The importance of CRF design in cell and gene therapy studies
Case study
Overview
The success of cell and gene therapies (CGT) has transformed the delivery of clinical trial services, requiring the design of new workflows, processes and tools to address the vast differences in how CGT trials are executed compared to traditional trials.
The challenge
ICON’s cell and gene therapy experts in biostatistics were engaged by a biotech sponsor to support the CRF design of an oncology cell therapy study.
The sponsor sought guidance on the selection of the optimal immune response criteria and standardisation of data collection for cell therapy toxicity. After consulting with our cell therapy and immuno-oncology experts, the sponsor opted to adopt the iRECIST (Immunotherapy Response Evaluation Criteria in Solid Tumors) criteria. Recording data for multiple response criteria presents challenges particularly in minimising data duplication – while the RECIST v1.1 and iRECIST assessments essentially mirror each other, divergences arise at RECIST v1.1 progressive disease juncture. By utilising our standardised iRECIST forms and cell therapy toxicity CRFs, the team was able to minimise data duplication, reassure the sponsor and build confidence in this approach.
Furthermore, the protocol included specific criteria permitting re-treatment with the cell therapy product.
The solutions
CRF standardisation
ICON determined that our standard Case Report Forms (CRFs) could be utilised for two response criteria, RECIST and iRECIST, covering target lesions, non-target lesions, new lesions, and disease response assessments.
Therapeutic expertise
ICON also involved subject matter experts (SMEs) in cell therapy, leveraging their extensive experience across various scenarios and therapy types, to provide guidance on the proper collection of cell therapy- related toxicities.
Flexibility and adaptability
ICON demonstrated flexibility by presenting multiple solutions, enabling the sponsor to select the option that best aligned with their study requirements, budget, and timelines. The sponsor was eager to re-treat subjects immediately, so the team utilised the existing CRF structure to allow investigators to re-treat subjects within the required timeframe, thereby avoiding a more costly and time-consuming database migration.
The outcomes
Successful data collection
To date, successful data collection on re-treated subjects has been accomplished, with ICON advising the sponsor on iRECIST response criteria, cell therapy toxicity scenarios, and their effective implementation.
Efficiency and consistency
ICON continues to maintain multiple CRF templates for oncology response criteria and cell and gene therapy. As a testament to this approach, subsequent studies now engage ICON’s cross-functional cell and gene therapy SMEs from the outset of CRF design.
Risk mitigation, data integrity and timeline adherence
Feedback from this and other sponsors highlights the value of ICON’s CRF designs in cell and gene therapies. ICON’s team of experts, along with its thoroughly vetted templates, enable sponsors to meet CRF development timelines while minimising the risk of critical data omissions.
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