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