This white paper celebrates the 50th Anniversary of the Kefauver-Harris Amendments to the Food, Drug, and Cosmetic Act, which were signed into law by President John F. Kennedy on 10th October, 1962. Since then, the well-conducted RCT has become the gold standard for generating evidence of efficacy. The Amendments can be meaningfully regarded as a powerful engine that has driven and continues to drive development of the statistical and ethical sciences that underpin the RCT. The paper therefore reflects on the role that randomized clinical trials have played in drug development across the last half century, and also discusses developments that will impact clinical trial design and operational execution in the coming years. It is hoped that the paper will be of interest to all individuals interested and involved in integrated biopharmaceutical medicine, including everyone involved in lifecycle drug development and also health professionals who prescribe, dispense, and administer pharmaceutical medicines to patients.
White Paper January 15, 2013