Clinical Drug Trials Helpful or Harmful? Dilemma of Using Drugs to Prevent Diseases in People Who are at Risk but Otherwise Healthy, Especially When the Drugs Have Serious Side Effects

Host: Nick Adams; Reporter: Joe Palca

Reprinted with permission of National Public Radio

NOAH ADAMS, host: Any doctor will tell you that preventing a disease is better than treating one. But disease prevention can be tricky. Virtually anything you do to lower the risk for one disease raises the risk of something else. For example, even a daily jog to prevent heart disease puts you at greater risk for sprained muscles and twisted ankles. As part of a year he just spent as a Kaiser Family Foundation Media Fellow, NPR’s Joe Palca did some research on the difficulties of designing studies aimed at preventing disease.

JOE PALCA reporting: Paige O’Brien is 55 years old. Three years ago, her mother died of breast cancer.

Ms. PAIGE O’BRIEN (Daughter of Breast Cancer Victim): And then three months after my mom passed away, my sister got a breast cancer.

PALCA: That hurt a lot, since her mother was elderly but her sister was not even 60. Paige’s sister had surgery, radiation and chemotherapy, then started taking a drug called tamoxifen. Unfortunately, none of the treatments worked. As a woman with a mother and sister who both died from breast cancer, Paige’s risk of getting the disease is much higher than most women her age. So before she died, her sister gave Paige some advice.

Ms. O’BRIEN: She encouraged me to speak with a doctor about possibly taking tamoxifen or if there was anything I could do to help reduce my risk.

PALCA: Although it didn’t work for Paige’s sister, tamoxifen is quite often effective in treating breast cancer, and a recent study has shown it may be able to prevent breast cancer from developing in the first place. Paige’s doctor suggested she join a new clinical study that is comparing the effectiveness of tamoxifen to a similar drug called raloxifene. Paige jumped at the chance.

Ms. O’BRIEN: I feel that it’s such an honor to be able to do this not just for myself, but for future generations of women who might be face with the risks of breast cancer that I do.

PALCA: But people like Paige who enthusiastically embrace clinical studies of a drug to prevent disease make biomedical ethicist Daniel Sulmasy uncomfortable. Sulmasy, who works at St. Vincent’s Hospital in New York, says before giving a healthy woman like Paige any drug, you have to be absolutely certain you’re doing her more good than harm.

Mr. DANIEL SULMASY (Biomedical Ethicist, St. Vincent’s Hospital): People have this profound thought that medicine, you know, can prevent them from being sick, and we all want to believe that.

PALCA: Sulmasy says that may make it hard for researchers to convince people there’s a downside to taking a drug. Even if she does nothing, Paige may never get cancer. But if she starts taking a drug like tamoxifen, she exposes herself to tamoxifen’s rare but very real side effects like stroke and a kind of uterine cancer. Sulmasy says people have a faith in medicine that leads to something he calls the prevention misconception, where people think just because a new drug is being tested, it will help them.

Mr. SULMASY: I think that those who are conducting the trials have an especially important moral obligation to be sure that what they’re doing has a reasonable study beforehand that suggests that it’s safe before they even offer it to people, because that sort of prevention misconception is so great that as soon as they offer it, people are going to snap it up.

PALCA: Sulmasy believes modern medicine is in part to blame for the prevention misconception because it has oversold medical science’s ability to help people. But Norman Wolmark thinks Sulmasy’s concerns are overblown. Wolmark runs the research group conducting the study of tamoxifen and raloxifene for preventing breast cancer, the study Paige O’Brien entered. He says women will only snap up tamoxifen when they are convinced it will help them. As proof, he points to a large study of tamoxifen as a prevention for breast cancer his group completed in 1998. Wolmark says that study showed that tamoxifen did reduce the number of breast cancers in women who were at increased risk because a mother or sister had gotten the disease.

Mr. NORMAN WOLMARK (Head of Research Group): There was a great fear that we’d see millions of women go on tamoxifen for prevention, and that has not occurred. I think people are being thoughtful and selective about going on tamoxifen in the prevention setting.

PALCA: Still, Wolmark admits that doing research designed to prevent breast cancer is entirely different from research on new drugs to treat the disease. And while studies of tamoxifen have clearly shown it reduces the number of cases of breast cancer, those studies have also shown that tamoxifen does increase the number of cases of strokes and uterine cancer. That leaves some wondering whether, in the aggregate, it’s clear that tamoxifen is helping. Oncologist Mary Daly of Fox Chase Cancer Center is one of the lead researchers on a new breast cancer prevention study. She says in treating cancer, doctors are used to giving powerful drugs with serious side effects because their patients need them to stay alive. But in prevention, people aren’t sick. It’s one thing to routinely add a benign vitamin to milk to prevent bone loss; it’s quite another to give a healthy woman a drug that can make her sick to prevent a disease she may never get. Daly says researchers have to remember that.

Ms. MARY DALY (Oncologist, Fox Chase Cancer Center): We’re not used to treating healthy, unaffected individuals. So there’s a little bit of lack of training in terms of thinking about primary prevention and the perspective that one would have. But I think physicians are willing and interested, particularly if, like me, you’ve seen hundreds of women die of breast cancer. I mean, I want to prevent it, so I’ll do anything to try and prevent that from happening to another woman.

PALCA: Even those who believe that tamoxifen is an appropriate drug to give some healthy women who are at high risk for breast cancer, no one thinks that tamoxifen is the ideal prevention drug because of its side effects. The challenge is for the medical community and the pharmaceutical industry to be scrupulous about not suggesting that it is, and for patients to remember that prevention can carry its own price. Joe Palca, NPR News, Washington.



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