Today's editorial in the WSJ.

S.O.S. to the FDA

By STEVEN WALKER

Our Food and Drug Administration is often praised for establishing
the "gold standard" for drug approvals. If it is FDA-approved, folks
say, all can be sure that the drug has been rigorously shown to be
safe and effective through years of careful review. Unfortunately,
the people making this claim increasingly work at the FDA. Those
waiting for FDA decisions, mainly dying patients and those who care
for them, view the agency as a barrier to new treatments that they
desperately need to live. The agency's inability to recognize and
adjust to the accelerating pace of medical research has tarnished its
gilt.

Never has this been more evident than now. At a recent major cancer
research meeting in Chicago, two announcements were made regarding
breakthrough drugs for colon cancer , the second leading cause of
cancer deaths in the U.S. (According to the American Cancer Society
it will kill 57,000 this year.) The first drug is Imclone Systems
Inc.'s Erbitux, the not-so-new targeted drug that is inexplicably
more famous for its ill-conceived rejection by the FDA in December
2001 and the ensuing scandals than for its effectiveness as a cancer
drug.

Erbitux has once again been shown to be an important advance in
treating colon cancer. Results of the latest trials are identical to
the results the FDA rejected in 2001, and they more than meet the
FDA's current standards for accelerated approval. Since the
rejection, about 80,000 Americans have died from colon cancer without
getting Erbitux. Erbitux shrank tumors for about 23% of patients for
whom nothing else would work, and controlled the cancer for an
average of four-plus months. Considering that the best FDA-approved
treatment for colon cancer only controls tumors for about 10 months,
adding this drug to the arsenal as a follow-up treatment is a major
advance.

* * *
I know from direct observation how well Erbitux can work. Near death
in September 2002, my wife Jennifer managed to enroll in a small
clinical trial for Erbitux. The treatment lifted her off her deathbed
in two days, resolved the symptoms of her cancer in two weeks, and
allowed us to return to a normal life, skiing, hiking and working.
Many patients in the trial experienced similar results. The sole side
effect was a tolerable skin rash. Erbitux worked for six months. It
stopped working in March this year. Out of accessible options to
control her cancer , Jennifer died in June -- knowing that she was
being denied access, by a plodding government agency, to even newer
investigational drugs that might have further extended her life.

Another drug, whose results were kept secret by its sponsor and the
FDA until the Chicago cancer meeting in June, is Avastin, a drug
developed by Genentech. It extends the effectiveness of the first-
line treatment given to colon cancer patients by more than four
months, and extends survival by four months too, with almost no
increase in side-effects when given in combination with the approved
first-line treatment known as the Saltz regimen. Although not yet
comprehensively tested in late-stage, resistant cancer patients,
Avastin might have been useful to Jennifer and thousands of others
had they been able to try it in combination with other drugs.

So just like that, we now have the ability to extend the lives of
colon cancer patients by an average of more than eight months (or in
some cases longer), a significant increase when considering that
advanced colon cancer patients can expect to live little more than a
year. Tragically, patients can't get Erbitux or Avastin because of
the FDA's antiquated approach to recognizing and approving cancer
drugs. The key to availability of any new drug is approval by the
FDA, and neither drug is likely to be approved sooner than early next
year. The drugs can't be purchased for any price, and aren't
available outside small clinical trials and a small expanded access
program for Erbitux. The FDA has six months to review Avastin and
Erbitux from the date they receive complete applications. The
application for Erbitux was submitted on Aug. 14, and an application
has yet to be submitted for Avastin. Before blaming the companies for
the time they are taking to file their paperwork, understand that the
FDA is a notoriously nitpicky agency, concentrating on the most minor
details even when those are not relevant to those who will be treated
with the drug. Americans shouldn't die, for example, because the FDA
is hung up on a few words in the package labeling.

The great majority of those finding out they have advanced colon
cancer in the coming months will not get Avastin with their first-
line treatment, costing them an average of at least four months of
life. Nearly all of those finding out that their cancers will no
longer respond to the existing approved treatments will be denied
access to Erbitux, costing them at least four months of control of
their disease. Some might quickly blame the companies for not giving
the drugs away, and the FDA will claim they would allow this if the
companies would do it, but no company can afford to treat thousands
of patients for free with drugs that cost hundreds of millions to
develop, produce and administer.

So just like that, two significant victories in our war on cancer
will be denied to cancer patients. Using a conservative estimate
based on American Cancer Society numbers for new cases and deaths,
and the clinical trial results, about 14,500 Americans will be denied
Avastin and about 28,500 will be denied Erbitux over the next six
months while the FDA waits for and processes paperwork, assuming it
reviews the applications quickly, by no means a certain prospect. The
cost in human life adds up to about 14,300 years. If approval takes
longer the losses will mount. The actual cost in life will be further
increased because off-label use for patients with other forms of
cancer will also be precluded. The situation with Erbitux and Avastin
is not isolated. It is business as usual.

At the FDA, the process and strict adherence to regulations, guidance
and policy always comes first, and the agency's power over
availability of drugs is absolute. My wife's battle with cancer and
the setbacks she suffered at the hands of the system are typical of
the challenges faced by all Americans fighting life-threatening
diseases. Too many people are dying at the hands of a bureaucracy
that does not have an approval mechanism that could ease the loss of
life.

We at the Abigail Alliance for Better Access to Developmental Drugs
and the Washington Legal Foundation have given them one. Called "Tier
1 Initial Approval," it lowers the barriers imposed on cancer
patients by the FDA's gold standard. It would give the agency the
ability to respond to those with immediate needs without weakening
its ability to ensure that new drugs are safe and effective. In fact,
it would strengthen our drug development system, forcing it to be
more responsive to the patients it exists to serve.

As Mark McClellan, the new FDA commissioner, continues his efforts to
repair inherited problems with the regulatory process, he also should
race to modernize his agency from the ground up. Doing less will
render his agency incapable of keeping pace with accelerating medical
breakthroughs that are already transforming the prospects of some ill
Americans from despair, to hope, to life. Some will oppose him
vigorously because old ways die hard.

We are finally beginning to win the war on cancer. The cancer
patients have always been courageous foot soldiers in the fight,
contributing mightily in clinical trials to get us here. It is now
time to see if there are heroes at the FDA with the vision, courage
and resolve to clean the tarnish from our gold standard. A lot of
lives -- and very possibly yours -- depend on it.

Mr. Walker, adviser to the Abigail Alliance for Better Access to
Developmental Drugs, is the husband of the late Jennifer I. McNeillie.