Orchestrating cell and gene therapies

The landscape of medicine is undergoing a profound transformation, with advanced therapeutics like cell and gene therapies (CGTs) leading this revolution. These innovative treatments hold the promise of curing previously untreatable diseases, offering hope for patients and health care providers worldwide. However, the intricacies associated with CGT commercialization present unique challenges that require a departure from traditional pharmaceutical models.

Technology plays a pivotal role in facilitating patient access to CGTs. An effective orchestration platform is such a technology that is now essential in optimizing processes, fostering collaboration, and ensuring the safe and effective administration of CGTs. These orchestration platforms are crucial tools for approved treatment center (ATC) staff to navigate the complex processes involved in CGT ordering, scheduling, delivery and infusion of lifesaving therapies to patients. While custom-built orchestration platforms are common, fostering standardization is proving to be a more desirable and ATC-friendly approach.

The evolving challenges of CGT orchestration

The personalized nature of these therapies necessitates intricate supply chains with multiple stakeholders, and stringent temperature and timing requirements. From patient enrollment and scheduling to the collection of starting material from each patient through to manufacture, transport and administration, every step in the process must be meticulously coordinated with careful management of the chain of custody and chain of identity to ensure timely delivery of each CGT product to the correct patient. Failure to establish robust processes is not only costly but also almost impossible to achieve at scale without the use of technology.

Manual processes that can hinge on disparate systems used by stakeholders across the supply chain can lead to duplication of effort, transcription errors, lack of standardization, and poor customer experience for busy ATC staff. Further to this, fragmented data, communication gaps, and a lack of real-time visibility can hinder efficiency.

One approach taken by early developers of currently approved CAR-T products including BMS, Kite, Novartis and J&J was to create their own bespoke software solutions to support the supply chain for their products. But bespoke solutions have contributed to a lack of standardization across the industry. Differences in nomenclature, process steps, definitions, and data formats across various orchestration platforms are causing confusion, additional workload and delays to patient treatment. This has been given the name ‘portal fatigue’ by ATC staff, who must navigate multiple platforms with distinct requirements, contributing to increased burden and inefficiency in health care delivery.

The hidden costs of bespoke platforms

User experience

In the specialized field of cell and gene therapy, developing a custom-built orchestration platform may seem like the ideal solution. It gives companies the ability to tailor the software to exactly match their specific needs. However, manufacturers need to consider their system’s impact on health care users. What seems like a good idea in a meeting room may translate into extra work for no return to a health care professional. As health care staff are the most critical stakeholders in the supply chain, it is essential to take the views of end users on board.

Failure to focus on user experience also comes with commercial implications. As more products come to the market targeting the same indications, often with little to differentiate them in terms of efficacy and safety, is introducing a new unfamiliar technology to health care users going to help or hinder sales? Recent industry feedback revealed that the ability to schedule a patient’s treatment in an appropriate timeframe can be a deciding factor in which therapy to use. Many current platforms require health care users to perform a lot of work in the system before they are provided with a treatment schedule. Making this type of information accessible to end users earlier in the process would greatly benefit the health care professional, and possibly the manufacturer of the product supported by the system.

System design, build and maintenance

Building customized software is expensive and consumes thousands of work hours, both in-house and externally. As with any IT implementation, underestimating the time and costs associated with developing a bespoke solution is common. Projects often take longer, and cost more than initially anticipated, potentially delaying critical timelines and impacting budgets.

“Given the complexities and challenges inherent in cell and gene therapy orchestration, selecting the right technology platform is crucial for success.”

Even after a custom-built system has been implemented, costs continue to mount. CGT remains an emerging, rapidly evolving therapeutic area. What works today may quickly become obsolete due to evolving needs, changes in regulations and/or advances in technology. For a custom solution, costs and time associated with maintaining software in line with these changes are borne by the manufacturer. In addition to costs associated with building and maintaining bespoke software, manufacturers should also consider costs of end user training each time there is an update, and how this is delivered and managed. Finally, custom-made solutions often lack access to specialized features readily available in proven orchestration technologies.

Data integrations

Successful delivery of CGT products requires close collaboration across a network of stakeholders. This network includes hospitals, community clinics, payers, 3PLs, courier providers, CDMOs, QA/QC labs, and many others. Coordinating data exchanges and tasks across this ever-growing network requires data integrations. Building bespoke integrations between stakeholders can be costly and time-consuming for all parties, and these often need to be retested and potentially rebuilt any time there is a system update. As with initial build and maintenance costs, companies should bear this in mind if considering development of a custom solution.

The future

Sharing data between different systems becomes more complex when standards are not in place. This can hinder collaboration between stakeholders, impede data analysis, and slow down decision-making processes. Navigating multiple, disparate systems forces health care providers to spend valuable time on menial tasks, such as logging into different platforms and searching for information. This reduces their efficiency and takes away time that could be better spent on patient care.

Furthermore, the lack of standardization can make it difficult for companies to adapt to new technologies and regulations. For example, artificial intelligence offers huge potential in analyzing supply chain data to identify and help stakeholders address potential points of weakness. Delivering on this needs data that currently resides across multiple disparate systems. Lack of data sharing will limit innovation and hinder progress across the CGT sector.

A path towards standardization and collaboration

Going forward, the FDA expects to approve nearly 10- 20 new CGTs per year. This influx of novel therapies will exacerbate the challenges posed by custom-built solutions, making it increasingly difficult for health care providers to cope.

To address these challenges and unlock the full potential of CGTs, the industry must embrace collaboration alongside a transition to standardization. Technology providers play a vital role in driving this shift by developing platforms that adhere to industry standards and promote interoperability. These platforms can serve as a central hub for data exchange, enabling secure and efficient communication between manufacturers, treatment centers, and other stakeholders.

Open communication and knowledge sharing between manufacturers, technology providers, and health care professionals can drive the development and adoption of standardized practices. Technology providers can champion standardization by actively participating in industry initiatives and developing platforms that adhere to established guidelines and promote interoperability. By working together, stakeholders can create a unified ecosystem that facilitates seamless data exchange, reduces ambiguity, and ultimately accelerates the delivery of lifesaving therapies.

The intricacies of the CGT supply chain demand robust and standardized orchestration platforms. These platforms serve as central hubs for coordinating stakeholders, tracking the COC and COI, managing logistics, and ensuring regulatory compliance. By optimizing processes, enhancing real-time visibility, and promoting interoperability, orchestration platforms can significantly improve efficiency, mitigate risks, and expedite the delivery of therapies.

Choosing the right 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, especially in light of the push towards standardization and collaboration. Key features to look for include:

• 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 usercentric 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.

• Adaptability and future-proofing: The CGT landscape is constantly evolving, with new therapies, technologies, and regulations emerging frequently. 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.

• 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.

When evaluating orchestration platforms, companies should prioritize solutions that embrace industry standards. This ensures interoperability with existing systems, facilitates seamless data exchange, and reduces the risk of errors caused by inconsistent terminology or processes. By selecting a platform that champions standardization, organizations contribute to a more efficient and collaborative CGT ecosystem.

Beyond these essential features, it is also important to consider the platform’s cost-effectiveness and scalability. For smaller companies, or those with limited resources, a clear and transparent pricing model with options that cater to different needs and budgets is crucial. Equally important is the platform’s ability to scale seamlessly, accommodating growing data volumes and user numbers as the company expands. This ensures that the platform can handle the increasing demands of the CGT landscape without compromising performance or efficiency.

Strong vendor support and commitment to industry collaboration are also vital factors. 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.

Finally, decision-makers should consider the platform’s potential for maximizing value. Factors such as lower upfront costs, faster deployment, robust data security, and a clear product roadmap outlining future developments can significantly enhance the platform’s effectiveness and contribute to a greater return on investment.

By carefully evaluating these considerations, companies can choose an orchestration platform that empowers their organization to navigate the intricacies of CGT commercialization, accelerate the delivery of therapies, and ultimately improve patient outcomes.