: those who received Luxturna, using specific NDC (71394006501, 71394041501, 71394071601) and HCPCS (J3398) codes (Cases), and those who did not receive Luxturna (Controls). To ensure homogeneity between the cohorts, exact matching was applied based on demographic factors, age group, and comorbidity profile (CCI) in 1:3 ratio. Analysis covered a 6-month pre-Luxturna and a 24-month post-Luxturna period. Natural language processing was used to assess changes in vision, and outcomes of mobility and visual tests such as Visual acuity testing, Visual field testing, light sensitivity testing, and other tests along with developmental and educational needs status.
RESULTS: The study included 60 HRD patients with Luxturna and 191k HRD patients without Luxturna. Clinical notes of 10% of the cases were analyzed in which 100% showed preservation in vision, peripheral visual field, and ability to read in dim light. Patient’s visual distance holding improved from 20/400 pre-surgery to 20/250 post-surgery. Further comparison of healthcare resource utilization, provider specialty, and cost between the cohorts is planned.
CONCLUSIONS: Luxturna, a novel gene therapy approved by the US FDA in December 2017, that aims to help the cells in the retina function better and slowing down the progression of the disease. The findings of this study demonstrate encouraging outcomes.
METHODS: A retrospective study was conducted using the Optum® Market Clarity Dataset, focusing on patients with claims or EHR records for Hereditary Retinal Dystrophy (HRD) between January 1, 2017, and March 31, 2024, identified using ICD-10 H35.5*. Identified patients were divided into two mutually exclusive groups: those who received Luxturna, using specific NDC (71394006501, 71394041501, 71394071601) and HCPCS (J3398) codes (Cases), and those who did not receive Luxturna (Controls). To ensure homogeneity between the cohorts, exact matching was applied based on demographic factors, age group, and comorbidity profile (CCI) in 1:3 ratio. Analysis covered a 6-month pre-Luxturna and a 24-month post-Luxturna period. Natural language processing was used to assess changes in vision, and outcomes of mobility and visual tests such as Visual acuity testing, Visual field testing, light sensitivity testing, and other tests along with developmental and educational needs status.
RESULTS: The study included 60 HRD patients with Luxturna and 191k HRD patients without Luxturna. Clinical notes of 10% of the cases were analyzed in which 100% showed preservation in vision, peripheral visual field, and ability to read in dim light. Patient’s visual distance holding improved from 20/400 pre-surgery to 20/250 post-surgery. Further comparison of healthcare resource utilization, provider specialty, and cost between the cohorts is planned.
CONCLUSIONS: Luxturna, a novel gene therapy approved by the US FDA in December 2017, that aims to help the cells in the retina function better and slowing down the progression of the disease. The findings of this study demonstrate encouraging outcomes.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO141
Topic
Clinical Outcomes
Topic Subcategory
Clinician Reported Outcomes, Comparative Effectiveness or Efficacy
Disease
SDC: Sensory System Disorders (Ear, Eye, Dental, Skin), STA: Genetic, Regenerative & Curative Therapies