The concept of personalized or precision medicine is hot enough that President Obama is launching initiatives for it, but how close is it to moving beyond hype? Here are 3 key steps we need to attain to know that personalized medicine, particularly in the world of cancer care, isn't just delivering false hope for most patients.

1. Moving Beyond the Hype:
Three Key Steps for Personalized Medicine to
Live Up to its Promise in Cancer Care
H. Jack West, MD

2. 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.

3. • 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

4. 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.

5. 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?

6. 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.