Q&A Document Released | PRIME/ Breakthrough Therapy Applications

January 30, 2024

The EMA and the US FDA have released a Q&A document about Quality and GMP aspects linked to PRIME/Breakthrough Therapy Designation.

Both agencies have have programmes, PRIME and Breakthrough Therapy Designation, respectively, that are aimed at expediting the development of innovative medicines that address unmet medical needs.

Even with expedited processes, these programmes require comprehensive clinical, non-clinical, and chemistry, manufacturing, and control (CMC) information to meet approval standards.

  • Challenge: Addressing the challenges of generating CMC information on compressed timelines, EMA and FDA have engaged with industry stakeholders.
  • Solution: On November 26, 2018, they held a workshop focusing on quality development in early access approaches.

The workshop discussed scientific and regulatory strategies to ensure that expedited product development maintains high standards of safety, efficacy, and quality.

Real case studies and panel discussions highlighted challenges and potential solutions, covering areas like process validation, control strategy, GMP compliance, comparability, stability, and regulatory tools.

The workshop concluded with a consensus on certain areas needing further discussion. FDA and EMA agreed on the potential for post-approval data submission to manage some risks, as detailed in ICH Q12. This includes:

  • Post-approval change management protocols.
  • CMC commitments for post-approval data collection.


Following the workshop and continued discussions, EMA and FDA/CDER prepared four Q&A documents to compile their current thinking on these topics:

  • Q&A on Control strategy considerations for PRIME/BT applications
  • Q&A on Process validation approaches for PRIME/BT applications
  • Q&A on Alternatives for determination of re-test period or shelf-life for PRIME/BT applications
  • Q&A on GMP considerations for PRIME/BT applications


They reflect the consensus from existing guidance documents and are presented as annexes to the workshop report.

  • Applicability: For EMA, the Q&As apply to chemical and biological medicinal products, including complex biologicals like ATMPs, unless stated otherwise. For FDA, the discussions and annexes apply only to CDER-regulated products, excluding CBER-regulated products such as ATMPs.
  • Regulatory Guidance: The documents are informational and do not constitute formal regulatory guidance. Applicants are encouraged to discuss their specific product strategies with the relevant regulatory authority before marketing submission.


Contact us to schedule a meeting to discuss how this new document may affect your company: [email protected]

You can find the complete document in the following link: EMA-FDA joint Q&A document

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