Fri. Feb 3rd, 2023
Old, generic drug for rare disease gets new price tag: $89,000 a year

Remember when that pharmaceutical trade group launched a flashy ad campaign to convince consumers it was different from the industry’s price-gouging Shkrelis? Well, one of the members just took an old cheap drug that cost $89,000 for a year.

The steroid drug, deflazacort, which treats Duchenne muscular dystrophy, has been approved abroad for years and is sold as a generic drug. Families here imported for about $1,200 a year.

But Marathon Pharmaceuticals (a member of the PhRMA trade group) finally got it approved by the FDA on Thursday under “orphan drug” status, which covers drugs that treat rare diseases. (Duchenne affects about 15,000 people in the US.) Under that status, Marathon has exclusive rights to sell deflazacort in the US for seven years.

Like other pharmaceutical companies that have high list prices for their drugs, Marathon CFO Babar Ghias told the Washington Post that the list price is not what a patient will pay. The price drops to $54,000 after discounts and rebates, he said. And then insurance and financial programs ensure that most patients will pay little or nothing out of pocket.

Aaron Kesselheim, an associate professor of medicine at Harvard Medical School, told the After:

Instead of making the price at a level that is reasonable for patients, they make it a very high price and offer this avenue that patients may not be eligible for, they may not know, there may be limitations to it . So it’s a marketing move and not really a public health solution.

Marathon also received a voucher from the FDA to get the orphan drug approved. The voucher is intended to encourage pharmaceutical companies to develop treatments for rare diseases. However, the vouchers can be sold, often for hundreds of millions of dollars.

Other medicines treat Duchenne muscular dystrophy, a severe form of muscular dystrophy that mainly affects boys (it is X-linked recessive). Those with the disease usually lose the ability to walk in their teens and have a life expectancy of about 25 to 30 years. Medications can slow the loss of muscle strength. But some think deflazacort (brand name Emflaza) is the most effective and has the mildest side effects.

By akfire1

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