FDA Most Expensive Medicine

FILE - This Aug. 13, 2016, file photo shows a logo of Swiss pharmaceutical company Novartis in Seoul, South Korea. On Friday, May 24, 2019, U.S. regulators approved Zolgensma, the most expensive medicine ever, a therapy meant to cure a disorder that rapidly destroys a baby's muscle control and kills most within a couple years. (AP Photo/Ahn Young-joon, File)

The record $2.1 million price tag for Novartis’ gene therapy Zolgensma — a one-time treatment that was approved by the Food and Drug Administration recently for a deadly childhood disease — evokes two very different responses. Critics see out-of-control pricing behavior. Supporters say the sticker-shocked are ignoring the creation of a possible cure for a disease that kills children before their second birthday.

They’re both valid reactions. But we’re heading toward a point where spiraling prices on gene therapies threaten to hamper access or effectively ration usage by income or coverage quality. The problem isn’t just Zolgensma. It’s the dozens of other coming gene therapies that will use this price as a reference point. Miracle cures don’t do much good if they aren’t accessible.

There’s an argument for premium pricing for gene therapies; for one, they could replace a lifetime of costly alternative treatments in one go. Zolgensma costs $425,000 annualized over five years. That’s a discount relative to prolonged use of the current noncurative standard of care, Biogen’s Spinraza.

But pharma has a thumb on the scale. Drug prices have skyrocketed, particularly for the rare conditions targeted by many gene therapies. Charging several times an already inflated price isn’t as much of a deal. The next generation of gene therapies will be even more effective. How much will they cost? It’s easy to see a path from more approvals at higher prices to an increase in barriers to access, even if gene therapies deliver long-term savings.

Providers of private health insurers don’t have a robust incentive to think ahead; they’re typically focused on annual budgets and are judged by quarterly earnings, while Americans swap coverage so frequently that they may never see the promised overall savings from these treatments filter down to the cost of their own plans.

Another issue with the price of these treatments and insurers’ willingness to pay it is that they haven’t completely proved themselves as one-and-done cures. BioMarin Therapeutics released updated data on Tuesday for its hemophilia gene therapy Valrox, which could cost as much or more as Zolgensma. The medicine can eliminate harmful bleeds for many patients, but there are concerns that the results won’t last. Paying a huge upfront price for a benefit that’s uncertain in several ways isn’t enormously appealing.

It’s too hard for drugmakers and insurers to agree on contracts that spread the cost of drugs out over time and promise refunds if they don’t work as promised. Part of that is on the government, which needs to remove regulatory barriers to such deals. We also have limited measures of value in health care, leaving pharma to create its own poorly calibrated scale. The fragmentation of insurance coverage also deserves plenty of blame, as it makes fighting back exceptionally difficult.

Fixing these issues won’t be easy or quick. But the time to start is now, rather than when seven-figure price tags aren’t remarkable anymore.

Max Nisen is a Bloomberg Opinion columnist covering biotech, pharma and health care.

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