The FDA's decision not to rush approval for Eli Lilly's experimental Alzheimer's treatment solanezumab—a drug that turned out to be ineffective—may have saved American taxpayers as much as $100 billion over the past 4 years, an analysis concludes.

The analysis comes amid pressure on FDA to use less–strict standards in deciding whether a drug should be approved. Some agency critics have called on the government to approve all drugs that are not toxic and let market forces determine which are best.

"The issue right now in the national conversation is this push to approve drugs faster and faster at all costs," researcher Dr. Chana Sacks, Harvard Medical School and Brigham and Womens Hospital, Boston, told Reuters Health.

"The conversation usually focuses on the toxicity—the risks and benefits—of the drugs themselves. We wanted to think about the financial toxicity, which is very real, and what the financial implications might be of lowering standards," she said.

Final–phase testing of the anti–amyloid drug showed it was no better than placebo, Sacks and colleagues reported in the New England Journal of Medicine, May 4th.

Two tests in 2012 found that it didn't improve cognitive or functional abilities in people with mild or moderate Alzheimer's, but hope for the drug remained alive after Lilly said it reduced cognitive deterioration among people with mild dementia by 34%, an accurate but misleading figure.

On a 90–point scale, solanezumab recipients showed an improvement of a mere 1.7 points. Reporting results as percentages can often make small improvements appear much more dramatic.

"Lilly’s 2012 announcement led to hope that solanezumab could alter the disease’s course, although perhaps only for patients at an earlier stage of disease," the researchers write in a commentary.

After the results of the final test, known as Expedition 3, were announced in 2016, Lilly abandoned the drug as an Alzheimer's treatment.

If solanezumab had been approved in 2012 based on a looser standard that only required a hint of effectiveness, billions would have been spent before it was discovered that it didn't work, and discovery of its ineffectiveness would have taken much longer because it would have been harder to get volunteers to sign up for a study where they might get a placebo instead of the drug. Meanwhile, Medicare would have had to pay for an ineffective drug.

Although Lilly never announced a price, it's not unusual for such drugs to cost $14,000 to nearly $30,000 a year.

"Even conservative estimates suggest that the total costs of solanezumab would have been staggering," the research team writes. "Of the more than 5 million people in the US with Alzheimer’s disease, about half can be categorized as having mild disease, the subgroup initially thought to benefit from solanezumab. If the price had been set at $10,000 per patient per year and just one tenth of those patients had been treated, the cost would have been almost $10 billion over the past 4 years."

If half the eligible population had used it and the cost had been $20,000 a year, total spending would have hit $100 billion over the first 4 years of sales and marketing.

Even that estimate may have been low, said Sacks.

Lilly, asked for reaction to the commentary, released a statement: "The emotional and economic toll this disease, if left untreated, takes on society and families is astronomical. While the results of Expedition 3 were not what we had hoped for, Lilly will continue to focus on finding disease–modifying therapies, diagnostics and solutions to ultimately help the 47 million people worldwide who are waiting for a cure of this horrific disease. We remain committed to Alzheimer’s disease research, as we have been for nearly 30 years."

—Gene Emery