‘A Therapy Is Useless If No One Can Afford It’: $2M Drug Poised To Hit Market Stirs Up Debate Over Cost Of ‘Miracle’ Cures
The new treatment that has a potential $2 million price tag can cure spinal muscular atrophy, an inherited disease that typically kills babies before they turn two. But as more gene therapies hit the marketplace, insurers balk at the expense. Meanwhile, the FDA takes an unexpected step to introduce competition into the marketplace for an extremely pricey drug that treats a rare disease.
The Wall Street Journal:
A $2 Million Drug Is About To Hit The Market
A new treatment for an infant muscle-wasting disease is about to go on sale at a potential cost of $2 million, a record price tag likely to fuel the continuing scrutiny of how companies price their drugs and how insurers pay for them. Novartis AG has yet to set a price for the gene therapy called Zolgensma, but executives say the drug’s potential to cure spinal muscular atrophy, an inherited disease that typically kills babies before they turn two, justifies a seven-figure price. Gene therapies target diseases that result from a faulty gene by introducing a working version into the body. (Roland, 5/7)
Stat:
FDA Approval May Dampen Controversy Over A High-Priced Drug
The Food and Drug Administration just added an unexpected twist to a simmering controversy over a rare disease drug that earlier this year briefly became a poster child for high-priced medicines. In a surprise move, the agency approved a medicine from Jacobus Pharmaceuticals, a small, family-run company, for treating a neuromuscular disorder called Lambert-Eaton myasthenic syndrome, or LEMS, for children ages 6 to 17. However, the approval potentially adds unforeseen competition for Catalyst Pharmaceuticals (CPRX), which only last December won an FDA endorsement to market its own treatment for adults. (Silverman, 5/6)
Meanwhile, President Donald Trump pushes importation strategy as solution to cut costs —
Politico:
Trump Directs Azar To Work On Florida Drug Import Plan
President Donald Trump on Monday directed Health and Human Services Secretary Alex Azar to work with Florida Gov. Ron DeSantis on the state’s drug import plan, according to Rep. Matt Gaetz (R-Fla.). Trump and Azar spoke at an Oval Office meeting — attended by DeSantis — that lasted for about 45 minutes Monday afternoon, said Gaetz, who also was at the gathering. (Glorioso, 5/6)
