Sure to be controversial is a plan to give promising new cancer drugs to some patients in clinical trials and to give placebos to other patients.
Some cancer researchers strongly oppose the use of placebos. Placebo trials have not generally been used in life-threatening diseases such as cancer, because it has been considered unethical to give placebos when any kind of effective therapy might be available. However, some companies, including Bayer, Pfizer, and Genentech, are adding placebo arms to their trials in an effort to speed promising new drugs to market. Because placebo trials make it easier to verify results, the strategy can reduce the need for additional studies and lead to faster regulatory approval.
Many in the cancer community maintain that this approach denies desperately ill people a last best hope. The M.D. Anderson Cancer Center in Houston and the University of Michigan Cancer Center in Ann Arbor have refused to put patients in clinical trials that use placebos. Patient-advocacy groups have urged companies to change their minds about running trials with placebo arms.
It has become difficult to get patients to participate in cancer-drug studies in the first place. One reason is that patients fear getting a placebo; patients receiving placebos have died sooner than those receiving drug therapy.
Some pharmaceutical companies say that the very nature of these new cancer drugs makes it imperative to have a placebo arm for comparison. Unlike traditional chemotherapy, which is designed to shrink or eradicate tumors, many of these drugs aim to stop or slow tumors’ growth and allow some patients to live with their cancer. This makes it difficult to measure whether it is the drug that is working or whether the tumor is simply less aggressive.