ASTM E3443 : 24

1.1 The intent of this guide is to discuss and provide recommendations for reducing the volume requirements for the stability assessment of low yield biopharmaceutical products.

1.2 The recommendations include considerations for both drug substance (DS) and drug product (DP).

1.3 The scope of this guide may be applicable to any modality that has yield constraints or small batch size, however the example provided is specific for rAAV gene therapies.

1.4 The need for specific recommendations for low yield biopharmaceutical products has been identified by observing that stability guidance (for example, ICH Q1 and ICH Q5) are mainly designed for large-scale biologic/small molecule products and are not fully applicable to the unique specificities of low yield products (for example, gene therapy). The recommendations focus on strategies for routine stability studies. Development and supporting studies such as freeze/thaw stability are excluded from the scope of this guide.

1.5 Before application to specific products, companies should ensure internal alignment with the proposed approach, including a documented scientific rationale, and discuss as appropriate with applicable regulatory agencies.

1.6 Units—The values stated in SI units are to be regarded as standard. No other units of measurement are included in this standard.

1.7 This standard does not purport to address all of the safety concerns, if any, associated with its use. It is the responsibility of the user of this standard to establish appropriate safety, health, and environmental practices and determine the applicability of regulatory limitations prior to use.

1.8 This international standard was developed in accordance with internationally recognized principles on standardization established in the Decision on Principles for the Development of International Standards, Guides and Recommendations issued by the World Trade Organization Technical Barriers to Trade (TBT) Committee.