Comparative effectiveness could impede cancer therapy progress

In an editorial published the New York Times, a strong argument was made for studying the relative effectiveness of screening colonoscopy and sigmoidoscopy.  Based on a review in JAMA of three studies conducted outside the US that showed no difference in colon cancer mortality or incidence when the two procedures were compared, the Times proposed a US study to answer this question.

Comparative effectiveness (CE) research, a relatively new concept in the lexicon of US medicine, pushed to the forefront by health care reform, appeals to the data-driven orientation of oncologists but could put the rapid pace of progress in cancer therapy at some risk.  Research to compare similar treatments could “use up” thousands of patients who might otherwise be considered for participation in studies of newly developing therapies.  In addition, the energy and resources that are currently devoted to developing studies that test the efficacy of new drugs and technologies are not limitless and might be taxed by CE research.

For example, although answering the question about colonoscopy and sigmoidoscopy is worthwhile, is it as important as finding out if new molecular screening methods for colon cancer and precancerous changes could minimize or replace invasive screening?  Although these studies could be done simultaneously on the same patients, this type of coordination will take new levels of cooperation between academia, industry and government.

If we are not careful, we will find the answer to the question, which of our current treatments is most effective, when we should be applying our energy, imagination and resources to answering the question, how can we significantly improve on current treatments.   As hard as it is to believe today with the high short term costs of new anti-cancer therapies and technologies, ultimately, better treatments and technologies will improve quality and drive down cost of care.  If we focus on cost reduction as the primary goal, we may miss out on or delay the benefits of future improvements.

Richard Leff is Chief Medical Officer of Conisus.

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  • Healthcare Observer

    This is just wrong thinking. We are probably years away from effective biomarkers, and work is going on here in any case. But making improvements to the existing gold standard of care could save lives now.

  • bev M.D.

    I agree with the above comment. NO ONE outside the political realm EVER said that CE research was all about cost. It is about finding out what works and what doesn’t – like bone marrow transplants for breast cancer, for instance. Who knows how many women died prematurely by pursuing this ultimately proven ineffective treatment and developing fatal complications, when they might have had a longer and better quality of life without it. I find this post to be scientifically unsound and, perhaps, a conflict of interest (I have not looked up the company for whom the author works, but I guess I had better.)

    • George Miranda

      @bevMD: stem cell transplants for breast cancer were NOT proven ineffective. They were proved to be NO MORE EFFECTIVE than standard therapy in prolonging overall survival. However, our experience with breast cancer transplants at our center was a significantly longer “treatment-free” survival than traditional therapy; i.e. both cohorts had equivalent survival, but the transplant patients were not being constantly treated for the rest of their lives as were many of the patients treated conventionally. And while Peters, et al, had a significantly high treatment-related mortality in the early years of their trials, which significantly impacted the overall survival curves, our center had less than 3% treatment-related mortality for breast transplants. Higher than conventional treatment, but not by much.

  • db

    I have blogged angrily about this post on my site. This logic represents the worst arrogance of basic science research that I can recall seeing!

  • Marc Gorayeb, MD

    Dr. Leff outlines an excellent counterpoint to the conventional wisdom.
    There is an articulable rationale for making all treatments meeting the FDA’s safety and effectiveness requirements available to the market. Isn’t it better to have a half dozen drugs to treat a condition, providing the patient and doctor with choices to find the best individualized fit?
    Or would you rather be limited to only one or two drugs that are deemed by authorities to provide the most cost effective result for the average patient?
    What’s the point of doing research on personalized medicine if you don’t have a variety of treatments available to you?

  • Easton

    I think comparative effectiveness is a great idea in medicine, but I think drug companies would have to be dragged, kicking and screaming, to the table. Too many times, drug companies compare their drug against placebo, when there are already drugs approved to treat a medical condition. I’m not really that concerned about how the newest, greatest drug compares to placebo. I want to see how they match up, head to head, against the current drugs in a class.

    If a company brings a me-too drug to a class, they’d better prove that they’re either better or cheaper.

    Although oncology meds are a somewhat special case, I still think comparative effectiveness would be beneficial.

  • Michael F. Mirochna, MD

    How many b-blockers do we need?
    How many statins do we need?
    How many ACEs do we need?
    How many ARBs?
    How many OCPs?
    How many triptans?
    How many SNRIs?

    Maybe treatment advances in other important areas could occur if funding wasn’t being used up for more “me too” drugs to get a piece of the already succesful market. But, I guess that’s the right business decision.

    Let us not forget, the one treatment set that benefits all would be eating well, exercising and smoking cessation. The mortality benefit would be astronomical.

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