E-cig epidemic enforcement incoming

The FDA is getting serious about e-cigarette enforcement. In a statement released last week, the agency announced a crackdown on 1,300+ retailers and five manufacturers who make up 97% of the US market. That includes JUUL, which has been widely criticized for making vaping cool (Editor note: LOL is this really that cool?) and itself accounts for over half the US market. The FDA says vape use has increased to epidemic levels in teens, and it is intent to not “allow a whole new generation to become addicted to nicotine.” The agency expects the manufacturers to submit plans within 60 days to explain how they’ll stop teens from getting addicted to their products. If not, the agency could pull e-cigs from the market, a move which Big Tobacco is a fan of.

These dogs dig their dystrophin

Duchenne’s Muscular Dystrophy, or DMD, has been in the news a lot recently. It’s the most common form of muscular dystrophy and was most recently featured in the Right to Try fight. The FDA also approved the first treatment of DMD’s symptoms in 2017, but a new paper published in Science points to a possible treatment of the root cause of the disorder. Scientists used a system-wide application of CRISPR to efficiently restore dystrophin expression in four dogs, a result that “exceeded [the lead author’s] most optimistic expectations.” There’s a ton of hurdles this treatment would have to pass to be a real therapy in humans, but this could be huge for DMD patients. Also, hey, it’s Labor Day weekend, maybe consider donating to the Muscular Dystrophy Association for Jerry’s Kids.

Pfizer calls out peers

Pfizer filed a Citizen Petition with the FDA requesting regulators issue guidance to “…ensure truthful and non-misleading communications by sponsors concerning the safety and effectiveness of biosimilars….” With lagging performance from its Remicade copy, Inflectra, as the backdrop, Pfizer is calling BS on innovators’ communications regarding biosimilars, which Pfizer believes intentionally confuse consumers and prescribers and erode confidence biosimilars. Specifically, Pfizer notes how J&J describes that biosimilars act in a “similar” way to Remicade when, in fact, they employ the same mechanism of action. Amgen claims that switching to a biosimilar “carries risk” if your current product is working. And Genentech states that biosimilars are “highly similar but not identical” to branded products. Pfizer claims that’s only half the story. While Pfizer’s motives are almost certainly not altruistic in nature, they probably have a point about intentional marketing half-truths.

Gottlieb: “we’re not going to play regulatory whack-a-mole”

In last week’s He said, she said InsightCity article we talked about how regulators in Britain don’t think the price of biosimilar products is low enough. FDA Commissioner Scott Gottlieb has kicked it up a bit. In prepared remarks, Gottlieb blasts the pharma industry. Some quotes: “the FDA approved 11 biosimilars, only 3 are marketed in the U.S.” – “competition is, for the most part, anemic.” – “if Americans had the opportunity to purchase successfully marketed, FDA-approved biosimilar prescription drugs, they could have saved more than $4.5 billion in 2017.” – “branded drug makers thwart competition by dangling big rebates to lock up payors in multi-year contracts right on the eve of biosimilar entry.” All this was part of FDA releasing their Biosimilars Action Plan and the final guidance on biosimilar labeling. Might be time to keep your head down.

Real-Time Oncology Review

Earlier this week, FDA granted its first approval as a part of two new pilot programs that aim to make the development and review of cancer drugs more efficient. The drug is Novartis’ Kisqali and the two programs are the Real-Time Oncology Review (allows for the FDA to review much of the data earlier, after the clinical trial results become available and the database is locked) and the Assessment Aid Pilot Project (used by sponsors to organize their submission into a structured format to facilitate FDA’s review of the application). FDA Commissioner Gottlieb says, “With today’s approval, FDA used these approaches to allow the review team to start analyzing data before the submission of the application and help guide sponsor’s analysis of the top-line data.” Looking for a heavyweight fight? Watch sales of Pfizer Ibrance vs. Novartis Kisqali.

Important imports?

The Department of Health and Human Services has directed the FDA to consider the importation of foreign drugs to address price spikes. The proposed policy would only apply to drugs unaffected by patent or exclusivity, in an effort to avoid intellectual property issues. However, pharma companies may still have something to say through their lawyers if such a policy were to be implemented, especially as an act of a federal agency instead of legislation. If it were implemented though, it would avoid patient access issues in situations like when an HIV medication jumped in price from $13.50 to $750, which had the incredibly unfortunate side effect of catapulting Martin Shkreli’s infinitely punchable face into the limelight. That drug still costs $750 by the way. Or just 5-10 cents in India.

FDA and digital health, a marriage…

… “in the making” might be the best way to describe the relationship. On one side, the FDA has recently approved 14 new digital health products. On the other side, recent FDA guidance leads Bradley Merrill Thompson, a partner at Epstein Becker Green who specializes in FDA law to say, “Industry wants faster approvals. FDA wants more control over industry. So, FDA’s proposing an exchange: faster approvals for more FDA authority.” All this hullabaloo centers on whether FDA should certify every digital health product or can they certify digital health at the company level. We don’t know. What we do know is that digital health is hot, really hot, like there’s been $1.62B invested in digital health companies in Q1 2018. That’s $18M per day. Per day! Prefer smaller numbers? It’s $12,500 per minute. Muy caliente.

Everyone’s getting the short stick

Drug shortages are nothing new, that’s why the FDA updates their list of shortages daily. But things seem a little worse than normal—9 in 10 physicians say their emergency departments lack critical medicines. That includes mainstays like diltiazem (a go-to treatment for hypertension) and morphine. You can blame market forces and manufacturing issues. Most of the drugs in shortage are sterile injectables, which can be difficult to both make and make a profit on. Those low margins can lead to less incentive to maintain the quality of sterile injectable manufacturing facilities as they age, which in turn leads to issues with the quality of the drug products. It’s things like that which can cause cardboard to contaminate batches of “sterile” injectables. If only they had contaminated them with cash instead…