Cost-Effectiveness Analysis of Eladocagene Exuparvovec-tneq for Aromatic L-Amino Acid Decarboxylase Deficiency (AADC-D) and Comparison with Other Rare Disease One-Time Treatments in the United States
Author(s)
Berrin Monteleone, MD1, Rongrong Zhang, MSc2, Paul Castellano, Jr., MBA3, Thomas OConnell, BA, MA4, Yixi Teng, MS4, Ioannis Tomazos, MBA, PhD3;
1NYU Long Island School of Medicine, New York, NY, USA, 2PTC Therapeutics Sweden AB, Askim, Sweden, 3PTC Therapeutics Inc, Warren, NJ, USA, 4Medicus Economics, Boston, MA, USA
1NYU Long Island School of Medicine, New York, NY, USA, 2PTC Therapeutics Sweden AB, Askim, Sweden, 3PTC Therapeutics Inc, Warren, NJ, USA, 4Medicus Economics, Boston, MA, USA
Presentation Documents
OBJECTIVES: AADCd is a rare, infantile-onset, neurometabolic disorder, characterized by motor dysfunction and developmental delays. In November 2024, the one-time gene therapy eladocagene exuparvovec-tneq received accelerated approval from the US Food and Drug Administration. This study aimed to assess the cost-effectiveness of eladocagene exuparvovec-tneq compared to best supportive care (BSC) from a US payer perspective.
METHODS: Multi-state modeling was implemented to track disease progression from a “no motor function” health state to achievement of meaningful improvements in motor function, as measured by: (1) multiples of the meaningful score difference (MSD) for Total Peabody Developmental Motor Scales-Second Edition (PDMS-2) score (previously estimated at 40 points); and (2) motor milestones (full head control, sitting unassisted, standing with support, walking with assistance). Three single-arm, open-label eladocagene exuparvovec-tneq trials informed clinical inputs. Health-state utilities were from a US time-trade-off study that valued the quality-of-life impact of AADCd by motor-milestone state. Costs and quality-adjusted life years (QALYs) were estimated undiscounted and discounted (at 3% annually). Results were compared to 16 CEAs of 33 other rare disease and/or one-time treatments conducted by the Institute for Clinical and Economic Review (ICER) and compared to results for eladocagene exuparvovec-tneq vs. BSC.
RESULTS: Compared to BSC, incremental QALYs for eladocagene exuparvovec-tneq were 43.87 (undiscounted) and 20.83 (discounted) based on multiples of the MSD for Total PDMS-2, and 38.36 (undiscounted) and 18.44 (discounted) based on motor milestone achievement. The incremental cost-per-QALY ranged from $99,682-$112,744 (undiscounted) and $199,007-$224,104 (discounted). In CEAs of other treatments reported by ICER, only 5 of 30 reporting a modified societal perspective achieved cost-per-QALY ≤$250,000, as was observed for eladocagene exuparvovec-tneq vs. BSC.
CONCLUSIONS: Eladocagene exuparvovec-tneq has substantial QALY gains compared to BSC and demonstrates superior cost-effectiveness relative to other rare-disease and one-time treatments. These findings highlight the transformative impact of this gene therapy for treating AADCd.
METHODS: Multi-state modeling was implemented to track disease progression from a “no motor function” health state to achievement of meaningful improvements in motor function, as measured by: (1) multiples of the meaningful score difference (MSD) for Total Peabody Developmental Motor Scales-Second Edition (PDMS-2) score (previously estimated at 40 points); and (2) motor milestones (full head control, sitting unassisted, standing with support, walking with assistance). Three single-arm, open-label eladocagene exuparvovec-tneq trials informed clinical inputs. Health-state utilities were from a US time-trade-off study that valued the quality-of-life impact of AADCd by motor-milestone state. Costs and quality-adjusted life years (QALYs) were estimated undiscounted and discounted (at 3% annually). Results were compared to 16 CEAs of 33 other rare disease and/or one-time treatments conducted by the Institute for Clinical and Economic Review (ICER) and compared to results for eladocagene exuparvovec-tneq vs. BSC.
RESULTS: Compared to BSC, incremental QALYs for eladocagene exuparvovec-tneq were 43.87 (undiscounted) and 20.83 (discounted) based on multiples of the MSD for Total PDMS-2, and 38.36 (undiscounted) and 18.44 (discounted) based on motor milestone achievement. The incremental cost-per-QALY ranged from $99,682-$112,744 (undiscounted) and $199,007-$224,104 (discounted). In CEAs of other treatments reported by ICER, only 5 of 30 reporting a modified societal perspective achieved cost-per-QALY ≤$250,000, as was observed for eladocagene exuparvovec-tneq vs. BSC.
CONCLUSIONS: Eladocagene exuparvovec-tneq has substantial QALY gains compared to BSC and demonstrates superior cost-effectiveness relative to other rare-disease and one-time treatments. These findings highlight the transformative impact of this gene therapy for treating AADCd.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE402
Topic
Economic Evaluation
Disease
SDC: Rare & Orphan Diseases