Assessing clinical data as part of PPA

Objectives
WP 8 aims at:

• defining the current state of the art of existing collected clinical data, namely in order to define a catalogue of clinical data, appropriate to provide information on the quality and safety of human blood, cell, and tissue therapeutics once applied to patients, under the conditions of current state-of-the-art processing and testing protocols for human blood, cell, and tissue therapeutics

• defining a risk-based framework to assess whether the current state-of-the-art criteria fit to a new processing or testing protocol. Such criteria should be appropriate to evaluate the established catalogue of clinical data for completeness and suitability in case of introduction of innovation to the current processing and testing protocols for human blood, cell, and tissue therapeutics

• defining a methodological framework, to evaluate quality and safety based on clinical outcome data requested for authorization processes upon introduction of innovation to the current processing and testing protocols. The framework should have adequate characteristics in order to allow for an appropriate degree of harmonization as well as for an appropriate degree of flexibility, to evaluate quality and safety of human blood, cell, and tissue therapeutics based on clinical outcome data requested for authorization processes upon introduction of innovation to the current processing and testing protocols for human blood, cell, and tissue therapeutics

• Building a data model of information on clinical outcome of application of human blood, cell, and tissue therapeutics, building also bridges to existing clinical databases such as ECCTR, EBMT, EuroGTPs, registries.

Description of work and role of partners
This WP will be co-lead by FIMEA and BST Part A (1) Specification of a set of existing clinical data appropriate to provide information on the quality and safety of human blood, cell, and tissue therapeutics once applied to patients, under the conditions of current state-of-the-art processing and testing protocols (6 months) (2) Specification of appropriate risk-assessment models taking into account cumulative and aggregative risk assessment (1.5 months) (3) Specification of criteria, appropriate to define “innovation” in processing and testing protocols for human blood, cell, and tissue therapeutics taking into account the output of work package 5b of VISTART (1.5 months) (4) Specification of a risk-based set of criteria, appropriate to evaluate the established catalogue of clinical data for completeness and suitability in case of introduction of innovation to the current processing and testing protocols for human blood, cell, and tissue therapeutics (6 months) (5) Review of data set by experts and stakeholders for completeness and relevance (3 months) Part B) (6) Setting up tools for data compilation (2 months) (7) Proof of concept: data acquisition, evaluation of quality and validity of data ( 12 months) (8) Definition of a methodological framework, appropriate to allow for an appropriate degree of harmonization as well as for an appropriate degree of flexibility, to evaluate quality and safety of human blood, cell, and tissue therapeutics based on clinical outcome data requested for authorization processes upon introduction of innovation to the current processing and testing protocols for human blood, cell, and tissue therapeutics

Expected Deliverables
D8.1: Catalogue of existing clinical data appropriate to provide information on the quality and safety of human blood, cell, and tissue therapeutics once applied to patients, under the conditions of current state-of-the-art manufacturing and testing protocols
D8.2: Catalogue of risk-based set of criteria, appropriate to evaluate the established catalogue of clinical data for completeness and suitability in case of introduction of innovation to the current manufacturing and testing protocols for human blood, cell,
D8.3: Methodological framework to evaluate quality and safety of human blood, cell, and tissue therapeutics based on clinical outcome data requested for authorization processes upon introduction of innovation to the current manufacturing and testing protocols for human blood, cell, and tissue therapeutics
D8.4: Data model of information on clinical outcome of application of human blood, cell, and tissue therapeutics

WP8 Leaders: Laakealan Turvallisuus-JA Kehittamiskeskuss (FIMEA), Finland - Banc de sang i teixits (BST), Spain