FDA let hope get in way of sound science with Alzheimer’s drug

By David Farmer

Hope is not a plan. Or a medical treatment.

But that seems to be the path taken by the U.S. Food and Drug Administration, which last week approved a controversial drug to treat Alzheimer’s disease.

After more than a year of many of us repeating the mantra to trust science, to follow the research, to mask up and to get vaccinated, the FDA — the federal agency charged with approving new drugs — appears to be going in the other direction and approving a bank-busting treatment for Alzheimer’s that hasn’t been shown to be effective.

My mom died of Alzheimer’s disease in October 2015, but we actually lost her years before. The disease had taken away her memories and left her dependent on others. Her personality — outgoing, radiant — was dulled, though her charm persisted until nearly the end.

When we realized she was sick, my family would have done just about anything to slow the inevitability of the disease, to buy a little more time, to cheat fate.

That’s why health care is not a rational marketplace, and why none of us are really consumers when an emergency strikes. Love and fear blind us to rationality, to cost and to consequence. Nobody shops for the cheapest emergency room, and few people would say no to a drug treatment — no matter how expensive — if there was hope to avert a horrible outcome.

That’s why we depend on doctors and nurses and experts to keep us grounded and force us to think with our heads and not our hearts.

With the approval of aducanumab, I fear that the FDA is letting hope — and pressure from advocates desperate for progress against a terrible, terrible disease — get in the way of sound science.

Last year, an FDA advisory committee voted that there was not enough evidence to show that the drug was effective. Of two major studies, the conclusions were split and a third-party evaluation found that “insufficient to conclude that the clinical benefits of aducanumab outweigh its harms.”

After the drug’s approval, three experts on the FDA advisory committee resigned in protest.

One of those experts, Dr. Aaron Kesselheim, and a colleague, Dr. Jerry Avorn, both professors of medicine at Harvard Medical School, accused the FDA of lowering the bar for drug approval in a column for the New York Times.

“The aducanumab decision is the worst example yet of the FDA’s movement away from its high standards. As physicians, we know well that Alzheimer’s disease is a terrible condition. But approving a drug that hardly works and causes such worrisome side effects is not the solution,” they wrote

Despite questions about its effectiveness and potential side effects, the drug will come with a staggering price tag, estimated today at about $56,000 per year per patient. For the company making the drug, it could generate billions of dollars in revenue — mostly from Medicare.

But the costs will impact everyone, as Dr. Joseph Ross, a policy expert at Yale, told the New York Times: “This is really what keeps me up at night: A therapy of this cost is going to have enormous implications for everyone. And by everyone, I literally mean you, too. There’s going to be some 60- and 70-year-olds on your plan. If they start getting this treatment, you will see your premiums will go up.”

The U.S. health care system is broken, and it needs major reform to improve quality and access and to control costs.

And it also requires regulators to hold the highest standards before approving new treatments that might not work.

Hope sells. I would have bought it. But the risk of damage to our system — and to families — is too great if regulators take shortcuts for hope’s sake.

Farmer is a public affairs, political and media consultant in Portland, where he lives with his wife and two children.