A pricey new drug that may not work? Why drug pricing critics are staying quiet.

“Everyone is a bit terrified by Alzheimer’s, so the average person hears about the FDA approval of a treatment, and they don’t know about the controversy over whether it works or not,” said Craig Garthwaite, a health economist at Northwestern University’s Kellogg School of Management who lambasted FDA’s Aduhelm approval. “They hear there’s a new treatment and, that’s great, it’s a sign of hope. Do you want to be the politician who says, ‘I want to take that away from you?’”

The drug was the first cleared for Alzheimer’s in nearly 20 years, and it’s the first aimed at slowing cognitive decline rather than just addressing disease symptoms. But the agency’s fast-track approval has been criticized by many drug experts, prompted the resignation of three outside FDA advisers, and has hardened opposition to acting FDA commissioner Janet Woodcock, who is President Joe Biden’s apparent favorite to permanently lead the agency.

The severity and complexity of Alzheimer’s, an incurable neurodegenerative disease that afflicts roughly 6 million Americans and levies a harsh toll on their families, makes it difficult for even many of pharma’s Democratic critics on the Hill to cry foul. Patient advocacy groups for years lobbied hard for Aduhelm’s approval after seeing many other candidates fail over the years. And Biden, who is calling for billions of dollars to accelerate research into cures for Alzheimer’s and other diseases, was pleased with the drug’s approval, POLITICO reported Thursday.

For Republicans, the weak evidence for Aduhelm’s efficacy makes it harder to tout the treatment as an example of the breakthrough cures that they argue Democratic drug pricing proposals would stifle. Sen. Jerry Moran (R-Kan.), who was one of the few senators to mention the drug during a Thursday hearing with Woodcock, praised the approval and said he hoped it could set the stage for faster development of other treatments.

“I’m convinced that with more timely approvals that there will be more private investment in research to find these lifesaving and life-improving drugs,” Moran said.

The few Democratic lawmakers who have criticized the drug’s price are those who tend to be the most outspoken on the issue, including Senate Finance Committee Chair Ron Wyden (D-Ore.), who’s writing the party’s drug pricing overhaul in the upper chamber. Rep. Peter Welch (D-Vt.), a pharma hawk, acknowledged the drug’s tricky politics but said Democrats risked missing an opportunity to make their case for reining in drug prices.

“All of us are really supportive of a cure or a treatment for Alzheimer’s, so we want it,” Welch said. “But it’s misguided on our part to not address the here-we-go-again price gouging.”

Biogen declined to comment for this story, but CEO Michel Vounatsos in comments this week said the drug’s price was justified by the value it will bring to patients and a society less burdened by Alzheimer’s. That’s a message that was echoed by some Republicans, who have been aligned with the industry on opposing Democratic efforts to rein in prices.

“I think there’s a general hope for the new drug,” said Rep. Kevin Brady of Texas, the top Republican on the House Ways and Means Committee. “I believe we’re assessing the pricing of this, although Alzheimer’s is both devastating in both human cost and health care costs as well.”

Some progressive groups stressed that lawmakers are still reviewing the FDA’s decision, which caught many by surprise. They said backlash may build in the coming weeks as more experts raise concern over the drug’s approval and the financial stress it could put on the health care system and patients.

“I’ve not gotten a clear read as to why there’s been a relative silence around this so far on Capitol Hill,” said Frederick Isasi, the executive director of Families USA, a consumer advocacy group that supports the drug pricing bill House Speaker Nancy Pelosi is pushing to include in infrastructure legislation.

However, a senior Democratic aide expressed doubt that many party lawmakers will turn Aduhelm into a rallying cry for that bill, which would mandate government drug price negotiations. But the aide also said the muted response didn’t indicate anything about the party’s appetite for addressing drug prices.

“Hopefully there is a more clearly effective drug for Alzheimer’s on the horizon,” the aide said. “If this is the sort of cost that we’re talking about, there has to be price negotiations. Or else, what do you do? Or not everybody gets the drug.”

Much of the scrutiny around the drug in recent days has focused on the FDA’s decision-making. The agency provided broad approval for the drug, even though no one on its independent advisory panel voted in favor. The FDA isn’t required to follow the recommendations of its advisory panels, but it usually does.

Though the agency usually requires two clinical trials showing a drug’s effectiveness before issuing an approval, two Aduhelm trials produced mixed results. The FDA is requiring Biogen to conduct a follow-up study to prove its effectiveness. It can revoke approval if that fails, but it does not have to and such reversals rare.

FDA said it approved the drug because it reduced the presence of the amyloid protein in the brain, which many scientists think causes Alzheimer’s. But that link has never been proven — and the failure of Biogen’s second trial was interpreted by many researchers as a strong blow against the so-called “amyloid hypothesis” of the disease.

While Garthwaite, the health economist, has been critical of Democrats’ proposal for Medicare drug negotiations, he said the shaky basis for Aduhelm’s approval could build the case for it. He and other experts worry that the low bar set for this drug could spur other manufacturers to work on similar treatments rather than investing in potential major breakthroughs for the disease — in which case the FDA’s actions would diminish research.

The Alzheimer’s Foundation of America, an influential nonprofit that celebrated Aduhelm’s approval, didn’t respond to a request for comment on the price. AARP, which represents Americans 50 and older, declined to comment.

Robert Egge, chief public policy officer for the Alzheimer’s Association, said the group is “upset about the cost” and in a Capitol Hill briefing this week flagged it as an “insurmountable barrier.”

“The Alzheimer’s Association is focused, as we all should be, on the fact that this treatment may benefit people in the early stages of Alzheimer’s disease and these individuals must have access to this treatment,” he said. “We’re committed to doing everything in our power to ensure access to the drug, any diagnostic or other tests needed during the treatment process.”

Isasi of Families USA called the complicated policy questions invoked by the drug a “bomb on a slow fuse.” The federal Medicare agency still hasn’t said how it will cover the drug, which is delivered as monthly intravenous infusions. It could install clinical guidelines that effectively narrow who’s eligible for the drug — a cost-saving move that could draw resistance from patient advocates.

Health experts predict the costs will hit the Medicare program hard. The program could face $29 billion in additional costs annually if just one quarter of the 2 million beneficiaries now using already-available Alzheimer’s treatments are prescribed Aduhelm, according to a recent Kaiser Family Foundation study.

“It would be better for Congress to act now than to look back and realize that this was a watershed movement and they didn’t act,” said Mark Miller, executive vice president for health care at Arnold Ventures, which funds work on health care reform efforts.

Source:Politico