Americans Are Paying Billions To Take Drugs That Don’t Work

Companies are increasingly using fast-track approval processes to get unproven treatments to patients. Americans Are Paying Billions To Take Drugs That Don’t Work

One ALS drug made $400 million in sales for its maker. It doesn’t work. A cancer treatment brought in $500 million. That one turned out to have no effect on survival.

A blood cancer medication made nearly $850 million before being withdrawn for two of its uses. That drug had been linked to patient deaths years prior.

All of them were allowed to be sold to Americans because of the US Food and Drug Administration’s drive to get new drugs to patients quickly — sometimes even before they’re done testing.

The agency has been under pressure to move faster, a dynamic that has roots in the AIDS crisis when patients were dying while waiting for new medications.

But in the years since, it has evolved into an approval process that critics say is driving confusion and could be putting people at risk.

Drug companies are profiting, though. Since 2014, they’ve made at least $3.6 billion in global sales of medications that have either later been shown to be ineffective or had most or all of their uses withdrawn in the US, according to data compiled by Bloomberg.

“We get drugs faster and faster and know less and less about them and pay more and more,” says Yale University public health professor Gregg Gonsalves, a former AIDS activist who was part of a group that urged the FDA to get quicker — but also more rigorous — in the 1990s.

Patient groups that are now pushing for speedy approvals took the wrong lessons from the HIV crisis, he said. “The idea is not to have more drugs. It is to have drugs that work.”

There are a number of ways a drug company can get its treatment to patients faster: There’s the “priority review” pathway, then “fast track,” “accelerated approval” and “breakthrough therapy.” Companies can also use vouchers to ensure speedier reviews for drugs that wouldn’t normally qualify.

Bloomberg’s data analysis showed that the majority of new drugs in the US are approved through one or more of these sped-up pathways. Last year two thirds of all new drugs reached the market this way.

These avenues can help get drugs to patients in some cases years quicker than a traditional approval. The pathways allow less research into the drugs prior to approval, speedier reviews of the research that has been done or in some cases more access to top FDA officials.

The agency argues these efforts improve medical care and that some uncertainty comes with the territory. Many drugs brought to market through expedited approvals have made “a significant impact on disease,” FDA spokesperson Cherie Duvall-Jones said in an email.

In many cases, the expedited process works well and gets important drugs to people that much faster.

“These programs are intended to speed the availability of new therapies to patients with serious conditions, especially when there are no satisfactory alternative therapies, while preserving appropriate standards for safety and effectiveness,” she said.

Because there is some inherent uncertainty in the accelerated approval process, “it is expected that certain products may fail to verify clinical benefit.”

Stocks Swing

Investors are also impacted by rushed drug approvals. Shares often shoot up when a company announces one of its drugs has qualified for an expedited approval pathway. Later, when a drug shows signs of not working, the stocks normally tank.

Amylyx Pharmaceuticals Inc.’s ALS drug is one such example. It had only completed one small trial that critics said hadn’t shown a clear-cut benefit when the company sought approval in 2022.

One of the founders promised to yank it if studies eventually confirmed the treatment was, in fact, ineffective.

The company’s stock surged more than 50% after FDA advisers voted in favor of approval. Amylyx priced the treatment at $158,000 a year after getting to the market through the “priority” pathway.

Nearly a year and a half after the drug hit the market, a large study confirmed what skeptics had already suspected: Amylyx’s drug was no better than a placebo. The company’s stock plummeted over 80%.

Amylyx is now taking its drug off the market — after reaping $400 million in sales from it.

Decades ago, the FDA reviewers weren’t very flexible and drugs sometimes languished for years as the agency demanded more data.

“Now they’ve gone so far in the opposite extreme direction, sometimes approving things where there’s literally no evidence except wishful thinking,” says Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank.

The FDA’s Duvall-Jones said the agency only approves drugs if there is “substantial evidence” they work and the benefits outweigh the risks.

In theory, sped-up approval programs are designed for the most promising drugs that address some of the most crucial medical needs.

In practice, the drug industry has found ways to expand use of these pathways beyond the relatively narrow uses for which they were originally envisioned.

That may be because of pressure on the FDA from patients and their loved ones, who often join forces through nonprofits that make getting drugs approved quickly part of their mission.

The ALS Association, for example, said in its annual report that it “led a campaign to encourage the FDA to act with urgency and flexibility” to get Amylyx’s drug to patients.

After Amylyx’s drug failed in the larger study, the ALS Association put out a statement expressing disappointment and noting that they had supported the early approval because the drug appeared safe and patients were “willing to take the risk if it turned out to be ineffective.”

Additional Approvals

One of the issues with the FDA letting a drug onto the market quickly is that it can open the door for similar drugs to be approved, too.

That’s what happened with a group of blood cancer drugs. Gilead Sciences Inc.’s Zydelig was approved in 2014 through sped-up pathways for two types of lymphoma.

But within two years, the company halted multiple studies of the drug due to serious side effects and deaths of patients in trials.

The FDA put out safety warnings, yet in the years after, the agency continued to grant sped-up approvals for a number of similar drugs.

One of those was TG Therapeutics Inc.’s blood cancer pill Ukoniq, which snagged accelerated approval in February 2021 for two types of lymphoma based on small trials that didn’t assess whether patients lived longer.

The company’s shares shot up 12% on the speedy approval.

But less than a year later, a larger study of TG Therapeutics’s drug in a third blood cancer found a potential increased risk of death when compared to patients on standard treatment.

The company, at the urging of the FDA, took the drug off the market in April 2022 and stopped pursuing approval in the additional cancer.

The FDA, in response to questions about Ukoniq, said the agency had required further study of the drug to make sure it worked.

“These indications were for patients with limited available effective therapies,” the FDA’s Duvall-Jones said, adding it was only on the market for a limited time.

The company didn’t respond to requests for comment for this story but at the time of withdrawal, TG’s chief executive officer Michael Weiss said he was “very disappointed” by the survival results.

That same year, Gilead announced it would voluntarily withdraw Zydelig’s two lymphoma uses in the US, some six years after it had halted trials because of patient deaths.

A Gilead spokesperson said the withdrawal was not due to safety concerns but rather because of challenges enrolling people in additional research trials required by the FDA.

Gilead’s drug is still on the market for the use that it had been originally approved for, chronic lymphocytic leukemia.

Gilead reaped $842 million in global sales of the drug before withdrawing it for some uses in the US, where it made nearly half of those revenues, a JAMA Internal Medicine study found.

“Six years prior to withdrawal there were already serious toxic effects concerns, and minimal evidence” from new studies showing the drug helped patients, Titas Banerjee, an Oregon Health and Science University clinical assistant professor, said in the research paper she co-wrote last year.

“The findings of our study suggest that patients would benefit from regulatory reform and increased oversight of the accelerated approval pathway.”

One of the problems is that sometimes drugmakers resist pulling a drug off the market, even after it’s obvious it doesn’t work. Makena, a drug to reduce the risk of premature birth, received a sped-up approval in 2011.

Eight years later, a large trial found it didn’t work.

Yet it took another four years for the FDA to force it off the market. Makena, which was owned by a number of companies in its 12 years on the market, generated over $1.6 billion in sales.

The FDA is trying to take a stronger hand to get problematic drugs off the market. In February, it used a new “expedited withdrawal” procedure for the first time to rescind the approval for Pepaxto, a bone marrow cancer drug from Oncopeptides AB after the agency concluded that a new trial had failed to confirm the drug worked and actually suggested patients taking the drug died sooner.

Oncopeptides’ shares, which hit a two-year high in Swedish trading shortly after the accelerated approval in February 2021, have since lost more than 95% of their value.

The company disputed the FDA’s conclusion that the trial failed, a company spokesperson said in an email. Based on the same data, the drug is still on the market in Europe.

“The short answer is that it does work,” Oncopeptides spokesperson David Augustsson said.

The FDA’s budget is increasingly coming from the drug industry for fees associated with drug reviews. Last year, such payments accounted for close to half of the agency’s nearly $7 billion budget, up from about a quarter in 2010.

The agency has been approving an increasing number of drugs in recent years, with approvals of new therapeutic drugs reaching unprecedented levels last year, according to an analysis of data compiled by Boston Consulting Group.

A company can also use more than one sped-up pathway on the same drug in the hopes of getting to market that much faster.

In 2016, Eli Lilly & Co.’s cancer drug Lartruvo was approved after having snagged all four expedited pathways.

The accelerated approval was based on one trial of 133 patients that showed promising survival results in sarcoma, a cancer that affects muscles and other soft tissues, even though the drug’s effect on slowing cancer growth weren’t clear cut.

Just over two years later, a larger study showed the drug had no effect on patient survival and Lilly pulled it from the market.

A Lilly spokesperson said the company works to quickly conduct studies needed to confirm its drug works and that the accelerated approval program is an “impactful mechanism” for accessing promising treatments quickly.

Americans Are Paying Billions To Take Drugs That Don’t Work

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