The U.S. Senate versus the FDA.

By Clifton Leaf and Sy Mukherjee
July 13, 2017

Healthcare is an investment, a down payment on human infrastructure. The idea is straightforward enough: The medical R&D and other expenditures we make today toward the goals of improving human health and preventing early death will ultimately pay off in a happier, more productive society—just as investing in bridges, highways, and high-speed connectivity will pay off in the long run, too. Or so the theory goes.

And in the past two days, we have seen two strikingly different views on the importance of such investment—each, notably, from a different arm of the U.S. government.

Yesterday’s verdict was rendered by a drug advisory panel to the Food and Drug Administration, which unanimously recommended that the agency approve an important new therapy for certain very hard-to-treat blood cancers in children and young adults. The expert panel voted 10-to-nil to encourage the FDA to approve an experimental Novartis treatment called CTL019—a complicated approach that involves extracting specific immune cells from each individual patient, retrofitting those cells with genetic instructions that make them fiercer and more accurate cancer hunters, vastly increasing their numbers until they are a veritable army in themselves, and then injecting them back into the patient’s body.

CTL019—which is part of a class of engineered hybrids known as CAR-T cells, for “chimeric antigen receptor T-cells”—is no magic pill, I should point out. It’s risky: The cross-effect from revving up the immune system can sometimes cause a deadly internal storm of its own. This “living drug” is difficult to mass-produce: Each treatment is precisely tailored to an individual patient. It’s time-consuming to make and, heck, it’s expensive. But as with other new immune therapies, CTL019 has led to some miraculous results in patients—in some cases, curing otherwise surely lethal malignancies outright.

It’s also, importantly, a story of investment. No, I don’t mean Novartis’s here, though that should be applauded too. I mean the investment in medical science that got us to this point—a down payment in the seminal work of investigators like Steve Rosenberg at the NIH; Jim Allison at MD Anderson (and before that, at a slew of other institutions); Carl June at Penn; and countless others going back to William Coley—who, in 1891, reported on his efforts to elicit an immune response against cancer by way of an ingested stew of bacterial toxins.

Novartis is seeking FDA approval now to market CTL019 (Tisagenlecleucel) as a treatment for just a select group of patients in the U.S.—those (aged 3 to 25) who have B-cell acute lymphoblastic leukemia that has either relapsed or resisted standard treatments—a cohort that may number as few as 5,000 people. But there are about 100,000 cases of the broader class of B-cell [lymphoma] tumors that express the protein that CTL019 targets, Dr. June told me in an interview at Penn last year. “We haven’t found one yet that doesn’t respond to our CD-19 CAR-T,” he said.

So what of that other view of healthcare investment I noted up top? Well, that can be seen in the now-revised Obamacare-replacement bill the Senate unveiled today. In short, it looks much like the old bill, slashing spending for Medicaid by hundreds of billions of dollars, just as the earlier version would do. (The CBO, Congress’s in-house bill auditor, will estimate the full financial, economic, and insurance-coverage effects of the bill in the coming days.)

That’s not a down payment on the health of the American people, in my view. That’s a foreclosure.

The news below.

Clifton Leaf, Editor in Chief, FORTUNE
@CliftonLeaf
clifton.leaf@fortune.com

DIGITAL HEALTH

Turning cells into hard drives. File this one under, “Just Plain Cool.” Harvard Medical School researchers successfully placed one of the world’s most iconic, pioneering short films inside of bacterial DNA. This data can be extracted at will and multiplied into new cells ad infinitum—a truly remarkable milestone for using DNA as a vessel for data storage. One day (admittedly far off in the future), the foundations of this technology could be used to glean far more detailed insights into how cells behave once diseases strike. (New York Times)


INDICATIONS

The House unanimously passed a health care bill yesterday. It may seem strange given the ongoing Obamacare drama (more on that below). But, on an unanimous voice vote, the House of Representatives passed a critical piece of health care legislation on Wednesday. The Food and Drug Administration Reauthorization Act (FDARA) continues the Prescription Drug User Fee Act (PDUFA), a program which allows the FDA to collect fees from pharma companies in order to expedite the drug approval process for certain experimental treatments. The legislation must now be passed by the Senate.

Pharma warns of patient consequences if Brexit is disorderly. The fallout from Brexit continues to affect the life sciences industry, with pharma leaders warning that a haphazard exit from the European Union good wreak havoc on the drug supply chain (and, consequently, patients). “In the case of an unorderly withdrawal, there is a risk that all goods due to be moved between the UK and EU could be held either at border checks, in warehouses or manufacturing and/or subject to extensive retesting requirements,” wrote European biopharma and medical research executives in a letter to Brexit minister David Davis and chief EU Brexit negotiator Michel Barnier. “In fact this would lead to a severe disruption of most companies’ supply chains, which would lead to potential supply disruptions of life-saving medicines.” (Reuters)


THE BIG PICTURE

Did Philip Morris try to subvert a landmark anti-smoking treaty? An exclusive Reuters report alleges that internal documents from tobacco giant Philip Morris show that the company has been waging a behind-the-scenes campaign to undermine the World Health Organization’s anti-smoking treaty, which is widely considered one of the most critical avenues for curbing preventable deaths from tobacco use. (Reuters)

Senate releases latest draft health care legislation. The Senate has released yet another version of its health care legislation. And, so far, it’s unclear that the newest iteration will fare much better than previous Better Care Reconciliation Act (BCRA) drafts. For one, the bill still preserves massive cuts to Medicaid. It also adopts a controversial amendment from Sen. Ted Cruz of Texas which would allow insurance companies to sell plans that don’t comply to various Obamacare protections and benefits requirements, raising concerns about a “raise-to-the-bottom” in which deductibles soar while insurance coverage grows more paltry and prices sick people out of the system. At least two—and possibly three—Senators seem to still adamantly oppose the bill, which would be enough to sink it. More on this later. (Politico)

The Justice Department is charging hundreds in a new opioid crackdown. In a new effort to stymie the wave of U.S. opioid overdoses, the Department of Justice is embarking on a new quest to crack down on opioid fraud–and is charging hundreds of doctors, pharmacists, and nurses for their alleged part in “maliciously contribut[ing]” to the epidemic. (Washington Times)


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