FDA Internal Document: Use of Placebo-Controls in Life Threatening Diseases

The efficacy and safety of a new drug that treats a serious and life threatening illness in premature infants will be studied versus sham/placebo in Latin America. The sponsor plans to apply for FDA approval, in addition to local and European registration. There are approved therapies (surfactants) for this illness (Respiratory Distress Syndrome, or RDS) in those countries where this trial is proposed to take place, and surfactants are even used in some of their hospitals. However, surfactants are completely unavailable to infants at many other hospitals, secondary to rationing or economic limitations. Continue reading →