Orchestrating a Commercially Successful Cell and Gene Therapy
The Role of Technology in Operational Scalability
Akshay Peer, Senior Vice President, Product Development and a Co-founder of TrakCel
In this article, Dr Akshay Peer, TrakCel’s Senior Vice President Product, explores the evolving challenges of cell and gene therapy orchestration and the critical role of technology in streamlining processes, ensuring patient safety, and promoting standardization. Akshay highlights the pitfalls of custom-built solutions and key considerations for selecting the right orchestration platform.
The cell and gene therapy (CGT) landscape is rapidly evolving, offering groundbreaking treatments for previously incurable diseases. However, the complexities of CGT commercialization present unique challenges for manufacturers, researchers, and healthcare providers alike. These therapies often involve intricate supply chains, personalized treatments, and stringent regulatory requirements, necessitating a paradigm shift from traditional pharmaceutical models.
In this evolving landscape, technology plays a pivotal role in streamlining and simplifying processes, fostering collaboration, and ensuring the safe and effective administration of CGTs. Technology platforms like TrakCel are essential for navigating the complexities and accelerating the delivery of life-saving therapies to patients.
The evolving challenges of CGT orchestration
Unlike conventional drugs, CGTs often involve personalized treatments, intricate supply chains with multiple stakeholders, as well as stringent temperature and timing requirements. Each step, from a patient’s biological sample collection (starting material), and manufacturing to transport and administration, must be meticulously coordinated. This necessitates careful management of the Chain of Custody (COC) and Chain of Identity (COI) to ensure the right product reaches the right patient at the right time.
Relying on manual processes and disparate systems can lead to transcription errors, lack of standardization and poor customer experience for busy healthcare providers. Further to this, fragmented data, communication gaps, and a lack of real-time visibility can hinder efficiency.
Early developers of currently approved CAR-T products including BMS, Kite, Novartis and JNJ have chosen to create their own bespoke software solutions to support their products and pipelines. This extremely expensive and time-consuming approach has also led to a lack of standardization across the industry. Varying terminology & nomenclature, process steps and definitions, and data formats are creating confusion and delays, contributing to "system fatigue" for healthcare providers who have to navigate multiple platforms with different requirements.
This lack of standardization and the resulting system fatigue can have significant consequences:
Time: Approved treatment centre (ATC) staff at most (if not all) are working tirelessly to treat patients as quickly and efficiently as possible. The need to interact with multiple, disparate systems forces ATC staff to spend valuable time on menial tasks — remembering different logins, processes, communication protocols, and terminology — diverting attention from providing optimal patient care.
Increased training burden: Healthcare providers must learn to navigate multiple systems with varying requirements, leading to frustration and decreased efficiency.
Risk of errors: Inconsistent processes and terminology can increase the risk of miscommunication and mistakes, potentially impacting patient safety and treatment outcomes.
Difficulty with data integration: Sharing data between different systems becomes more complex when standards are not in place, hindering collaboration and analysis.
FDA expects nearly 10-20 new products per year to be approved from 2025 and if each manufacturer continues to develop their own/bespoke solutions the ATC staff will not be able to cope.
This highlights the growing need for robust orchestration platforms that can effectively manage the intricacies of the CGT supply chain, streamline workflows, and promote standardization. These platforms offer a centralized system for coordinating all stakeholders, tracking the Chain of Custody and Chain of Identity, managing logistics, and ensuring compliance with regulatory requirements. By streamlining processes and providing real-time visibility, orchestration platforms can significantly improve efficiency, reduce risks, and ultimately accelerate the delivery of life-saving therapies to patients.
The pitfalls of custom-built solutions
While the allure of customization is strong, particularly in a specialized field like cell and gene therapy, it often masks significant pitfalls. Developing a custom-built orchestration platform can seem like the ideal solution to tailor processes to specific needs. However, this approach often leads to unforeseen challenges that can hinder efficiency, strain resources, and ultimately impede the delivery of therapies to patients. Key challenges or pitfalls:
1. Initial build cost: One of the most common pitfalls is underestimating the time and costs involved in developing a bespoke solution. These projects take far longer and cost more than initially anticipated, potentially delaying critical timelines and impacting budgets. Furthermore, adapting a custom-built platform to evolving needs and regulations can be cumbersome and expensive, requiring considerable recoding and revalidation.
2. Isolated view: Furthermore, custom-built solutions often lack the voice of the user in their design. Without incorporating feedback from healthcare providers and other end-users, these systems may not adequately address their needs or streamline their workflows. This can lead to frustration, resistance to adoption, and ultimately, a less efficient process.
3. Regular Maintenance: Maintaining and supporting a custom-built platform also presents significant challenges. Companies must invest in dedicated IT resources or outsource to further develop the system, address bugs, and implement updates. This can divert funds from core competencies, such as research and development or even patient care. Moreover, as technology, or regulatory landscape evolves, custom-built solutions will become obsolete, requiring further investment to remain functional and relevant.
4. Integrations: CGT products require a network of stakeholders to work together and deliver for each patient. This network includes hospitals, community clinics, payers, 3PLs, courier providers, CDMOs, QA/QC labs and many others. Bespoke platforms require the manufacturer to invest in developing, integrating and then maintaining this ever-growing network. The cost of managing this scale-up is high but also very time consuming.
5. Product evolution: Finally, custom-built platforms will lack access to specialized features readily available in commercial solutions. Even if manufacturers utilize easily available platforms like Salesforce or similar, the rate of change or evolution required to support their needs is very slow.
Projects exceeding budgets and timelines, systems struggling to adapt to new regulations, and platforms failing to meet user needs are common occurrences. These real-world consequences highlight the need for alternative approaches that can provide greater efficiency, adaptability, and value.
Key considerations when choosing an orchestration platform
Given the complexities and challenges inherent in cell and gene therapy orchestration, selecting the right technology platform is crucial for success. Decision-makers must carefully evaluate various factors to ensure the chosen platform aligns with their specific needs and the evolving demands of the CGT landscape.
1. User-centric design and ease of use for ATCs
ATCs are at the forefront of CGT administration, and their experience with the orchestration platform significantly impacts efficiency and patient outcomes. A user-centric design, with intuitive interfaces and streamlined yet relevant workflows, is essential to minimize the training burden on ATCs and reduce the risk of errors. Addressing "portal fatigue" by providing a unified platform with consistent workflows across different therapies is also crucial.
2. Adaptability and future-proofing
The CGT landscape is constantly evolving, with new therapies, technologies, and regulations emerging regularly. Choosing an adaptable platform that can readily accommodate these changes is vital for long-term success. Configurability, modular design, and a commitment to ongoing development from the technology provider are key factors to consider.
3. Robust integration capabilities
Seamless integration with existing systems and partners is key for efficient data exchange and collaboration. The platform should readily integrate with electronic health records (EHRs), manufacturing execution systems (MES), and other critical tools used across the CGT supply chain. Open APIs and established partnerships with key vendors can facilitate smooth integration and data flow.
4. Cost-effectiveness and scalability
Cost-effectiveness is a crucial consideration, particularly for smaller companies or those with limited resources. The platform should offer a clear and transparent pricing model, with options that cater to different needs and budgets. Scalability is equally important, ensuring the platform can accommodate growing data volumes and user numbers as the company expands.
5. Strong vendor support and commitment to industry collaboration
Beyond the platform itself, the vendor's commitment to customer support and industry collaboration is essential. Ongoing maintenance, timely updates, and responsive support can ensure smooth operation and address any challenges that arise. Furthermore, a vendor who actively participates in industry initiatives and promotes standardization demonstrates a commitment to the long-term success of the CGT ecosystem.
6. Maximizing platform potential:
In addition to the core functionalities, several other critical factors can significantly enhance the value and effectiveness of an orchestration platform. These factors contribute to faster deployment, increased efficiency, and reduced costs, ultimately empowering organizations to maximize their return on investment and accelerate their CGT initiatives.
● Lower upfront costs: Platforms with lower initial investment costs can be attractive, especially for early-stage companies.
● Faster deployment: Rapid implementation can enable companies to realize the benefits of the platform quickly.
● Ensuring compliance and data security: The platform should adhere to relevant regulations and security standards to protect patient data and ensure compliance.
● Product roadmap of advanced features: A clear roadmap outlining future developments and enhancements demonstrates the vendor's commitment to innovation and meeting evolving needs.
By carefully evaluating these considerations, decision-makers can choose an orchestration platform that empowers their organization to navigate the complexities of CGT commercialization, accelerate the delivery of therapies, and ultimately improve patient outcomes.
Industry best practices: Driving standardization and collaboration
Standardization is crucial for streamlining processes, improving efficiency, and reducing errors in the complex world of CGT. Industry-wide adoption of common terminology, data formats, and best practices can significantly benefit all stakeholders. This standardization facilitates seamless data exchange between different systems and organizations, enabling better communication, collaboration, and ultimately, improved patient outcomes.
Technology providers play a vital role in driving standardization and facilitating collaboration. By developing platforms that adhere to industry standards and promote interoperability, they can empower organizations to work together seamlessly. These platforms can serve as a central hub for data exchange, enabling secure and efficient communication between manufacturers, treatment centers, and other stakeholders.
Collaboration within the CGT industry is equally crucial. Open communication and knowledge sharing between manufacturers, technology providers, and healthcare professionals can foster the development and adoption of standardized practices. This collaborative approach can lead to:
● Improved efficiency: Standardized processes and data formats can streamline workflows and reduce manual effort, freeing up resources for more critical tasks.
● Enhanced patient safety: Clear communication and consistent processes can minimize the risk of errors and improve patient safety.
● Accelerated innovation: Collaboration can foster the development of new technologies and therapies, driving progress in the CGT field.
● Developing common terminology and data formats: Creating a shared language and standardized data structures for seamless exchange between systems.
● Defining best practices: Establishing clear guidelines for patient identification, Chain of Custody/Chain of Identity management, and data security.
By embracing standardization and collaboration, the CGT industry can create a more efficient, effective, and patient-centric ecosystem.
Navigating the complexities: Choosing the right orchestration partner
As the cell and gene therapy field continues to advance, the need for robust orchestration platforms becomes increasingly critical. These platforms offer a centralized system for managing the intricate logistical and operational challenges inherent in CGT, streamlining workflows, and ensuring patient safety. However, with a growing number of providers offering orchestration solutions, selecting the right partner can be a complex decision.
The ideal platform should do more than just the basics. It needs to handle the unique challenges of CGT, such as tracking Chain of Custody and Chain of Identity, providing real-time visibility, and adapting to changing regulations. It should also be flexible enough to handle new therapies and technologies as they emerge.
User experience is key, especially for healthcare providers who use the platform daily. An intuitive interface, streamlined workflows, and minimal training needs are essential for efficiency and reducing errors. The platform should also easily integrate with existing systems like electronic health records (EHRs) and manufacturing systems (MES) to allow for smooth data sharing and collaboration.
Cost-effectiveness and scalability are also important. The platform should have a clear pricing structure and be able to handle increasing amounts of data and users as the organization grows. Strong vendor support, including maintenance, updates, and responsive customer service, is also essential.
Finally, the vendor should be committed to working with others in the industry and promoting standardization. A partner who actively participates in developing and adopting best practices shows they are dedicated to the long-term success of CGT and improving patient outcomes.
Careful consideration of these factors will allow organizations to choose an orchestration partner that helps them navigate the complexities of CGT, speed up the delivery of life-saving therapies, and contribute to this transformative field. The future of CGT depends on working together, innovating, and putting patients first. By embracing these principles and choosing the right orchestration platform, the industry can unlock the full potential of CGTs and change healthcare for the better. This will pave the way for a future where these groundbreaking therapies are more accessible and ultimately more successful in transforming patient lives.
Author Bio
Dr Akshay Peer is the Senior Vice President of Product Development and a Co-founder of TrakCel. He has over 12 years of extensive experience within the cell and gene therapy industry. His longstanding tenure in this field reflects a dedicated commitment to advancing innovation and contributing to the industry's growth.
