On July 31, 2019, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) published a joint report on their efforts to support the development of and expedite access to breakthrough therapies that address unmet medical needs.  In addition to outlining the tools currently available to manufacturers seeking expedited approval of breakthrough therapies, the report recognizes the need for further regulatory innovation to advance emerging scientific knowledge and support timely access to new medicines without compromising established quality and manufacturing standards.

To obtain marketing approval for breakthrough therapies, sponsors face challenges to obtain clinical, non-clinical, and manufacturing information that meets the standards for regulatory approval on compressed timelines.  FDA/EMA recognize this, and on November 26, 2018 held a workshop on FDA’s Breakthrough Therapy Designation (BTD) and EMA’s Priority Medicines (PRIME) programs.  The aim of the workshop was to facilitate discussion with industry stakeholders on challenges associated with quality, manufacturing, and data requirements during early development, and possible scientific and regulatory approaches to address them.

With regard to challenges related to quality and manufacturing data, the report provides FDA/EMA’s perspectives and outlines follow-up initiatives being considered for process validation and control strategy, current good manufacturing practices (cGMP) compliance, comparability and stability data, and regulatory tools to support early access.  The key takeaways relating to quality and manufacturing data are provided below: