A new wave of cancer treatments is raising questions about the way drug trials are conducted with one group of patients barred from trying a promising new medicine–as illustrated by The New York Times in a tale of two gravely ill cousins.
Both young men had melanoma, a particularly lethal form of skin cancer. One saw his tumors stop growing after two months of taking the drug PLX4032 in trials at the University of California, Los Angeles. The other, assigned to the control group, or the group not on the experimental medication, received a chemotherapy treatment described by The Times as ”notoriously ineffective”. Tumors spread to his lungs, liver and spine. He died less than a year later.
Doctors say a new generation of genetically-targeted cancer drugs, such as PLX4032, intensifies a conflict between their responsibility to help patients and the need to generate scientifically sound data to guide future cancer treatment.
Defenders say control groups are necessary to determine whether treatments truly extend lives more than existing drugs. Critics say some new drugs are so much more effective than their predecessors that putting some patients in control groups causes needless suffering, and that more flexible rules for drug trials are needed.
Dr. Richard Pazdur, director of the cancer drug office at the Food and Drug Administration, told The Times that the new wave of drugs in development — especially for intractable cancers like melanoma — might require individual evaluation. “This is an unprecedented situation that will, hopefully, be increasingly common, and it may require a regulatory flexibility and an open public discussion,” he said.
