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The record $2.1 million price tag for Novartis AG’s gene therapy Zolgensma – a one-time treatment for a deadly childhood disease that was approved by the Food and Drug Administration on Friday – 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 threatens to hamper access or effectively ration usage by income or coverage quality. The problem isn’t just Zolgensma. It’s the dozens of other incoming gene therapies that will use this price as a reference point. Miracle cures don’t do much good if they aren’t accessible. (Max Nisen, 5/28)

Gilead Sciences, a California biopharmaceutical company, scored a major breakthrough in 2013 when the Food and Drug Administration approved Sovaldi, a pill that could cure hepatitis C when combined with another antiviral medication. And then Gilead delivered another breakthrough, albeit not a good one: It priced Sovaldi at $1,000 per pill, or $84,000 to $168,000 per course of treatment. The pricing stunned patients, insurers and state governments, which were suddenly exposed to billions of dollars in potential prescription costs. But as it turns out, Sovaldi was just a milepost on the way to the six- and seven-figure prices that a new generation of specialty drugs are expected to command. (5/23)

As soon as this summer, TV ads for prescription drugs are going to look a little different: the Trump administration recently finalized a rule that will require drug makers to show a medication’s list price. As a future physician, I am against this change. Adding the information would make the ads less helpful and more confusing for patients, who are powerless to change the complex system driving out-of-control prices. (Aaron Troy, 5/28)

The 30 million Americans with rare diseases should feel as encouraged as I am by the ongoing and enthusiastic commitment the Food and Drug Administration has made to advancing the approval of orphan drugs. But a loophole exploited by a pharmaceutical company threatens the integrity of this vital act. Over the past few years, fully one-third of new drugs approved by the FDA have been for rare diseases. The total number of orphan indications approved by the FDA jumped from 594 in 2016 to more than 770 in 2018. These approvals reflect not just a commitment by the FDA but also the success of the Orphan Drug Act, passed by Congress in 1983 to provide incentives for companies to develop orphan drugs. The act gives companies seven years of exclusivity for drugs given an orphan indication, a 25% tax credit for qualified clinical trials, and the waiver of application fees. (Diane Dorman, 5/28)

Geoffrey Chaiken, co-founder and CEO of Blink Health, says he and his brother, Matthew, have a simple mission: To make prescriptions affordable for everybody in the country. “The way we’re doing that is by moving the category online,” Chaiken said. “When a category moves online, it brings transparency. You see the price before you get to the counter.” (Daniel D'Ambrosio, 5/23)