How Much Evidence Do We Really Need to Change Treatment Recommendations? – October 10

By Dr. H. Jack West

One ongoing controversy in the world of managing cancer, especially one in which cures are elusive, is how high the bar should be to guide treatment recommendations. The “gold standard” to change treatment is a significant improvement in the primary endpoint, ideally overall survival, in a prospective, randomized phase III trial with several hundred or even several thousand people. In some settings, treatment recommendations may not even change until a few randomized phase III trials show the same significant improvement with a new treatment approach.

But the reality is that sometimes we want or need to apply promising ideas that don’t have an overwhelming amount of evidence yet. That may be because a very promising result in phase II will typically take 3-5 years before the results of a subsequent larger phase III trial is completed and reported, with another 6-12 months before that new agent or combination is FDA approved and commercially available. That may also be because some of these clinical questions are too rare to test in a large phase III trial setting. For people with a ROS-1 rearrangement, who represent 1% of NSCLC, or those with leptomeningeal carcinomatosis, we can’t realistically expect to complete a randomized phase III trial with hundreds of patients assigned to one treatment or another.

  • So the first question is whether the medical community should be guided by a specific level of evidence to shape cancer treatment, and can the standard be lowered for clinical subgroups or settings in which the numbers are too small to ever run a large trial? And should the standard be higher in a curable setting like early lung cancer (is stereotactic body radiation therapy an appropriate alternative to surgery?) vs. metastatic disease because there is “more to lose”?
  • Second, should patients be able to dictate a lower level of evidence being required if they provide “informed consent” that one approach is conceptually attractive but not well tested?

And related to both of these points, is there a lower standard required when the cost of the treatment is low and/or the patient is paying and not insurance/broader society? If proton beam radiation therapy is three times as expensive as conventional radiation and has no actual evidence it’s superior, is it appropriate for institutions to market it (at a handsome profit) and/or
patients to expect it? Is it more appropriate if patients pay for some or all of the cost difference vs. passing on the costs to the others?

These are timely, important questions, so please join us on Thursday, October 10th, at 8 PM Eastern, 5 PM Pacific. Just get on twitter, filter your messages and add the hashtag #lcsm to your tweets, and join the conversation!

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