November 1, 2021

Patient data – why it is critical for cell and gene therapy stakeholders

The first blog post in this series provided an overview of how the advanced therapies supply chain is different from traditional and biologic drug products in respect to patient data. This second installment will explore in more detail why patient/donor data  is critical in the cell therapy and gene therapy (CGT)  supply chain — and overall value chain —and provide an overview of how it is used by just some of the key stakeholders.

The criticality of patient and/or donor data applies both in a micro, individual patient and hour-by-hour aspect, and at a macro level. Unlike other therapies, patient data is captured and utilized by all stakeholders in the value chain from patient identification and manufacturing to treatment, reimbursement, and long-term follow up. Here are just a few of the critical stakeholders, and how they rely on this mission-critical data.

Title: Patient data drives the cell and gene therapy value chain
Subtitle: Unlike traditional therapeutics, all cell and gene therapy stakeholders from end-to-end require patient/donor data to drive workflows and ensure patient safety.

Healthcare Providers: as the frontline contact with patients, healthcare providers (HCPs), rely on patient and/or donor data to understand the participants’ health. Patient health and biometric information will be used to determine if a patient is the right fit for a therapy and if a donor/patient is a suitable provider for the critical raw material. 

Manufacturers: Patient and/or donor data are critical for continuous, real-time management of patient and product journeys. The workflows for CGTs are complex and time sensitive with many steps that must all happen in a synchronized way and be continuously monitored. Key information at each point is needed to complete each step, provide real-time transparency and status for stakeholders, and track the patient and product journeys to ensure delivery of a safe and efficacious treatment in time for the patient to receive it.

Title: Patient/donor-related data along the CGT supply chain
Subtitle: Each point in the supply chain has key pieces of data that must be captured and immutably linked to the patient/donor.

In addition, new therapies are not yet always well-understood or characterized and the development process is more iterative than established therapy types. Data for individual patients and in totality is needed to develop patient, product, and process knowledge — and this is an ongoing, continuous effort from the earliest stages of development and clinical trials, continuing after commercialization. And more knowledge will sometimes need to be gained with fewer data points and fewer patients in situations such as rare diseases, small patient populations, and accelerated regulatory pathways. To understand the detail and complexity of data required, see the overview in the graphic titled “Data specific to advanced therapies typically includes...” Collecting, understanding and utilizing all of this data helps companies reduce risk and accelerate the development of CGT products.

Title: Data that is specific to CTGs differs from traditional drug products
Subtitle: Data for individual patients/donors is generated along the entire process and is needed on an individual patient/donor level and in aggregate to develop patient/donor, product, and process knowledge.
Title: Improving patient, product, and process knowledge with patient/donor data
Subtitle: Data captured along the end-to-end supply chain — tied together with a solid COI — enables the ability to analyze and gain critical insights into aspects of CMC that can be used for improving the patient and product journey and regulatory filings.
[Reference 1]

Regulators: The evolving nature of the sector and regulatory requirements have increased the data demands on CGT developers. The developers are racing to understand and improve the products, with the ultimate goal of bringing safe and effective products to market quickly. Regulators have established strict traceability requirements, are requiring robust Chemistry, Manufacturing, and Controls (CMC) packages to ensure efficacy and safety, and are requesting that companies establish long-term follow-up activities such as patient registries and confirmatory trials.2

Title: Chemistry, Manufacturing and Controls (CMC) has heightened importance in CGTs
Subtitle: The nature of CGT products makes demonstrating a safe, consistent, high-quality product difficult, especially with limited data. Patient/donor data — individually and in aggregate — is foundational to developing a robust CMC package.
[Reference 3]

Patient and donor-related data is woven throughout the CGT value chain and is critical to all stakeholders — on an individual, hour-by-hour basis and in aggregate.

With these data requirements established, the third blog post in this series takes a closer, more detailed, look at the drivers behind the importance of patient/donor data and the important ways it is used in the value chain.

Blog #1 – Patient data – the unique role in cell and gene therapies (CGTs)
Blog #3 –  Patient data – unique challenges, critical uses in the cell therapy value chain
Blog #4 – Integrated patient data – the future of advanced therapies


  1. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research. Chemistry, Manufacturing, and Controls (CMC) Changes to an Approved Application: Certain Biological Products: Guidance for Industry. Silver Spring, MD. The FDA. June 2021.:
  2. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research. Long Term Follow-Up After Administration of Human Gene Therapy Products: Guidance for Industry. Silver Spring, MD. The FDA. January 2020.:
  3. 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.: