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Moving Beyond the Hype: Three Key Steps for Personalized Medicine to Live Up to its Promise in Cancer Care H. Jack West, MD
Talking the Talk vs. Walking the Walk Everyone, including President Obama, has jumped onto the personalized medicine bandwagon. This makes sense: it’s intuitively appealing and lends itself to a promise that it will transform medical care in general, and cancer care specifically. With all of the hype about it, one might mistakenly believe that it has actually been proven to help groups of patients significantly. It hasn’t. Here are 3 criteria personalized/precision medicine needs to achieve to move beyond breathless promises.
Thus far, so called triumphs of personalized medicine in cancer care have been based on very low hurdles. Companies and researchers tout detection of “actionable” or “targetable” mutations as the endpoint. That’s garbage. A targetable result typically means that either there is a clinical trial being done somewhere on the planet for which the patient might be eligible, or the company scrounged up a study in mice or a test tube model that tenuously suggests an unproven treatment in the world could possibly be beneficial. That’s many steps away from being truly beneficial. It’s time to aim higher. Show that personalized medicine improves patient survival. 1) Clinically Meaningful Endpoints, Because Low Hurdles are Just for Kids
2) Show Benefit in Populations, Not Just Anecdotal Cases The lottery also highlights rare winners, but most people lose. Our medical care has to be better than Powerball. For personalized medicine to prove real value, we need to see a randomized phase III trial of an entire patients who undergo genomic testing and tailored therapy vs. those who receive our best standard treatment without it. Show a survival benefit in the population of genomically tested patients in that trial and that’s really something. But we aren’t close to that yet. It’s time for personalized medicine researchers to start showing not just an example of one patient who was found to have a mutation for which there was a treatment that seemed to help for a while. That’s great for one patient, but stop ignoring the denominator: how many people were tested who weren’t found to have a treatment that helped them.
3) Improve Survival of Patient Populations without Breaking the Bank In addition to showing an improvement in patient survival as a REAL endpoint, and in clinical trial populations rather than just cherry picked patients, personalized medicine needs to be able to do this at a reasonable cost. Show that personalized medicine keeps costs at or below levels of care without it, or that it’s at least a fair value for the survival benefit. For management that costs $150,000/yr, this would allow a patient to live in a $500/day luxury hotel in Maui for 10 straight months. Would a patient want this treatment more than 10 months of aloha?
These are fair goals. But personalized medicine is still more an unrealized potential than a proven value. Broad genomic testing for cancer hasn’t yet demonstrated a survival benefit in populations of patients, and we don’t know if it will simply add cost and no significant benefit. Finding a “drugable target” shouldn’t be the goal of an intervention, especially if you need to get on a plane for a trial or try an unproven treatment outside of study to get it. Case studies of a few patients who happen to benefit from a treatment may well be the rare exception. Even a broken clock is right twice per day. In the meantime, research efforts in personalized medicine are very likely to move the field forward, but it’s worth clarifying what it delivers now vs. hopes to deliver in the future.