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24 March 2005, The Wall Street Journal
A14
(Copyright (c) 2005, Dow Jones & Company, Inc.)
Of all the opinions being expressed about
the Terri Schiavo case, the hardest to understand is
cynicism about the politics of Congressional intervention.
Only phony "federalists" question Washington's
competence on matters of fundamental rights. And whether
you side with Mrs. Schiavo's parents or husband, it
is hard not to be impressed by the spectacle of the
nation's highest legislative body convening over the
fate of a single person.
No, if our solons have exposed themselves
to criticism by taking up the Schiavo case, it should
be focused not on their motives but on their inconsistency
and lack of proportion. To wit: If Terri Schiavo deserves
emergency federal intervention to save her life, people
like Kianna Karnes deserve it even more.
The 44-year-old Mrs. Karnes -- mother
of four and grandmother of one -- is not brain-damaged.
And the possibility (albeit remote, at this point) exists
that she could return to a fully normal life. But she
will almost certainly die in the near future as long
as the federal government continues to deny her treatment
for the kidney cancer that has by now spread throughout
her body.
What makes Mrs. Karnes's predicament so
depressing is that two different developmental drugs
have shown great promise for several years now against
this once near-untreatable disease. But not only has
the Food and Drug Administration not moved with dispatch
to approve the drugs, it has begun imposing new testing
requirements that make it all but impossible for their
developers -- Bayer and Pfizer -- to provide them to
terminal patients on a "compassionate use"
basis.
The problem here is the FDA's unethical
-- and let us stress, unscientific -- insistence on
gathering information about drugs by way of "blinded"
placebo-controlled trials, in which a subset of study
patients are knowingly denied the new treatment and
in some cases denied access to any active treatment
at all. This may be moral with an antihistamine; it's
certainly not with treatments for a terminal disease.
What's more, it's entirely unnecessary. We already know
what happens to most cancer patients who don't get treated.
They die. We generally know, on average, how long that
will take.
So placebo groups are entirely unnecessary
to prove significant anti-cancer activity, as the yet-unnamed
Bayer (BAY 43-9006) and Pfizer (SU 11248) compounds
have already done. Yet the FDA is mandating an unethical
placebo trial for the Bayer drug. (The Pfizer drug is
at least being tested against another form of care,
albeit one that's already all but certain not to work
as well.) A deadly follow-on effect of the placebo fetish
is that it gives companies a disincentive to run compassionate
use programs for unapproved drugs. That's because companies
won't be able to satisfy FDA demands to enroll patients
in placebo trials if patients know they can get the
drug for sure (instead of running the risk of getting
a sugar pill) through compassionate use. Hence Mrs.
Karnes's deadly predicament.
"If the only alternative is death,
then for God's sake let 'em have the drug," says
Mrs. Karnes's father, John Rowe, who himself survived
leukemia only by getting himself into a clinical trial
where he could get another investigational therapy (Gleevec,
since approved). Who could disagree?
Well, a few bureaucratic MDs at the FDA
do. More specifically, one Richard Pazdur. He is the
current head of cancer drugs evaluation at the FDA,
and is unfortunately a leading candidate for a new position
that would give him the power to thwart the would-be
revolution in biotech cancer treatments as well.
Late last year we reported how Dr. Pazdur
had undermined -- in fact, totally reversed -- the meaning
of guidelines issued by former Commissioner Mark McClellan
intended to speed up drug reviews. His latest attack
on the concept of accelerated approval has him demanding
that companies enroll patients in placebo-controlled
"Phase 3" trials before submitting applications
for very promising drugs that should be eligible based
on smaller "Phase 2" studies.
A few years back, Dr. Pazdur was the agency's
public face in explaining the rejection of Erbitux.
That drug has since been approved and become a clinical
hit. So has Eloxatin, which the FDA held up for years
even after it was approved in Europe but has since become
standard care for colon cancer here too.
In almost all recent cases of FDA dawdling,
the drugs are proving to be far more beneficial in practice
than even the supposed "gold-standard" of
placebo trials would have ever suggested. So could someone
explain, again, what the benefit is of doing such trials?
We're not suggesting Dr. Pazdur is some kind of ogre.
But he seems to be more worried about letting drug companies
get away with a so-called "race to the bottom"
on trial design than he is with getting good drugs to
patients. And it's obvious that he can't (or won't)
be educated in modern scientific and statistical methods
that would allow drugs to be released sooner.
We've never understood why the Republican
majority in Washington hasn't been more active on drug-approvals
over the past four years. What better way to demonstrate
compassionate conservatism and commitment to a "culture
of life"? Or to unite the free-market wing of the
GOP with the social conservative one? Finally, what
better riposte to the left's equation of support for
embryonic stem cell research with support for medical
progress?
The solution should be non-controversial.
We're not talking about potential Vioxxes that will
be widely used by generally healthy people. We're talking
about treatments for dying patients. So let's have legislation
mandating that the FDA grant access to these drugs as
soon as they show anti-cancer activity.
Instead of restricted-access placebo trials,
drug researchers could be using large, open access trials
in which everyone who wants the new drug can get it.
They could then take advantage of advanced statistical
methods to figure out whether the drug is working. Wall
Street traders use these kinds of math tools all the
time, and so do economists. So-called Bayesian statistics
are already used in medical device regulation, where
even the FDA recognizes that randomizing people into
sham surgeries is simply beyond the pale.
Well, what about cancer and other terminal
patients? They are now dying needlessly in placebo-controlled
trials. And would-be patients like Kianna Karnes are
dying outside of them because they make "compassionate
use" all but impossible. Won't Congress do something?
(Copyright (c)
2005, Dow Jones & Company, Inc.)
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