Throughout this site, we try to rely on the strength of evidence to support the value of one treatment over another.  This is based on a growing trend of evidence-based medicine, by which we shape our management decisions following the lines of clinical trials.  But this approach of “useless until proven valuable” is really the opposite of where many cancer patients feel that the onus needs to be.  When your options are not attractive enough, when oncologists agree that a cancer is incurable and may not even have any treatments of value, patients don’t usually demand that a new option have proven benefit.  Instead, many patients are interested in any treatment that has promise and provides some hope of clinical benefit.  And this is at the center of the tension that patient advocacy groups are experiencing with the FDA, because the FDA feels a need to protect patients against unproven cancer therapies, while patients often feel that they have nothing to lose and and desperately seek more options, even those in early development and with unknown value.

Have we proven the value of parachutes? 

   I just learned of a publication from a few years ago in the British Medical Journal that highlights a shortcoming of the evidence-based medicine approach by illustrating that we don’t actually have any good evidence from randomized, controlled prospective trials that proves the value of parachutes for “gravitationally challenged” people, namely those falling out of airplanes (paper here).

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Last year, one of the issues I was struck by in reading the comments on a few patient-oriented websites when I really began in earnest to participate in the online sites was the tension that can occur all too often between patients and oncologists. There are certainly many potential pitfalls, but one of the common themes is that many patients need a breakthrough fast and don’t need to see the results published in the New England Journal of Medicine from multiple large trials to be convinced that a promising approach is worth trying, while oncologists are often more conservative about providing access to commercially available drugs that aren’t indicated for a certain patient’s cancer. Whether it’s celebrex or sutent or DCA, these approaches represent legitimate hope when clinical and lab investigators describe the potential value of these treatments for improving survival. Often these conclusions are based on early data, from the lab or in some small clinical studies, and the media may provide a sound bite of brief analysis in which a brief story alludes to the next great breakthrough.

In fact, some of these treatments will, in fact, become valuable treatments, and a few will become miracles, or close to that. People with an unusual called a gastrointestinal stromal tumor (GIST) used to have very minimal therapy options, but with the new targeted therapy of Gleevec, they can have remarkably dramatic and long-lasting results. Tarceva, Avastin, and some newer chemo agents and targeted therapies may provide very gratifying benefits for many lung cancer patients. But there is often a cultural difference in the willingness of patients and oncologists to pursue a non-standard treatment. Read the rest of this entry »

Yesterday, a member here made a very fair comment (here) after one of my recent posts in which I raised my concern about giving treatments without evidence to support it. The issue was whether it is really appropriate to be so protective and insular in my thinking in a setting in which patients with lung cancer may have exhausted all of the proven treatment options. Would I really defend offering no further treatment to someone in whom the only proven path is to a very disappointing and to some people an unaccaptable place? Read the rest of this entry »