Nd4 Comparison of Trinetx Electronic Medical Record Database and National Ambulatory Medical Care Survey: Findings on the Inappropriate Medications Use in Parkinson's Disease in the United States

Abstract

patient, resulting in cost savings and QALY gains compared to nusinersen; i.e., AVXS-101 was dominant over nusinersen. In a scenario where sitting gene therapy patients experienced survival trajectory of walking patients, AVXS-101 undiscounted QALYs were 57.5 (if discounted at 3%: 21.9) and AVXS-101 again dominated nusinersen at a price of $3M. Conclusions: AVXS-101 is cost-effective compared to nusinersen and may deliver up to 57.5 QALYs (undiscounted). Discounting QALYs at the US standard rate of 3% results in a substantial underestimate of health benefits. Decision makers such as NICE consider undiscounted QALY gain in certain cases. We recommend that US-based decision makers also consider undiscounted QALY gain when assessing the value of innovative therapies.