T

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 people’s cells — that carries, and in some views, deserves, a pricey list cost.

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  • It’s sad that we don’t find solutions such as these to be invaluable so that we can find a way to make them available to whomever need them instead of reaching a consumer price point that effectively creates a barrier for someone with less money.

  • “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.” Although these types of cost-effective analyses are informative, I don’t believe they should be used as the basis for drug pricing, especially regarding gene therapies for rare diseases. The article poses the right question: what are cures/significant advances to diseases worth to us as a society? The fact is, if Spark knew ahead of time that Luxturna would only yield them $153-$217k per patient, they probably wouldn’t choose RPE65-mediated IRD as an indication to target, and that disease would continue to cause those affected to invariably become blind for who knows how many more years. Instead, Luxturna should cost even more than $1M because I believe as a society we should value the pursuit of treatments with curative potential, and companies need to know that they will be justly rewarded for their efforts so that the best possible treatments are developed. Let’s not forget this. High drug prices are not universally a cause for outrage, but entirely context specific. Don’t compare what’s happening with Luxturna with the likes of Martin Shkrelli and those who aren’t actively committing R&D but are rather just buying old pharma assets and re-branding them at a higher price in order to make a profit.

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