Terence Flotte, MD
Clinical Trials: In the early phase, it’s about both gaining knowledge and trying to help. To figure it out, we need some families to step up and go first
In this video a Principal Investigator explains that clinical trials are about both research and therapy; their goal is to gain knowledge and help the participants. Trials are a process and at each step, researchers learn things. He stresses research needs families to step up and agree to be in the early phases; those families are trailblazers. He acknowledges that when trial outcomes are not met, participants often feel like the trial is a failure but stresses that at each step scientific knowledge is gained.