Interesting...but why not tell the full story? The Zytiga trial that was stopped
in March, 2012, achieved statistical significant in only one of two primary
endpoints...progress​ion free survival (PFS). The trial was not stat sig for
overall survival (OS), which is the Gold Standard for FDA approval of cancer
drugs. Further, if you look at the data presented by JNJ in Chicago on Saturday,
you see there was no effect on survival noted until month 18 after initiation of
therapy in the treatment arm...4 months after therapy was stopped. By that time,
20% of the patients had died. What kind of treatment is that? ("Sir, we're going
to give you this drug...we're not sure it's going to work...check back in 18
months, if you're still alive, and we might know!") Zytiga costs more than
$5,500 per month, it was given for 14 months in the study, so not including the
prednisone that must be taken daily with it (cheap), the drug tests necessary to
monitor liver functions (moderately expensive), and the costs to address side
effects (possibly high), total cost for a treatment, depending on length, could
push up into Provenge's space. If a person is on Medicare and is pushed into the
'donut hole,' the copay could run $500-$800 per month. Recent data indicate 70%
of patients on Provenge have little or no out-of pocket expenses, and the
remainer incur minimal costs. Finally, the trial was stopped...the data JNJ will
take to the FDA are those that existed at the time it was stopped. A noted
statistician who presented on Saturday at ASCO in Chicago stated that the trial
had failed. Frankly, many were puzzled as to why it was stopped, when another
interim look by the Independent Data (Safety) Monitoring Board would have been
conducted in the June time-frame. By that time, it was conceivable the trial
would have evidenced stat sig OS, making the case for approval stronger. There
was talk at ASCO (and even prior to that) that JNJ faced mounting pressures from
patent expirations and competition, which led them to halt the trial early and
take their chances on approval with the FDA. Given the overall survival is THE
'gold standard' for approval by CDER/ODAC, it will be interesting to see if the
Cancer Czar will compromise his principles and bow to JNJ on this one! Yes,
Provenge's label states that it provides a MEDIAN survival advantage of 4.1
months (half of the men who take it die before 4.1 months, the other half live
longer, some as much as 6-7 years). But recent studies that account for the
crossover of patients in the placebo arm of Dendreon's pivotal Phase 3 study
(that is, patients who left the placebo arm and took a frozen form of Provenge)
indicate the life extension benefit ranges from 7.8 to 12.1 months. Now, I don't
expect the press to know or understand these nuances, but let's at least be a
little more 'fair and balanced' in our approach to these stories. We are not
describing the rescue of a dog from a well. We are talking about cancer drug
trials.