Section 9.1

9.1 Novel blood components, production processes and new whole blood collection bags

Aim

To describe how a proposed novel blood component or processing method 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 UKBTS/NIBSC Professional Advisory Committee that the component should be included in the 'Red Book'

provide sufficient information to prevent all Blood Transfusion Centres (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 process qualification.

Introduction

This document identifies the steps that a group of investigators will need to undertake to submit a novel blood component for inclusion in the 'Red Book' thereby allowing it to be produced on a routine basis throughout the UK.

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 9. In this case 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 by the technical subcommittee. If sufficient data are not included then a request for extra data will be made (Step 11).

Guidance on how novel components should be tested is included in Sections 9.2–9.4.

Table 9.1 Testing of novel components
Step	Details	Information
1. Investigators identify requirement for a novel blood component.	The requirement must be derived from R & D work or as the result of clinical discussions. The blood component needs to fulfil an unmet clinical need OR provide production benefit and have a blood service proposer. Investigators will need preliminary data to support their application.	The new component may be derived from a commercially available product, in this case data to support the submission may be derived from the manufacturer. Investigators must critically appraise data already available. All data mjst be maintained on file. It will be used to demonstrate validation has been completed in support of Blood Transfusion Centre licensing activities. Data required may include clinical outcome.
2. Investigators may obtain initial advice from SACBC Chair as to whether the component should be treated as novel.	Yes:- Go to step 3. No:- Undertake local validation and produce the component locally under the general principles of GMP and the 'Red Book'.	The proposed new component may require evaluation even if it complies with existing 'Red Book' Guidelines if a new production technique is involved (e.g. leucocyte depleted red cells produced by apheresis) there are different steps in the production process (e.g. white cell filtration immediately following collection) Definitive advice about the need for full scale evaluation will be provided from the SACBC following a written submission.
Characterise the new blood component
3. Apply the SACIT Chair for a development barcode.	Allocated by SACIT.	Allows component production, discard and use to be tracked using the Blood Transfusion Centre's IT system.This will allow the evaluation to be integrated within the Centre's Quality System.
4. Investigators define the intended specification for the blood component.	Written specification to include: expected characteristics (e.g. WBC, Platelet count) testing characteristics (blood grouping, microbiology, etc.) sampling time, sampling method and sample handling conditions to confirm that the component meets specification reference should also be made to the research papers from which the specification is derived.	Specify all key points which will allow subsequent production of the component to be well controlled.
5. Write the protocol for component evaluation.	Investigators' group writes procedures for: component production monitoring of performance clinical use outcome measurement adverse incidents in production/use of the blood component or use manufacturer's documentation to priduce 'in-house' protocols.	Principles of GCP and GMP should apply. Comply with generic protocols (Sections 9.2-9.5). Laboratory studies should comply with local standards. Must include in the procedure the sampling regimes, data analysis, and expected ranges which will be used to confirm that production of the component is under control. Must include detail of the data analysis methods.
6. Investigators should ensure their protocol complies with Chapter 9 and may seek advice from SACBC.
7. Obtain ethics committee approval.		Must comply with local consenting and ethics policies for the use of donated material.
8. Investigators apply protocol.	Document evidence of protocol being implemented. Investigation should be subject to independent quality audit.	Audit may be carried out on behalf of collaborating manufacturers even though this may be confidential regarding the data collected. A summary outlining non-compliances against GCP, GMP must be made available to the Blood Transfusion Service involved for submission as part of the supporting documentation to the SACBC.
9. Reports submitted to SACBC Chair for technical subcommittee to review outcome.	Investigators review outcomes and produce a report, which summarises findings anbd supports the case for a new blood component to be listed.	Investigators who have been conducting speculative research with a manufacturer may enter the process at this point.
Obtain SACBC listing of the component
10. Investigators submit report and supporting data to the SACBC for consideration.	SACBC decide if: the blood component is novel the data support the ability to produce the blood component on a regular basis the blood component is efficacious and safe.
11. SACBC decide whether the component may be recommended for inclusion in the 'Red Book' guidelines.	If SACBC decide that the blood component will be listed, submit this recommendation to the 'Red Book' Joint Professional Advisory Committee. If SACBC decide that the blood component will not be listed, inform the submitting group and provide an explanation.	SACBC may request further data in support of the submission prior to listing the blood component.
Joint Professional Advisory Committee
12. Consider the recommendation that a new component should be listed.	Write to SACBC notifying them of the decision. If not accepted, provide SACBC with detailed reasons for the decision.
SACBC
13. Communicate the Joint Professional Advisory Committee decision to appropriate parties.	If accepted inform investigators; request SACIT to proceed with the provision of appropriate labels. Write to Medical Directors of the four UK Blood Transfusion Services. Provide copies of the data and report used to accept the new blood component. If not accepted inform investigators, with supporting reasons.
SACIT
14. Provides codes for the new blood component.	Code will be unique. ISBT 128/ABC Codabar will be supported.
15. Provides a component label.	Label will be unique.	Label text will describe the key attributes of the component.
Blood Establishments
16. Begin production of the new blood component.	Base procedures on those used during validation studies. Complete process qualification.	Demonstrates without redoing the above validation that the blood component produced is equivalent to that defined in the UK guidelines.
17. Produce the blood component routinely.	Confirm procedures.	Continue to monitor production to the 'Red Book' specification.