"The FDA's Deadly Track Record"
By RONALD L. TROWBRIDGE and STEVEN WALKER
August 14, 2007; Page A17

Last week, the full D.C. Circuit Court of Appeals reversed an
earlier decision by its own three-judge panel and ruled 8-2
against a dying patient's right to pursue life by taking
investigational -- but as yet FDA-unapproved -- drugs.

The case was filed in 2003 by the Abigail Alliance for Better
Access to Developmental Drugs and the Washington Legal
Foundation. We argued that terminal patients with no options left
but death have a constitutional right to such therapy in the care
of a qualified physician.

The Alliance began pushing for access to investigational drugs
for terminal patients after its founding in mid-2001 upon the
death of Abigail Burroughs, who was denied an investigational
drug (Erbitux) that an early trial showed might have helped her.
She and her doctor were right, but she never got the drug.

Over the past five years, the Alliance has pushed for access to
12 exceptionally promising investigational cancer drugs which
have subsequently been approved by the FDA and now represent
standard care. At the time we began our advocacy, each of the
drugs had cleared at least preliminary Phase 1 testing, and in
some cases more-advanced Phase 2 or Phase 3 trials. In other
words, they obviously worked for some patients.

Gleevec set a tragic standard for loss of life at the hands of
FDA bureaucrats. Coming out of Phase I testing in 1998, it was
known beyond any reasonable doubt to be safe and effective. The
Alliance started requesting access to the drug for chronic
myelogenous leukemia (CML) patients in June 2001. By the time FDA
approved Gleevec in March 2003, approximately 3,600 patients had
been denied access to the drug. Many died waiting. More than 80%
of the small number of patients who got Gleevec in clinical
trials before the drug was approved are alive today.

Eloxatin, for advanced colorectal cancer, was summarily rejected
by the FDA in March 2000 despite its being approved in at least
29 other countries. In January 2002, we started to ask the FDA to
allow patients access. The agency delayed approval until August.
In between, about 40,000 Americans died without ever getting the
drug.

Erbitux, for the treatment of colorectal and head and neck
cancers, was rejected by FDA in December 2001 when the agency
refused to review the sponsor's application. The Alliance had
begun asking the FDA to allow patient access to the drug six
months earlier. The FDA delayed approval until February 2004.
Almost 179,000 people with colorectal and head and neck cancer
died waiting.

The Alliance began working for access to Revlimid, for multiple
myeloma and myelodysplastic syndrome, in June 2002. Patients had
to wait until December 2005 for FDA approval. Nearly 74,000
patients with these terminal cancers died without ever getting
Revlimid.

The Alliance asked that patients get access to Velcade in June
2002. Curiously, the FDA points to this drug as proof it can work
fast, but they didn't approve it until May 2003. At the time,
trial results suggested that only about 25% of multiple myeloma
patients should get the drug (since shown to be too low), but
even with that limitation, about 2,600 patients died without ever
getting Velcade.

Beginning in June 2004, we started pushing the FDA to make
Nexavar and Sutent, both highly promising drugs for kidney
cancer, available. The agency eventually approved Nexavar in
December 2005 and Sutent in January 2006. But that was only after
evidence of efficacy so compelling emerged for Nexavar that the
trial demanded by the FDA -- in which dying kidney cancer
patients seeking the drug were being given no other choice
(except certain death from their cancer) but to agree to a 50/50
chance of being blindly randomized to a sugar pill -- was stopped
by Bayer for ethical reasons and the placebo patients allowed to
get the drug. The sponsor seeking approval for Sutent was given a
similar option by FDA if it wanted its drug approved. About
20,000 kidney cancer patients died waiting for both drugs.

The Alliance began its push for availability of Avastin for
multiple cancers in June 2002. FDA finally approved this
obviously effective cancer drug in February 2004. It is now
approved for colorectal and lung cancers, and being successfully
used off label for several more. Almost 360,000 patients with
lung and colon cancer died without ever getting Avastin.

Tarceva is used for patients with lung cancer. We began pushing
for its availability in June 2001, the FDA approved the drug in
November 2004. In the interim, 531,000 people with lung cancer
died. Tarceva also extends the effectiveness of an existing drug
for pancreatic cancer, and about 102,000 patients died from that
disease during the FDA's delay.

In June 2002 we started pushing for availability and approval of
Bexxar for non-Hodgkin's lymphoma. FDA, after rejecting and
delaying this highly effective drug repeatedly over several
years, finally approved it under intense pressure from
oncologists in June 2003. About 26,000 died during the delay
without ever getting the chance to try the drug. The FDA's
regulatory hatchet job on Bexxar prior to its approval has caused
the drug to be dramatically underused, extending the damage done
by the agency's intransigence and incompetence.

In June 2002 we began our efforts to gain access to Alimta for
lung cancer patients. FDA didn't approve it until February 2004.
In the interim, approximately 249,000 lung cancer patients died
without the chance of trying this drug to see if it would control
their disease or extend their life.

The alliance started working for access to Tykerb for breast
cancer in June 2004 but the FDA didn't approve the drug until
March 2007. About 25% of breast cancers include the biomarker
predictive of benefit from Tykerb; nearly 28,000 women who had
this marker died from their cancer waiting for Tykerb. They
would, according to the FDA, have each lived an average of eight
months longer. Long enough, perhaps, to see a child graduate from
college or get married, or to meet a new grandchild.

In sum, these 12 drugs -- had they been available to people
denied entry to clinical trials -- might have helped more than
one million mothers, fathers, sons and daughters live longer,
better lives. We have actually underestimated the number of
"life-years" lost at more than 520,000, because we have not
included other safe and effective uses of these drugs that the
FDA has yet to approve.

Recently, it was decided that Provenge (another drug we have been
trying to get for years) will be kept away from prostate cancer
sufferers for up to three more years. The reason for the delay? A
small but aggressive club of FDA advisers hand-picked by the
director of the agency's Office of Oncology Drug Products, Dr.
Richard Pazdur, think the statistics are not yet perfect enough.

Recently, the FDA responded to our lawsuit by proposing "new"
regulations governing access to investigational drugs. They
propose to change nothing.

The American Cancer Society reports that some 550,000 cancer
patients die annually, making the number of cancer deaths from
1997 to 2005 about 4.8 million. Over that same period, the FDA
reports granting individual access to an investigational drug to
not more than 650 people per year for all diseases and drugs -- a
pathetic, even cruel, pittance. A few thousand more patients
managed to gain access by enrolling in relatively small clinical
trials or exceedingly rare expanded access programs.

The other 4.7 plus million cancer patients, not to mention
millions more with other diseases, were abandoned to die, denied
access to progress by their own FDA when they needed it most.

We will appeal the decision in Abigail Alliance v. Eschenbach to
the Supreme Court, and agree with only one thing in the majority
opinion. Congress should pass our pending legislation, called the
Access Act, now. It should be added to the FDA reauthorization
bill headed for a vote in September.

This is massive human tragedy, made even worse by the fact that
it didn't and doesn't have to be this way. Looking at FDA
automatons and the D.C. Circuit Court brings to mind T. S.
Eliot's question, "Where is the wisdom we have lost in
knowledge?"

Messrs. Trowbridge and Walker volunteer, respectively, as adjunct
scholar and chief adviser to the Abigail Alliance for Better
Access to Developmental Drugs. Mr. Walker also is co-founder of
the Abigail Alliance.

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