Spring Intro 2023

30/03/2023

What and Why ?

To obtain regulatory authority input on the most appropriate way to generate robust evidence on a medicine’s benefits and risks • e.g. Quality; Non-clinical; Clinical; Methodology; Overall Development Strategy Why? • Better designed studies will generate more robust and complete data • Where regulatory guidance is not available/out of date • Where it is expected the company will deviate from regulatory guidance • Where you have limited knowledge of medicine regulations (small companies) Examples • Quality – manufacturing; testing • Non-clinical - toxicology program • Clinical - choice of endpoints; patient population; comparators • Methodology - stats; data analysis; modelling • Overall Development Strategy - conditional MA; safety database; significant benefit

The Organisation for Professionals in Regulatory Affairs

29

Key points to remember

All forms of scientific advice are NOT legally binding on either the applicant or the regulatory authority

But… • Advice needs to be detailed in Module 1.2 of MAA • Transparency throughout MAA procedure

The Organisation for Professionals in Regulatory Affairs

30