Consider this scenario: An experimental Alzheimer’s drug fails two studies, but parsing the data shows the medicine may benefit a subgroup of patients with mild dementia. On that basis, the drug is approved and about 260,000 eligible patients are subsequently treated over the next four years.
Priced at $10,000 per person, the cost totaled roughly $10 billion during that time. To some, this would appear to be a bargain for a drug that combats a pernicious disease, yes? But what if it turns out the drug later failed yet another trial and patients with a mild form of Alzheimer’s weren’t helped, after all? The money —much of it spent by Medicare — would have been wasted and patients’ hopes dashed.
This outcome, happily, never became reality because instead, the drug maker, Eli Lilly, was required to run that third, unsuccessful trial before regulators would consider approval. But this hypothetical tale is a cautionary one, given the ongoing effort to alter FDA approval standards, according to Brigham and Women’s Hospital researchers, who published this scenario in The New England Journal of Medicine.
More analyses like the Harvard group need to be done; FDA collected some great info together on phase 3 failures on drugs in many different fields after earlier success:https://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM535780.pdf
So many small trials and sub-group analyses have been “positive” (“Breakthroughs!”) in what is marketed as ALzheimer’s disease. Most dementia is in people in their 80s and a mix of AD and other neurodegenerative changes as well as vascular disease. WHy are people surprised when these things don’t “work”? Those developing them are trying to get an approval based on some surrogate, misleading people into thinking it will really help
Great piece. Can you please send it to HHS Secretary Price and FDA Commissioner Gottlieb?
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