My last post introduced a dilemma regarding modern clinical trials: Is it ethical to randomize some patients to the "control arm" of a trial where they will NOT receive the trial drug?
According a NYTimes article, one particular trial of a therapy (called PLX4032) for malignant melanoma "ignited an anguished debate among oncologists about whether a controlled trial that measures a drug's impact on extending life is still the best method for evaluating hundreds of genetically targeted cancer drugs being developed."
Some clinicians maintain their belief that the control arm is indispensable to assessing whether new therapies are better. Why the stubbornness? Because innumerable past trials that included a control arm proved promising therapies to be, disappointingly, no better than the standard therapies.
Other clinicians believe "the new science behind the drugs has eclipsed the old rules -- and ethics -- of testing them." These oncologists believe it is unethical to put patients on a control arm when the following conditions are present:
- the standard therapy has been proven to be rarely or minimally effective
- the investigational therapy has
- mechanisms of action that are understood
- shown effectiveness in pre-clinical and early clinical testing
- minimal toxicity
Today's clinicians struggle with the tension between fulfilling their obligations (1) to their individual patients and (2) to gathering scientific knowledge that will lead to better therapies for tomorrow's patients.
Tomorrow: Why I entered a Phase I trial in 1993