November 1, 2021

Patient data – the unique role in cell and gene therapies (CGTs)

Posted in Cell and Gene Therapy by Vineti

Patient data is mission-critical data — especially in cell and gene therapy.

In cell and gene therapy (CGT), the patient is the process is the product. Patient and/or donor data is an essential part of manufacturing and delivery, in unique and complex ways. It is also fundamental to patient safety and product and process knowledge, providing the opportunity to reduce risk and accelerate development.

This unique reliance on patient-related data sets the sector apart from traditional drug products and other biologics, creating a new supply chain paradigm, and heightening the importance of data for drug product production, the development process, and long-term follow up. This four-part blog series will explore how the CGT supply chain is different from that of traditional drug products, why patient data is critical, how patient data is used, and why integrated systems are key to the sector’s future success.

**Title:** The FDA and EMA encourage modern, real-time data management for patient safety
**Subtitle:**
Real-time access to patient data during clinical trials is important for all drug products, and even more so for CGTs. The patient and donor data remains critical many years after products are approved and patients are treated.

For traditional and non-CGT biologic drug products, the supply chain is not aware of the individual patient, or individual patient data, up through the end of production and even much later in the process. The production can happen independently of selling and distribution, and the final patients. Large batches of drug product are made from GMP suppliers and in GMP facilities, stored, then distributed in bulk with no particular patient in mind until the healthcare provider or pharmacy interacts.

The opposite is true with advanced therapies. The supply chain is patient and/or donor-centric every step of the way, often with a dose of drug product being manufactured for one single, specific patient from the patient or a single donor’s live cells.

**Title:** The patient-centric autologous CGT process
**Subtitle:** Patient data is generated at every step of the complex process

Including the patient/donor in the process and the supply chain creates a more complex and interactive supply chain with more variables and risks (patient health issues, donor identification, cell collection challenges, logistics and manufacturing issues, non-GMP stakeholders). The process is also very time-sensitive due to the urgency to treat high-need patient populations and the constraints of working with live cells.

**Title:** Four critical risks for all CGTs
**Subtitle:** CGTs have inherent risks because the process includes the donors/patients and the product includes live human cells.

This paradigm shift in the supply chain changes not only the nature of the data, but how integral data — especially patient and donor data — is to every workflow. Data related to each donor/patient must be threaded through a wide network of internal and external stakeholders. Processes generate an extremely high volume of data, per patient and in aggregate, for a low number of patients. This data is variable in nature and is spread across multiple stakeholders and systems. Additionally, each participant in the value chain has touch points for individual patients and needs to collaborate with other stakeholders. Many stakeholders will benefit from a comprehensive individual patient view and an aggregate view by therapy or disease.

**Title:** Patient data drives the cell and gene therapy value chain
**Subtitle:** Patient and donor data flows through every part of the interdependent CGT value chain. All stakeholders rely on patient/donor data for multiple purposes.

The new supply chain and data paradigm of CGTs fundamentally changes how the supply chain operates and the role of data within the supply chain, especially patient data.

Now that a high level overview of the unique role of patient data in the advanced therapies supply chain has been provided, the next blog post in this series will provide a deeper understanding of how patient data is used and why it is so critical.

Blog #2 – Patient data – why it is critical for cell and gene therapy stakeholders
Blog #3 – Patient data – unique challenges, critical uses in the cell therapy value chain
Blog #4 – Integrated patient data – the future of advanced therapies

References

U.S. Department of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research. Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs): Guidance for Industry. Silver Spring, MD. The FDA. January 2020.: https://www.fda.gov/media/113760/download

US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) and Oncology Center of Excellence (OCE). Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics: Draft Guidance for Industry. Silver Spring, MD. The FDA. August 2018.: https://www.fda.gov/media/115172/download

European Commission, EudraLex. The Rules Governing Medicinal Products in the European Union, Volume 4, Good Manufacturing Practice: Guidelines on Good Manufacturing Practice Specific to Advanced Therapy Medicinal Products. London, UK. The European Commission. November 2017: https://ec.europa.eu/health/sites/default/files/files/eudralex/vol-4/2017_11_22_guidelines_gmp_for_atmps.pdf