Aims and introduction - Evaluation of novel blood components, production processes and blood packs: generic protocols - Chapter 8
This chapter aims to describe how a proposed novel blood component, production process or blood pack is to be evaluated to:
- gain sufficient data to validate the component and production method
- gain sufficient data to support the clinical use of the component
- allow the Standing Advisory Committee on Blood Components (SACBC) to recommend to the Joint UK Blood Transfusion and Tissue Transplantation Services Professional Advisory Committee (JPAC) that the component should be included in the Red Book, either within the ‘Specifications for blood components‘ section or as a Provisional Component specification in the ‘Provisional Components’ section
- provide sufficient information to prevent all Blood Establishments (other than those performing a full evaluation) from having to complete a full validation of the novel component before it enters routine production. They will only need to undertake installation and process validation.
The chapter starts by identifying the steps that a group of investigators will need to undertake to submit a novel blood component for inclusion in the Red Book (see Table 8.1a), thereby allowing it to be produced on a routine basis throughout the UK. Guidance on assessing the degree of novelty of components prior to embarking on the process is given in Table 8.1b.
It is recognised that some novel components may be developed by a group of investigators in conjunction with a commercial company undertaking speculative research. As a result, the group of investigators may wish to enter the process at Step 8. In this case the SACBC will expect any requirements for data collection in the preceding steps to be complied with when the protocols and reports are submitted to the SACBC Chair for consideration. If sufficient data are not included then a request for extra data will be made (Step 9).
It is also recognised that there may be a need for Blood Establishments to produce blood components for clinical use on a temporary basis. This may be to undertake a clinical study or operational assessment of a new component in order to inform the decision as to whether there is a need to manufacture the component on an ongoing basis. In such circumstances the most appropriate course of action is to seek approval for a Provisional Component specification (see section 8.1.1 and Table 8.1c).
Guidance on how specific novel components should be tested is included in sections 8.2–8.5, and is followed by information on generic protocols for the evaluation of apheresis equipment (section 8.6) and blood packs (section 8.7). For guidance on phases of validation and sample size, please refer to Table 8.1d
8.1.1: Provisional Component specification
This process should be used where it is uncertain whether there will be a requirement to produce the novel component on an ongoing basis, yet there is a need for clinical use of the component. Provisional Component specifications once approved will be posted in the ’Provisional Components’ section of the Red Book.
The purpose of approval of a provisional component specification is to:
- ensure that there is sufficient data to support progression from phase 0 to phase 1 & 2 studies and the clinical use of the component.
- document a draft specification for the component including suitable quality monitoring parameters and testing regime for phase 1 and 2 studies.