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.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!


What is it?

STAT Plus is a premium subscription that delivers daily market-moving biopharma coverage and in-depth science reporting from a team with decades of industry experience.

What's included?

  • Authoritative biopharma coverage and analysis, interviews with industry pioneers, policy analysis, and first looks at cutting edge laboratories and early stage research
  • Subscriber-only networking events and panel discussions across the country
  • Monthly subscriber-only live chats with our reporters and experts in the field
  • Discounted tickets to industry events and early-bird access to industry reports

Leave a Comment

Please enter your name.
Please enter a comment.

  • 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:
    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

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy