According to a report Placebos can ethically be used as a control in trials of new drugs for multiple sclerosis, even though partially effective treatments exist. (Annals of Neurology, May-2001)
American Neurological Association (ANA)
Multiple Sclerosis Research
Patients' Well Being Can Be Protected by Stringent Guidelines and Patient Education
According to a report in the May 2001 issue of Annals of Neurology, the research journal of the American Neurological Association, placebos (inactive pills) can ethically be used as a control in trials of new drugs for multiple sclerosis (MS), even though partially effective treatments exist. The multi-disciplinary Task Force on Placebo-Controlled Clinical Trials in MS was convened by the National Multiple Sclerosis Society (USA) to determine whether such practices are ethical. The Task Force adds that the administration of placebo substances should be limited to MS patients who have been educated about the current treatment options but do not appear to have benefitted from these drugs or have declined to take advantage of them.
Multiple sclerosis is a disorder of the nerve fibers of the brain and spinal cord. Depending on the nerve fibers affected, patients can experience problems ranging from weakness and clumsiness to numbness, visual disturbances, and even emotional and intellectual alterations. In some patients, MS manifests itself in cycles of relapse and remission; in other patients, the disease may progress to a stage of severe debilitation.
Treatments exist that can partially improve the condition of some patients with MS, by reducing the frequency of relapses, for instance. However, researchers are still searching for more effective treatments, as the ongoing cycles of MS relapse appear to cause permanent damage to the brain.
The research community is generally in accord that placebo-controlled trials are the most efficient, cost-effective, and decisive way to test the safety and efficacy of a drug. However, "most people in the field feel that it is problematic to utilize placebo-controlled trials in lieu of available therapy," according to lead author Fred D. Lublin, M.D., a neurologist at the Mt. Sinai School of Medicine.
The task force includes prominent researchers from the United States, as well as some from other countries, alongs with a bioethicist, and representatives from the Food and Drug Administration and the National Institutes of Health.
The panel concluded that MS patients could be given placebos during trials of new treatments as long as the study physicians have educated the patient about existing treatments and are actively encouraging them to use these treatments. This level of contact with the patient should continue throughout the trial, especially if new therapeutic information becomes available.
Bioethicist Jonathan D. Moreno, Ph.D., of the University of Virginia, who wrote an editorial accompanying the report, finds that the guidelines are especially timely and valuable in light of a recent statement by the World Medical Association (WMA) that seems to preclude the use of placebos in any trials where effective alternatives exist.
In an update to the 1964 Helsinki Declaration on the ethical use of humans as subjects in medical research, the World Medical Association was responding to criticism brought on by trials of AZT in HIV-positive pregnant women in developing countries. There was a placebo group of women in the study, two-thirds of whom were expected to bear infants infected with HIV. The WMA perceived this approach as exploitative and updated its guidelines for ethical research accordingly, by including a statement that prohibits giving placebos to study patients when a proven treatment, however effective, exists.
A potential problem with this blanket statement is that it ignores the reality that not all diseases have the dire prognosis of AIDS, nor are all drugs as effective as AZT is in protecting infants of HIV-positive women.
"In its current form the Declaration of Helsinki is unworkable, which is a sad event for international medical ethics," comments Moreno, who points out that compliance with the Helsinki Declarations is a prerequisite for research projects funded by the U.S. National Institutes of Health, the Food and Drug Administration, and the United Nations, among others.
Moreno believes that the conclusions of the MS task force represent a viable approach to this controversy.
"The task force concluded that if the risks are modest, the potential benefits significant, and the current treatment only modestly effective, then with fully informed consent a person may agree to be in a placebo-controlled study. This is a much more nuanced approach than that taken in the new Declaration of Helsinki," said Moreno.