At $850,000, price for new childhood blindness gene therapy four times too high, analysis says
https://www.statnews.com/2018/01/12/price-gene-therapy-childhood-blindess/
he $850,000 list price for a new medicine that treats a genetic form of childhood blindness is about four times too high for the value the drug provides, a nonprofit that studies the cost-effectiveness of new drugs said Friday, though it added that the price of the drug is cost-effective for select patients and with certain assumptions.
The report from the Institute for Clinical and Economic Review focused on the medicine Luxturna, the first-of-its-kind gene therapy approved for the U.S. market and the most expensive medicine by list price. It is the latest flashpoint in the debate over how to afford an innovative medicine in this case, a therapy that corrects a genetic mutation in peoples cells that carries, and in some views, deserves, a pricey list cost.
In its report, ICER said a cost-effective price for Luxturna would be $153,000 to $217,000 a discount of 75 percent or more. ICER cited a lack of data that Luxturna causes permanent improvements in vision as a key reason that its developer, Spark Therapeutics, should not be charging so much.
While the evidence is clear the therapy improves vision for patients over several years, the long-term duration of this benefit remains unknown, Dr. David Rind, ICERs chief medical officer, said in a statement. Assuming a 10- to 20-year period of benefit, at list price the treatment does not meet standard cost-effectiveness thresholds, even after accounting for the broader societal benefits improved vision has on productivity and education costs.