[Ip-health] Dying Patients Fight the FDA

[Joana Ramos]

http://www.businessweek.com/print/technology/content/jul2007/tc20070724_550=
192.htm

Business Week
News Analysis July 25, 2007,
Dying Patients Fight the FDA
A lawsuit could make access to experimental medicines a constitutional
right, and alter how drugs for life-threatening diseases are developed
(copied as fair use)

by Catherine Arnst

Everett Davis had been living with a rare, deadly blood disease for more
than a decade. By 2005 he needed a new liver to survive, but doctors
would not do the transplant unless they could figure out how to keep the
disease from destroying the new organ. His one chance: an experimental
drug called Soliris from Alexion Pharmaceuticals Inc.. Yet he didn't
qualify for the clinical trial. His doctors, friends, and family members
all searched for a way to get him the drug, finally calling his senator,
Arlen Specter (R-Pa.), who intervened. "I was informed on Thanksgiving
of 2006 that I would get the drug," says Davis, 32. "It's the best news
I ever got."

Some 97% of the sickest patients are ineligible for or don't have access
to the clinical trials that are testing experimental medicines. They're
getting help from patient advocates who are aggressively lobbying
Congress, other patients, and the Food and Drug Administration to either
speed up approval or relax rules restricting access to clinical drugs.

Landmark Case?

Before long, the results of that effort may prove more than anecdotal. A
lawsuit by a group of patient advocates could make access to
experimental medicines a constitutional right, resulting in a dramatic
shift in the way medicines for life-threatening diseases are developed
and regulated.

The Abigail Alliance for Better Access to Developmental Drugs filed suit
against the FDA in 2003 seeking expanded access to experimental drugs
for terminally ill people. The nonprofit group was founded by Frank
Burroughs in 2001 after his 21-year-old daughter and only child,
Abigail, died of head and neck cancer. She had been turned down for
clinical trials for ImClone Systems' (IMCL) Erbitux and AstraZeneca's
(AZN) Iressa, both of which went on to win FDA approval. After a long
legal battle, a three-judge panel from the District of Columbia Circuit
Court of Appeals ruled last year that the case could be heard in
district court.

The FDA requested that the court's full, 10-judge panel consider the
case, and its decision is widely expected this summer. Whichever side
loses is sure to appeal to the Supreme Court. "This could be a landmark
civil rights case," Burroughs says. That right, if the Abigail Alliance
prevails, would let a terminally ill patient try any drug that has
proven safe in early-stage human studies, even if there is no evidence
of its efficacy.
Cost Concerns

The prospect of such a right is disquieting to drug companies that say
they can ill afford to make experimental drugs more widely available.
There are currently more than 600 experimental cancer drugs in
development. All must go through years of testing in costly clinical
trials. Most will fail at some point.

Very sick patients who can't get into a clinical trial can petition the
company and FDA to try some drug on a so-called compassionate use basis,
but few companies offer such an option because the drugs are usually
provided free of charge (insurers typically will not pay for
experimental treatments).

"Very small companies like ourselves don't have the resources to give
away drugs free on an ongoing basis," says Timothy Walbert, chief
executive officer of IDM Pharma (IDMI), which is seeking FDA approval of
a drug for a rare form of childhood bone cancer called osteosarcoma=97a
disease that has its own advocacy group pushing for approval of the IDM
drug. "There are only about 900 kids a year in the U.S. who get this
disease," Walbert says. "We can't afford to provide it free to all the
kids who need it."
Provenge Firestorm

Even cancer specialists, who often seek compassionate use programs for
their patients, aren't keen on expanded access. "I understand
emotionally why patients would want this right, but the issues
surrounding experimental drugs are very complex, and I think this could
potentially backfire," says leading oncologist Dr. George Demetri of the
Dana-Farber Cancer Institute in Boston. "Patients really need to know
the relative benefit of a drug versus the relative risk, and expanded
access could prevent them from gaining that insight."

Activist patients, however, are disinclined to wait for such data when
it comes to diseases for which treatments are few to nonexistent.
Prostate cancer patient advocates in particular are up in arms these
days over the FDA's controversial refusal in May to approve Dendreon's
(DNDN) Provenge without further clinical trials, which could take a year
or more to complete. Provenge is a novel cancer vaccine meant to prime
the body's immune system to attack tumors, and if approved would have
been the first new treatment in more than 20 years for advanced, stage-4
prostate cancer, which is almost always fatal. Some 30,000 men die each
year in the U.S. from prostate cancer.

In a trial, Provenge failed to stop the disease from progressing, but
when it dug into the data it found that patients on the drug lived an
average of 4.5 months longer than those on standard treatment. Despite
the uneven results, an FDA advisory panel voted 13-4 in March to
recommend the drug for approval, and the FDA usually follows such
recommendations. So when the agency withheld approval on May 9, a
firestorm erupted. Prostate cancer activists (and disgruntled Dendreon
investors) have lobbied Congress, held demonstrations, and met with the
FDA's Commissioner, Dr. Andrew von Eschenbach.
Success Story

FDA officials say they do listen to such patient pleas. "The patient has
a right to be heard," explains Patty Delaney, director of the FDA's
cancer liaison program and herself a cancer survivor. "But in the end,
it's the data that matters. FDA opinions about safety and efficacy are
always based on data."

Delaney also notes that the FDA is very amenable to patient requests for
compassionate use of an experimental drug, if the company developing the
drug has such a program. Most smaller companies do not, however,
including Dendreon, which has never offered Provenge on a compassionate
use basis. If the Abigail Alliance wins its case, Delaney worries that
disreputable companies might start selling experimental drugs that they
know have slim chance of winning FDA approval. "You could really hype
something up," Delaney says. "Why would you ever do clinical trials in
any meaningful way if you are already allowed to sell the drug?"

If the Abigail Alliance prevails with its lawsuit, patients would have
to prove they have exhausted all standard treatments, and are not
eligible for clinical trials, before receiving an experimental drug. The
group also hopes a successful lawsuit will lead to insurers agreeing to
reimburse for experimental drugs under those conditions. "There has to
be some kind of incentive for companies," Burroughs says.

If the business approach doesn't work, patient advocates can point to
success stories like Davis'. Within months of getting the go-ahead to
use Soliris, he was well enough to get a liver transplant, in February,
2007. The FDA approved the drug a month later, on March 19. "I'm quite
sure I would not have made it until March," he says. Today, Davis is
healthy, working, and, together with his wife, in the process of
adopting a child.

For more, visit BusinessWeek's slide show.

Arnst is a senior writer for BusinessWeek in New York.

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Joana Ramos, MSW
Cancer Resources & Advocacy
Seattle WA USA
+1-206-229-2420
http://ramsolink.info/
www.cancersurvivorsproject.org
www.healthyskepticism.org
www.mavinfoundation.org