Publications by authors named "D H Rind"
Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA.
Appl Health Econ Health Policy
December 2024
Article Synopsis
- - The study evaluates the cost-effectiveness of a new gene therapy, Etranacogene dezaparvovec (EDZ), for severe hemophilia B, comparing it to the traditional treatment, factor IX (FIX) prophylaxis over a lifetime perspective in the U.S.
- - Results show that despite EDZ costing $3.5 million, it offers lifetime savings of $11 million and slightly improves quality of life by 0.64 QALYs compared to FIX, which has very high annual costs.
- - The cost-effectiveness of EDZ is influenced significantly by capping annual cost offsets, emphasizing how these limits can help balance price and value for healthcare systems.
Background: Dopaminergic antipsychotics for schizophrenia have modest effects on symptoms and can cause important side effects. KarXT is an investigational drug for schizophrenia with a novel mechanism targeting muscarinic receptors that may limit these side effects.
Methods: We conducted a systematic review and Bayesian random-effects network meta-analyses of short-term RCTs (3-8 weeks) that enrolled adults with schizophrenia.
Objectives: To demonstrate how health technology assessment methods can be used to support Medicare's price negotiations for apixaban and rivaroxaban.
Methods: Following the statutory outline of evidence that will be considered by Medicare, we conducted a systematic literature review, network meta-analyses, and decision analyses to evaluate the health outcomes and costs associated with apixaban and rivaroxaban compared with warfarin and dabigatran for patients with nonvalvular atrial fibrillation. Our methods inform discussions about the therapeutic impact of apixaban and rivaroxaban and suggest price premiums above their therapeutic alternatives over a range of cost-effectiveness thresholds.