Trial Basics
Introducing Courageous Parents Network's new unit EVALUATING THE CLINICAL TRIAL OPTION
EVALUATING THE CLINICAL TRIAL OPTION is an educational unit for families and medical providers caring for children living with rare disease, that presents a clear-eyed picture of the process, through the lens of families, researchers and professionals. The goal is to empower decision-makers with information and perspective to help them consider clinical trial participation.
1 / 19
Introducing Courageous Parents Network's new unit EVALUATING THE CLINICAL TRIAL OPTION
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
Everyone who participates in rare disease research is a medical hero
Clinical Trials: Phase 1 defined – focusing on safety, with less certainty about benefit. Phase 1 trials for rare disease are designed for some efficacy
Clinical Trials: The Role of the Principal Investigator, Sponsor, IRB and FDA.
Clinical Trials Sponsors, Internal Review Boards
Clinical Trials: Staggered enrollment, Cohorts and Dose Escalation
Clinical Trials: The logistics and limitations of gene therapy
Clinical Trials: A mom: “Eventually we are going to be looking at combination therapies
Clinical Trials are designed to get to FDA approval ASAP
Clinical Trials: A mom: We can’t ignore the economics.
Clinical Trials: A mom on dose escalation studies and gene therapy
The role of a Patient Advocate at a biotech company
A Patient Advocate explains the Placebo controlled trial.
Clinical Trials: With Gene Therapy, it is ONE and DONE
Clinical Trials: Dose escalation, Phase by Phase
Compassionate use is no substitute for the trial pathway
Clinical Trials: Defining Compassionate Use, Expanded Access