The Institute for Clinical and Economic Review (ICER), a Boston-based non-profit that evaluates the cost of medical treatments, has just published an Evidence Report assessing the comparative clinical benefit and value of Sarepta Therapeutics’ (SRPT -0.8%) Exondys 51 (eteplirsen) and golodirsen and PTC Therapeutics’ (PTCT -1.9%) corticosteroid Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD). The report will be reviewed at a public meeting of the New England Comparative Effectiveness Public Advisory Council in Cambridge, MA on Thursday, July 25.
The report cites the absence of “persuasive evidence” that exon-skipping therapies improve outcomes that matter to patients, including functional status, quality of life or length of life. The limited data that exists show that both drugs modestly increase dystrophin levels but the clinical significance of this is “uncertain.”
As far as cost is concerned (more than $1M/year in its model), eteplirsen would still be over-priced even if it restored DMD patients to perfect health for additional 40 years, adding that it is unable to calculate value-based price benchmarks for either medication due to the paucity of efficacy data.
It says that there is evidence that deflazacort, a corticosteroid developed years ago, delivers greater benefits with less risk than prednisone, but the U.S. price is way out of line, 50x higher than prices in other countries.
The value-based price benchmark range is no higher than $19,000 – 31,700 per year. Conceptually, the company would have to discount the wholesale cost as much as 83% to be in line with the model (price assumption: $81,400).
According to Endpoints News, representatives from both companies stated that they have not raised the prices of their medications and are unclear on the assumptions ICER used in its calculations.
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