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 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.
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"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: http://bit.ly/2qLgYHO New England Journal of Medicine,
online May 4, 2017.
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