Clinical Trials Supporting FDA Approvals of New Medications for Life-Threatening Diseases Must Be Held to a Higher Standard

Clinical Trials Supporting FDA Approvals of New Medications for Life-Threatening Diseases Must Be Held to a Higher Standard

New Study Raises Serious Ethical Concerns About the Use of Placebo-Only Control Groups in Clinical Trials and Impact on Human Subjects

WASHINGTON, D.C. – The use of placebos in clinical trials testing new medications for life-threatening diseases – for which treatments proven to be effective already are available – is widespread and can put human subjects at increased risk of harm, according to a new study by Public Citizen’s Health Research Group. The study was published today online in Open Access Journal of Clinical Trials.

The study examined the treatment provided to control subjects in 508 trials that supported the U.S. Food and Drug Administration’s (FDA) approval of new medications for nine life-threatening diseases – asthma, bipolar disorder, chronic obstructive pulmonary disease (COPD), diabetes mellitus, hypertension, osteoporosis, Parkinson’s disease, partial seizures and schizophrenia – from 2006 to 2011.

Of those trials, 201 (40 percent) were placebo-only-controlled – meaning that subjects in at least one control group did not receive an active intervention. This practice potentially placed more than 19,000 subjects in placebo-only control groups in these trials at increased risk of harm, according to the study.

Placebo-only-control groups were used in 77 percent of trials for severe COPD, 56 percent of trials for severe hypertension, 44 percent of trials for moderate or severe persistent asthma and 7 percent of trials for severely uncontrolled diabetes, according to the study. In addition, the study found that 16 percent of trials lasting between 26 and 52 weeks had placebo-only control groups.

“These findings should alarm any patient who is seriously ill and considering enrolling in a clinical trial,” said Dr. Michael Carome, director of Public Citizen’s Health Research Group and one of the study’s authors. “When human subjects with life-threatening diseases are given placebos and are therefore deprived of available, approved treatments that have been shown to be beneficial, they may be placed at significant risk of harm, especially if they have severe disease or the trial has a long duration.”

“Our finding that placebo-only-controlled trials are commonplace – even in trials enrolling patients with severe, life-threatening diseases – demonstrates the need for the FDA to revise its guidance on the use of placebos in such trials whenever proven alternative treatments are available,” said Dr. Sidney Wolfe, founder of and senior adviser to Public Citizen’s Health Research Group. “Moreover, trials with placebo-only control groups should require much greater scrutiny by sponsors, researchers, institutional review boards and the FDA.”

The study acknowledged in its limitations that it relied on the FDA medical reviews and other publicly available sources, raising the possibility that important data related to the trials was not reported. It also called for future research to assess how the use of placebo interventions in trials supporting FDA approval of medications adversely impacts placebo-group subjects.

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