EMA and FDA publish Q&A Document on their accelerated Approval Programmes

Recommendation

7/8 May 2024

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Regulatory Requirements and Practical Implementation

For some time now, the regulatory authorities in Europe and the USA have been trying to support the development of innovative medicines and therapies and have set up corresponding programmes for this purpose. In Europe, the EMA offers the "PRIME: priority medicines" programme to promote the development of medicines for which there is an unmet medical need. It offers the opportunity for increased interaction and early dialogue with the developers of promising medicines in order to optimise development plans and accelerate evaluation so that these medicines can reach patients sooner.

The equivalent in the US is Breakthrough Therapy, a process designed to accelerate the development and testing of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may represent a significant improvement over an available therapy on one or more clinically important endpoints.

However, in the context of such accelerated development programmes, the compilation of chemistry, manufacturing and controls (CMC) information within shortened timelines can be a challenge for applicants. Both agencies have therefore engaged with stakeholders to explore approaches to accelerate the development and authorisation of these products without lowering the quality standards expected of medicines.

The Q&A paper

On 4 December, the EMA and the U.S. FDA published a Q&A paper outlining their current thinking on various quality and GMP aspects related to PRIME/Breakthrough Therapy applications.
Back in 2018, the EMA and FDA organised a stakeholder workshop on quality development for early access approaches such as PRIME and Breakthrough Therapies and published a meeting report. In addition, the EMA developed and published a "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".

In the subsequent period, the EMA and the FDA's Centre for Drugs Evaluation and Research (CDER) held further discussions on these topics and exchanged their experiences and regulatory expectations in connection with PRIME/BT applications. As a result of these discussions, a Q&A document has now been published.

In addition to an explanatory introduction on the background and development of the document and a glossary, the document contains 4 Q&A sections covering different areas:

• 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

However, it should be noted that the document is for general information only and does not constitute regulatory guidance. Therefore, applicants interested in PRIME or Breakthrough Therapy approaches should always discuss the strategy required for their specific product with the competent regulatory authority before discussing their application for marketing authorisation.

Further information can be found directly in the published document "EMA-FDA joint Q&As on Quality and GMP aspects of PRIME/Breakthrough therapy applications".