We are excited to announce that the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have collaboratively released a comprehensive Q&A document, detailing their perspectives on various Quality and Good Manufacturing Practice (GMP) aspects associated with PRIME/Breakthrough therapy applications.

The PRIME initiative by EMA and the breakthrough therapy designation program by FDA are tailored to accelerate the development of innovative medicinal products that target unmet medical needs. Given the expedited nature of these development programs, there are inherent challenges for applicants in generating Chemistry, Manufacturing, and Controls (CMC) information within shortened timelines. Recognizing this, EMA and FDA have actively engaged with stakeholders to explore strategies to expedite the development and approval processes without compromising the high quality standards expected in pharmaceuticals.

On November 26, 2018, EMA and FDA jointly organized a stakeholder workshop focused on quality development in early access approaches such as PRIME and Breakthrough Therapies. Subsequently, they published a detailed meeting report documenting the outcomes of the workshop. Additionally, EMA has developed and issued a Toolbox guidance outlining scientific elements and regulatory tools aimed at supporting the quality data packages for PRIME and specific marketing authorization applications addressing unmet medical needs.

Since the initial workshop in 2018, EMA and FDA’s Center for Drugs Evaluation and Research (CDER) have continued their collaborative efforts, engaging in further discussions, and sharing experiences and regulatory expectations pertaining to PRIME/BT applications. As a culmination of these discussions, the joint Q&A document has been released.

Applicants interested in pursuing the strategies outlined in these Q&A documents are encouraged to initiate discussions with the relevant regulatory authority to tailor their approach according to the specific characteristics of their product prior to marketing submission.1

Access the report here.

References

  1. U.S. Food and Drug Administration & European Medicines Agency. (2023). EMA-FDA joint Q&As on Quality and GMP aspects of PRIME/Breakthrough therapy applications. In ema.europe.au. https://www.ema.europa.eu/en/documents/other/ema-fda-joint-qas-quality-and-gmp-aspects-prime-breakthrough-therapy-applications_en.pdf

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