The Institute for Clinical and Economic Review (ICER) in its latest evidence report on treatments for Duchenne muscular dystrophy suggests that PTC Therapeutics’ deflazacort would need to see a roughly 73 percent discount to be cost-effective.
ICER in its report reviewed Sarepta Therapeutics’ eteplirsen and golodirsen, and PTC Therapeutics’ deflazacort.
“Unfortunately, there is no persuasive evidence that these exon-skipping therapies improve outcomes that matter to patients, including functional status, quality of life, or length of life,” David Rind, ICER’s chief medical officer, said in a statement.
Rind said despite eteplirsen having been on the market for three years, there remains “notably inadequate data on patient outcomes.”
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