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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

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—a 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.

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