Objective: to outline the different steps of therapy development from idea, to proof of concept studies in model systems, to preclinical optimization studies, clinical trials, drug approval and post marketing surveillance studies
Objective: outline different models in preclinical research, their opportunities and limitations, and the need for standardized tests and the TREAT-NMD advisory committee for therapeutics (TACT)
Objective: explain the centralized system, how it is organized and how regulators decide whether drugs are eligible for marketing authorization, outline patient involvement in committees – focus on rare diseases
Objective: learning to have a critical look at preclinical research. In this mock session participants will have been provided with a fictitious TACT application from a company planning a clinical trial in the NMD space. Participants will be split into groups of 8-10 participants to discuss the strengths and limitations and outstanding questions of the application.