Objective: The gene therapy voretigene neparvovec (VN) is the first Food and Drug Administration-approved treatment for vision loss owing to the ultra-rare RPE65-mediated inherited retinal disorders. We modeled the cost-utility of VN compared with standard of care (SoC). Study Design: A 2-state Markov model, alive and dead, with a lifetime horizon. Methods: Visual acuity (VA) […]
— Updated cost-recovery benchmark price now framed as a range with maximum of $1,600 per 10-day regimen when incorporating assumptions regarding manufacturer’s 2020 development expenses, uptake of remdesivir, and consideration of early examples of real-world generic pricing — — Highlighted “cost-effectiveness” benchmark price shifts modestly higher to a range from $4,580 to $5,080 based on […]
— Models present prices according to two different paradigms: “cost recovery” approach and traditional cost-effectiveness analysis — — Pricing estimates based on preliminary data and will be updated regularly as further data are released and clinical use evolves to include earlier treatment; models will also be used to provide pricing estimates for future treatments as […]
— Public meeting reveals tensions inherent around high-priced treatments that lack adequate evidence of efficacy — BOSTON, August 15, 2019 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance assessing the comparative clinical effectiveness and value of the corticosteroid deflazacort (Emflaza®, PTC Therapeutics), and two exon-skipping therapies — eteplirsen […]
— No price can be suggested as a fair value-based price for eteplirsen or golodirsen because no persuasive evidence yet exists to demonstrate the clinical effectiveness of either drug — — For deflazacort, discounts of at least 73% from its list price would be needed to achieve commonly cited thresholds for cost-effectiveness — — As […]
— Independent appraisal committee notes family testimony in votes confirming broad benefits of both treatments; however, committee votes unanimously that price for Spinraza is too high to align fairly with these benefits, and urges fair pricing for emerging gene therapy Zolgensma to support sustainable access to innovation — BOSTON, April 3, 2019 – The Institute for Clinical […]
–Current pricing of Spinraza would require a substantial discount to meet traditional cost-effectiveness ranges; Zolgensma’s value-based price range is between $310,000-$900,000 using standard methodology but as high as $1.5 million using alternative measures of health gain– –As with all treatments for ultra-rare conditions, judgments of overall value require consideration of contextual issues and broader benefits […]
— Appraisal committee vote reveals differences in judgments on value given pricing far above traditional cost-effectiveness levels — –Policy recommendations call attention to the growing stream of treatments for rare disorders that cannot all be priced at similar levels without threatening the financial sustainability of the health system — BOSTON, February 14, 2018-The Institute […]
Independent appraisal committee voted that evidence is not adequate to demonstrate a superior net health benefit for ublituximab when compared to other monoclonal antibodies.
— Evidence is rated as insufficient to differentiate the net health benefit between ublituximab and other monoclonal antibodies; analyses suggest ublituximab and all currently available monoclonal antibodies labeled for MS would achieve common thresholds for cost-effectiveness if priced between $16,500- $34,900 per year, a range far lower than current market prices net of all rebates […]