Why Engage with TrakCel’s CGT Consultancy?

Over a decade working with cell and gene therapy (CGT) developers, has shown that things work best when stakeholders collaborate. Whether simply sharing information to facilitate the therapy journey, or a broader collaborative approach to develop best practice. CGT has unique demands in many aspects, so investing in growing ‘collective’ knowledge will be key for industry growth. 

As a sector, variability is often highlighted, the way that processes differ to support different therapy classes or stages can be confusing. But what is often overlooked are the similarities, and that there is much that can be learned and replicated from the experience of others. Generating processes from scratch is inefficient when pre-existing processes can offer a solid foundation on which to build. 

Too much variation in the CGT space is a potential risk. Be this down to the different therapy classes, indications treated, or simply down to an individual developer’s preferences and decisions. Many stakeholders are involved in delivering each therapy and unnecessary process variation introduces unnecessary risk.  

Over the variety of orchestration projects that TrakCel’s team has worked on, those that start with best practice reap the efficiencies later on. This may seem obvious, but there are compelling reasons why this is not always possible for early-stage developers. Identifying people with the right skillsets to assist with supply chain set-up can be challenging. Additionally, for early-stage trials, the work might be project-based or a part time position. 

There are also many ‘hats’ to wear when setting up early-stage processes. Some key early considerations are chain of identity (COI), chain of custody (COC), data protection, labelling, risk assessment and validation, and these require distinct skillsets. For example, COI strategy and data protection in CGT requires process and regulatory knowledge. COC strategy and supply chain risk assessment however may require a blend of supply chain and audit skillsets. 

For early-stage clinical companies where resources are tight, hiring the right combination of skills may not be possible. Even where budget can be stretched, finding candidates with the right blend of skills and experience is time-consuming, potentially delaying projects. To assist therapy developers, TrakCel has made subject matter experts (SMEs) aligned with the above challenges available as consultants.  

TrakCel’s consultancy team span its business and are experienced in industry, technical, project management, quality and validation. The team has also worked with a wide range of therapies at different stages of development. They are available for short sessions, project work, or to operate as part of your project team.  

The team work over many areas and can assist with a range of tasks, but there are some topics they are frequently asked about. 

1. COI Strategy 

There are many examples of successful COI strategies. These can vary and some suit specific products or starting materials better than others. It is important to ensure that the strategy you deploy is compliant, can maintain COI fidelity, and is practical. There are regulatory frameworks and related regulations such as data privacy that must be met and there are also some excellent models that can be implemented such as the ICCBBA COI identifier.  

It is recommended that the COI strategies being considered are assessed with the specific therapy and the related process in mind. The strategy should be reviewed as and when new products are added, or changes are made.  

Reviewing the processes will help to outline where the risk points are located, which assets are being tracked and their associated constraints. For example, is there more than one starting material sample, is in silico data also being tracked? 

There are many areas of the supply chain that rely on interactions of partner organizations who will not be as familiar with the specifics of a drug product’s value chain. Therefore, methods for coordinating partners and assuring compliance need to be deployed.  

The last element of COI strategy to be considered is how COI data is reported retrospectively. This will be relevant during audits and potentially future competent authority applications. 

2. COC Strategy 

COC strategy is sometimes treated as secondary to, or part of, the COI strategy. However, there are good reasons to review it in depth. The regulations governing human tissue sample handling are stringent and ubiquitous, proactive COC management will help safeguard samples and compliance. 

Tissues and cells that are transported in these therapy journeys are often patient-specific, subject to demanding and costly processes. Any loss of cells or reduction in the sample’s quality due to supply chain problems can be deleterious to the subsequent drug product’s quality.  

By engaging with the right partners and technologies, COC strategy can become a pro-active part of safeguarding drug product quality.  

Equally, there may be local requirements that need to be considered, for example when and how quickly shipments can clear customs, or local working/holiday hours.  

Streamlining COC strategy to the therapy process can transform COC from being a passive, recording process to a proactive system that adds value to the therapy journey.  

3. Data Protection and Security 

Data protection for CGT supply is particularly important, especially for personalized therapies as patient data may be used extensively for COI management. Data protection in CGT is complex when considering, the different datasets processed, regional variations, and the need to share data between different stakeholders.  

The first rule of processing healthcare data is to only share data that are necessary to the task at hand. Even for non-sensitive data, sharing more than is necessary will impact how quickly users can process it. When considering sensitive or protected data, detailed planning is necessary to demonstrate that regulatory standards (e.g. HIPAA) are met.   

An audit of all systems that process PHI (Protected Health Information) should be undertaken. Processing for data protection purposes is a broad term and includes collection, recording, alteration, transmission. However, it is sometimes overlooked  by organizations that, storage, structure, restriction, or erasure are also classed as data processing.  

There are obvious areas of concern such as drug product, shipping labels and where PHI is shared between third party systems. Robust data sharing agreements are needed which reflect the local regulatory environment (e.g. the use of Business Associate Agreements in the USA) to ensure that shared data is managed correctly.  

The output of the audit should be a full data processing flow diagram including both in-house and partner processes. This should inform both the data risk assessment and the data-processing agreements with third parties.  

4. CGT Labelling 

As with the other areas considered in this article, labelling requirements are partly defined by the specific therapy and, where relevant, the associated starting material. The nature of the drug product and starting material, with the selected primary packaging will determine both the physical attributes of the label and the text used. For example, a blood bag will permit a much larger label than a sample tube.  

Another issue to consider is where a label is generated. If the label is printed at a partner or manufacturing site, their compliance with GMP labelling standards must be verified.   

There are many standards that can be leveraged to generate compliant labels, as well as established iconography. Compliance should be assessed in relation to relevant regulations e.g., Annex 13 of The Clinical Trials Directive.  

Once templates are agreed and implemented, the focus can then shift to controlling printing and tracking of labels and the data they contain.  

5. Supply Chain Risk Assessment 

The supply chain risk assessment begins with an end-to-end review. From this the supply chain is broken into units of activity, process steps and systems for which the functional requirements are detailed.  

Our consultants generally work with FMEA (Failure mode and effects analysis) methodology. This method identifies and classifies potential failure modes and their associated effects or impacts. It will also assess the prevention controls that are in place and their effectiveness.  

Once complete, the FMEA will allocate a likelihood measure and overall risk level for each element assessed. This allows the creation of an informed risk management plan that can be prioritized in terms of severity. The assessment of risk can be done by your own in-house team or through consultants. 

6. SaaS in Pharma 

The use of Software as a Service (SaaS) platforms  by pharmaceutical companies is becoming more common; SaaS can offer more flexibility and are potentially cost-effective to deploy and maintain. However, as with all software within CGT and other pharma applications, they must be appropriately validated.  

Validation will require a GXP and validation risk assessment to produce a validation plan. This validation plan will control the introduction of your SaaS solution and ensure it meets required standards.  

The validation plan is an involved process. Documenting and consolidating user requirements, vendor assessments and then relevant validation protocols and reports.  

Before implementation, you must audit  the partner that is providing and supporting the SaaS solution that you plan to implement. Whilst vendor audits are common, there are a few more things that should be considered that are specific to a SaaS provider.  

Business requirements and associated standards provide the starting point for vendor qualification. This allows the scope of the qualification to be defined and agreed. This scope will drive the assessment and provide a structure for the assessment report and resulting recommendations. 

This list is by no means definitive, but it does offer a rough guide as to how some of the common challenges encountered can be approached. If you would like to book a consultation session with any of our specialists to discuss these or other challenges in more detail, please contact enquiry@trakcel.com

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