A prospective, randomized, double-blind, controlled clinical trial is the most rigorous clinical trial design, and the one that regulatory agencies mandate MUST be CONDUCTED to demonstrate a medication’s effectiveness and safety. In a NEW drug application, these studies represent the highest quality data regarding the drug and its actions, and form the basis for approval. In this study design, patients are carefully selected for participation and are randomly assigned to receive the experimental drug or a matching active drug or placebo. Neither the patient nor the TREATING physician knows which treatment was provided, thereby eliminating possible bias.

Individual definitions of the study descriptions are:

Prospective: Forward looking, beginning before the patient has started treatment.

Randomized: Patients are randomly assigned to receive the experimental treatment or alternative (e.g. STANDARD of care or placebo)

Double-blind: Neither patients nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo or standard treatment.

Controlled: One group of patients will be given an experimental drug or treatment, while a second group is given either a standard treatment for the illness or a placebo.

A prospective, randomized, double-blind, controlled clinical trial is the most rigorous clinical trial design, and the one that regulatory agencies mandate must be conducted to demonstrate a medication’s effectiveness and safety. In a new drug application, these studies represent the highest quality data regarding the drug and its actions, and form the basis for approval. In this study design, patients are carefully selected for participation and are randomly assigned to receive the experimental drug or a matching active drug or placebo. Neither the patient nor the treating physician knows which treatment was provided, thereby eliminating possible bias.

Individual definitions of the study descriptions are:

Prospective: Forward looking, beginning before the patient has started treatment.

Randomized: Patients are randomly assigned to receive the experimental treatment or alternative (e.g. standard of care or placebo)

Double-blind: Neither patients nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo or standard treatment.

Controlled: One group of patients will be given an experimental drug or treatment, while a second group is given either a standard treatment for the illness or a placebo.