The research loophole
We keep going on here about the importance of publishing clinical trials. Today (in Britain), the BBC program Panorama is showing a documentary about a doctor who has been running clinical trials of the same basic treatment regimen for twenty years, without publishing any results. And it’s not that these are trials that take a long time to run — the participants have advanced cancer. If the treatment was effective, it would have been easy to gather and publish convincing evidence by now, many times over.
These haven’t been especially good clinical trials by usual standards — not randomized, not controlled — and they have been anomalous in other ways as well. For example, patients participating in the trial are charged large sums of money for the treatment being tested (not just for other care), which is very unusual. Unusual, but not illegal. Without published evidence that the treatment works, it couldn’t be sold outside trials, but it’s still entirely legal to charge money for the treatment in research. It’s a bit like whaling.
According to the BBC, Dr Burzynski says it’s not his decision to keep the results secret
He said the medical authorities in the US would not let him release this information: “Clinical trials, phase two clinical trials, were completed just a few months ago. I cannot release this information to you at this moment.”
If true, that would be very unusual. I don’t know of any occasion when the FDA has restricted scientific publication of trial results, and it’s entirely routine to publish results for treatments that have not been approved or even where other research is still ongoing. The BBC also checked with the FDA:
But the FDA told us this was not true and he was allowed to share the results of his trials.
This is all a long way away from New Zealand, and we can’t even watch the documentary, so why am I mentioning it? Last year, the parents of an NZ kid were trying to raise money to send him to the Burzynski clinic, with the help of the Herald. You can’t fault the parents for trying to buy hope at any cost, but you sure can fault the people selling it.
Wikipedia has pretty good coverage if you want more detail.
Thomas Lumley (@tslumley) is Professor of Biostatistics at the University of Auckland. His research interests include semiparametric models, survey sampling, statistical computing, foundations of statistics, and whatever methodological problems his medical collaborators come up with. He also blogs at Biased and Inefficient See all posts by Thomas Lumley »