Dying Patients Fight the FDA

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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.

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Arnst is a senior writer for BusinessWeek in New York.