The nine committee members cast separate votes on safety and effectiveness. They voted unanimously in favor of the FDA approving Rilutek on safety grounds. The issue of effectiveness was tougher. The main controversy concerned the phase three data comparing patients taking various doses of Rilutek with those taking placebo, both in Europe and the United States. Effectiveness was clearly shown in the European studies, but in the American studies there was little or no difference between patients taking the drug and those taking placebo.
Partridge questions the validity of focusing on one portion of the data.
Statistical significance was reached when you included data from all 959 patients. Nevertheless, some committee members balked at approving a drug for the American market if it had been shown to be effective in Europeans but not in Americans. But the bottom line is that that wasn't the question. Miller recalls that another point of debate had to do with the fact that the effect of the drug is modest. The FDA administrator again rightly reminded them that their charge was not to assess the size of the effect.