The U.S. Food and Drug Administration'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 four 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 of Harvard Medical School and Brigham and Womens Hospital in 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.
In the case of solanezumab, final-phase testing of the drug showed it was no better than placebo, Sacks and colleagues reported in the New England Journal of Medicine.
The drug was designed to clear the amyloid plaques long believed to play a role in the incurable disease, which slowly robs people of their mental abilities.
Two tests announced 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 percent, an accurate but misleading figure.
In fact, 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, Sacks and her colleagues write.
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.
"These studies, if they are performed, are often not completed until many years after marketing begins," the Sacks team notes. 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 United States 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 four 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 four years of sales and marketing.
Even that estimate may have been low, said Sacks. "Current regulatory standards prevented ineffective medications from reaching patients and averted unnecessary spending (as well as unanticipated side effects)," the research team writes. "The public and private funds not spent on a useless drug remained available for other interventions that have been proven to work."
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."
SOURCE: bit.ly/2qLgYHO New England Journal of Medicine, online May 4, 2017.