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1. In this cost analysis, compared to standard care, it was found that gene therapy for sickle cell disease (SCD) is cost-effective at a societal level based on two simulation models. 2. Acceptable value-based prices (VPBs) for SCD gene therapy ranged from $1 million to $2.5 million. Evidence Rating Level: 2 (Good) Study Rundown: SCD
Lifetime reduction in acute pain episodes should offset high upfront costs of the therapy
This new economic valuation of the societal and health care benefits of gene therapy to treat sickle cell disease (SCD) concludes the highly personalized treatment has potential to be cost effective if priced below $2 million.
<p>A cross-sectional study of more than 6,500 adults aged 65 years and older found that, on average, older adults spent 3 weeks each year getting health care outside the home. In this nationally representative group, 11% of older adults spent 50 or more days each year receiving health care services. These days spent obtaining health care outside the home, or health care contact days, are a new patient-centered metric for evaluating care for older adults. The study is published in <em>Annals of Internal Medicine. </em></p>
Incremental cost-effectiveness ratios were $126,000 and $281,000 per QALY for 2 simulation models from a societal perspective.