Tuesday, September 23, 2008

Patient power

I've been thinking a lot about clinical trials lately, as you'd imagine, and one of the things I've been wondering what percentage of drugs at a given stage of trial eventually make it to market. 

I tend to think about it in sports terms -- that for every 20,000 high school basketball players, maybe 10 will play in division 1, and for every 100 D1 players, maybe five or ten make the league. But this is obviously vague, plus it leads to inappropriate analogies: Is this upcoming cancer drug a seven-foot-gazelle, or a 5' 11" point guard who shoots too much? So I was interested to see this passage in a Forbes article about the perils and possibilities of patient-directed medical research:
The patient groups are filling a void in drug research created by the industry's legitimate fear of failure. Biotech executives dread what they call "the valley of death," the period of time between a drug's conception in a lab and its first clinical trial. For every 10,000 would-be medicines chemists create, only one makes it to market. But if a drug has already been through enough tests in cell cultures and lab animals to justify starting clinical trials, the odds of success have risen to one in nine. Put the substance through early clinical trials with a few dozen patients, and the odds jump to one in six. At some point a drug for even the most uncommon disease becomes every bit as appealing to drug companies as an untested potential heart treatment or impotence pill.
I'm not sure I completely believe this -- one in nine drugs in phase-1 trials eventually make it to market? -- but it's encouraging. The rest of the article is interesting as well, and even features a quote from sarcoma celebrity and all-around oncological badass Dr. George Demetri. It's clear that there are highly-motivated and talented individuals (many with business expertise) who can make good things happen faster than they would otherwise, especially for rare conditions that may not have the "market share" to immediately attract pharma dollars. But the emotions involved are intense. Forbes describes prostate-cancer advocates running amok against doctors skeptical about the evidence backing an investigational drug. I get both the advocacy side -- just try to keep me away from something that may work -- and also why some kinds of advocacy absolutely are not helpful to researchers trying to practice good science and good medicine and minimize unintended consequences.

Sarcoma, incidentally, has at least one collaborative group pushing to link researchers at different institutions and secure funding for trials. Though the Sarcoma Alliance for Research through Collaboration was founded by academic researchers, there's at least one seat for a patient-advocacy organization on its board, and they sponsor an array of collaborations, studies and trials.

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