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Smaller Smarter Clinical Trials

Posted 3/19/2014

Posted in

  Repeat after me: everything that we know about cancer treatment is based on the results of clinical trials. We are all forever endebted to people, mostly women in our case, who agreed to participate. Otherwise, we would all be treated based on individual doctor's instincts and experiences and best guesses. Our way is definitely better!

  Having said that, most of us are aware that are system of clinical trials is imperfect, and we are always looking for ways to improve them: to get useful and correct information more quickly and more reliably. ASCO has been looking at this topic with particular interest of late and has come out with some recommendations as to the structure of cancer clinical trials. Their summary intent is to make them "smaller and smarter."  The core belief is that trials should be designed so that the measured endpoint of increased survival is meaningful.

  Sometimes now a trial indicates that a particular (usually very expensive) drug extends life for some patients for a few weeks or even a few days. This may, under the specific design, be statistically significant, but it is not so significant for patients who are hoping to live for at least months longer when signing on for a potentially difficult treatment. I also cynically suspect that not all patients understand (or maybe are even told) that drug X extended life for an average of nine days. They may instead have just been told, or just "heard", the extending life part. Now, I am very aware that none of us should dare to speak for others and each of us must be supported in her own decisions. But, I can't help wondering if some people would make a different decision if they were really fully informed about benefit and risks and costs.

  To some extent, this is irrelevant to the particular study and article that I am writing about today, but it is context for better understanding. This is a report from MedPage that likely will be of interest. Per usual, I give you the beginning and then a link to read more.

Panel Urges Higher Bar for Cancer Trials

Oncology clinical trials should be designed
so that the primary endpoint of overall
survival benefit is a more meaningful one,
and patients should expect no less,
according to an oncology research panel.
Using current overall survival
expectations, members of the panel
recommended a minimal improvement of
20% in the hazard ratio and absolute
improvement in the range of 2.5 to 6
months as goals for clinically meaningful
benefits, Lee M. Ellis, MD, of the University
of Texas MD Anderson Cancer Center in
Houston, and co-authors concluded.
For secondary endpoints of 1-year survival
and progression-free survival, the
committee recommended absolute
improvement of 8% to 15% and 3 to 5
months, respectively, depending on the
primary site of the cancer, they reported
online in the Journal of Clinical Oncology.


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