Toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME and certain marketing authorisation applications targeting an unmet medical need - Scientific guideline

This guidance summarises scientific and regulatory approaches that developers of medicines supported by EMA’s PRIME scheme can use to generate robust quality data packages for an EU marketing authorisation application, to enable patients to benefit from these therapies as early as possible.

It aims to address common challenges with meeting quality and manufacturing development data requirements during development and at the time of marketing authorisation application.

It covers medicines containing chemical, biological or biotechnologically derived substances and advanced therapy medicinal products (ATMPs).

Keywords: Priority Medicines (PRIME), quality development, Module 3, data, scientific elements, regulatory tools, flexibility, benefit-risk

Current effective version

Toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME and certain marketing authorisation applications targeting an unmet medical need

Adopted Reference Number: EMA/CHMP/BWP/QWP/IWG/694114/2019

Document history

Overview of comments received on 'Draft Toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME marketing authorisation applications' (EMA/CHMP/BWP/QWP/IWG/694114/2019)

Reference Number: EMA/CHMP/BWP/QWP/IWG/579239/2021

Draft toolbox guidance on scientific elements and regulatory tools to support quality data packages for PRIME marketing authorisation applications

Draft: consultation closed Consultation dates: 02/02/2021 to 31/07/2021 Reference Number: EMA/CHMP/BWP/QWP/IWG/694114/2019 Summary:

This guidance summarises scientific and regulatory approaches that developers of medicines supported by EMA’s PRIME scheme can use to generate robust quality data for an EU marketing authorisation application, to enable patients to benefit from these therapies as early as possible.

It aims to address common challenges with meeting quality and manufacturing development data requirements during development and at the time of marketing authorisation application.

It covers medicines containing chemical, biological or biotechnologically derived substances and advanced therapy medicinal products (ATMPs).

The completed comments form should be sent to [e-mail].