EMA and FDA Answer Questions About Breakthrough Therapies

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The agencies published a joint guidance document regarding quality development for breakthrough therapies and PRIME programs.

On Dec. 19, 2023, FDA announced a joint Q&A document with the European Medicines Agency (EMA) that addresses questions about FDA’s breakthrough therapy (BT) designation and EMA’s priority medicines (PRIME) scheme. Specifically, the Q&A document discusses expediting quality development for these therapies that treat unmet medical needs. It describes FDA’s policy on quality assessments for expedited products in the Center for Drug Evaluation and Research’s Manual of Policies and Procedures and FDA guidance documents.

The document was developed after a 2018 workshop held by FDA and EMA where quality challenges and scientific approaches were discussed by regulators and members of industry to be sure robust quality and manufacturing data packages are submitted in a timely manner. Real case studies were explored at the workshop that showcased challenges with chemical molecules, biologicals, and advanced therapy medicinal products (ATMPs). Process validation, control strategy, compliance with current good manufacturing practice (GMP) requirements, comparability, stability, and regulatory tools were chosen as topics for discussion.

“These conversations focused on enabling timely access to medicines for patients while keeping in mind the importance of drug safety, efficacy, and quality and maintaining current regulatory standards of approval. FDA and EMA have engaged in further discussions on these topics, sharing their experiences and regulatory expectations in the context of PRIME/BT applications, and this new document is the result of these discussions,” the agency stated in a press release.


The document is broken down into questions on the following four sections:

  • Annex 1. Q&A on Control strategy considerations for PRIME/BT applications
  • Annex 2. Q&A on Process validation approaches for PRIME/BT applications
  • Annex 3. Q&A on Alternatives for determination of re-test period or shelf-life for PRIME/BT applications
  • Annex 4. Q&A on GMP considerations for PRIME/BT applications.

“These documents are only intended to provide general information and do not constitute regulatory guidance. Applicants interested in pursuing the approaches described in these Q&A documents should discuss the strategy required for their specific product with the relevant regulatory authority ahead of their marketing submission,” the Q&A document states.

Source: FDA