CRED Understanding Clinical Development 2024

the grounds that the product is unlikely to be of significant therapeutic benefit or to fulfil a therapeutic need in this paediatric population. The PIP is under review. Answer the following questions based on the information you have been given and the knowledge you have gained from presentations over the previous 2 days. Question 3 Would you advise the Project Team to seek Regulatory Agency input on the Phase 3 programme? At what point should this occur? What topics should be discussed? Question 4 Describe the design considerations for the Phase 3 study for Devostatin and the rationale for your recommendations to the project team, with any comments as appropriate. Would you consider one or more Phase 3 studies? What sources of information would you consult as you consider the Phase 3 programme?

Parameter

Answer

Rationale/Comments

Main study objectives Type of study Inclusion of Comparator (& choice) Study duration/length of time safety data collected Age groups Study population & Key inclusion/exclusion criteria Concomitant medicines (allowed or not allowed) Endpoints of interest (Primary, Secondary, other) Other clinical parameters of interest including safety monitoring Size of study arms Number of studies Any other considerations?

4