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Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: Twitter: Dereklowe

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« A Drug's Target, Finally | Main | Experimental Update »

June 7, 2005

When the Alternative is Nothing

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Posted by Derek

Placebo-controlled trials are usually considered the standard (and most stringent) measure of a drug's efficacy. It's a surprisingly high hurdle to clear. All sorts of things that people swear by, and all sorts of new things that you'd be sure would work fail when they're up against a similarly sized and colored dose of sugar.

But you can't always run a placebo group, because it isn't always ethical to do so. For a life-threatening condition, the comparison group has to be the current best standard of care (which, after all, is what you're trying to beat.) For lesser diseases, a trial against a known therapy can also be appropriate, although it's usually done after a placebo-controlled one has already been run.

But there's one situation where you can run a placebo control for a deadly condition: when the best standard of care is nothing at all.

Several forms of cancer fall into that category. Pancreatic, renal, and hepatic cancers, for example, exhaust their best available treatments very quickly. Some of the patients in that situation then offer themselves as subjects for clinical research, for which we in the drug industry are extremely grateful. With any luck, we'll be able to find something that works well enough to unblind as quickly as possible.

And when that happens, the disease is no longer in the "placebable" category. There's now an active agent, a possible treatment, and thus a new standard of care. Several cancers have moved off the list in recent years, and here's hoping that the process continues.

Comments (2) + TrackBacks (0) | Category: Cancer


1. Charlie Hendrix on June 10, 2005 12:05 PM writes...

It's a heck of a lot easier to establish that a hopeful new drug is more effective than a placebo than it is to prove it is better than the current "best practice". This is a statistical fact of life. Detecting and "proving" a large difference requires a lot less data (subjects) than detecting and "proving" a small difference.
It's fine to begin with a placebo-controlled trial to establish an "ah ha, this thing is working". But proclaiming "this baby is better than the current best" that can require very large and expensive clinical trials. The more subject you have in those trials the more pesky side effects will appear.

Your friendly neighborhood statistician...

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2. Ian Ameline on June 16, 2005 7:14 PM writes...

Might be off topic, but what do you think of 17AAG and other Geldanamycin derivitaves blocking HSP90 as a workable treatment for cancers?

While 17AAG is only soluble in DMSO, there are other derivatives that are water soluble that are in the works...

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