The global cell and gene therapy (CGT) market is growing rapidly at a compound annual growth rate (CAGR) of 22.42% for the 2022-2030 period and is expected to reach USD 93.78B by 2030[i]. This sector is seeing thousands of drug candidates currently under clinical and pre-clinical development[ii] and an increasing number of commercial approvals (10 new CGT approved in 2022 [iii]). Manufacturing productivity across the sector, however is low and is being constrained by manufacturing immaturity in both automation and digital integration. To support the successful delivery of the increased number of therapies to patients, it is critical to leverage the value of digital capabilities across the development phases and among the various stakeholders. Yet leveraging digital capabilities is perceived as a complex affair[iv] that requires different skills[v] to what the industry is used to and digitalisation of a wide range of activities across the value chain of CGTs. The implications of digitalization to date are therefore expensive and lengthy projects, not always successful[vi].
The industry is moving in the right direction, evidenced by activity in the space by leading academic institutions[vii], the formation of various working groups and initiatives largely funded by private entities such as BioPhorum, International Society for Pharmaceutical Engineering (ISPE) or International Society for Cellular Therapy (ISCT) with the aim of providing tools, solutions and roadmaps for digital transformations in the biotechnology space. For example, the BioPhorum IT group for CGTs has created a set tools to help identify who needs to be involved in the supply of different types of CGTs and what they do at a high level with particular focus on how IT systems can support that. Yet as an industry, we are still in our infancy with respect to the adoption of digital solutions.
Collaborating to drive change – Workshop summary:
To gain further insight on what has hindered adoption up to now, as well as to understand the main data and digital challenges facing the industry going forward, PA Consulting and the Cell and Gene Therapy Catapult jointly hosted a workshop at Phacilitate’s Advanced Therapy Week 2023 in Miami on “Building a common digital approach to unleash the CGT sector”. Over 20 senior leaders across the CGT industry (Figure 1) joined a 2-hour session to align on priorities and start building a common ground for a harmonised digital approach tailored to CGT.
The session started by identifying the major barriers (from a data and digital standpoint) which affect the different players involved in the development, manufacturing and delivery of cell (autologous and allogeneic) and gene therapies. It purposely spans across a broad range of areas such as patient recruitment, enrolment and therapy delivery at the point of care, data collection and data analysis during the therapy development and manufacturing process, interaction between manufacturers, suppliers, logistics companies and hospitals, and more. Some examples of the identified barriers were:
- Not enough visibility and tracking of data, along with governance and privacy concerns as the materials move across the value chain.
- Vendor unique systems add complexity to data acquisition and connectivity to overarching IT infrastructure.
- No centralised data systems. Currently duplication of data is common in healthcare systems as they use different portals and platforms.
The discussion led to the identification of dozens more examples of practical challenges faced by different stakeholders in the industry. Yet from all those practical challenges, some common themes emerged and can be grouped into 7 key categories summarised in Figure 2.
When analysing these 7 overarching challenges it becomes evident to those outside of the CGT industry, particularly to those with experience in digitalization, that these are not new or too dissimilar to what other sectors have experienced. The conversations highlighted the importance of crosspollination to avoid embarking on digital projects seeking solutions that already exist and may well be easily tailored to service the CGT space. Below, we have highlighted some of these examples:
The size, shape, nature of data within CGT is complex and includes large data sets, such as genetic sequences, cell populations, and functional assays. The collection, processing and analysis of these data sets can be a costly undertaking for small organisations as specialised tools, significant computing power and expertise are often required.
However, large scale, non-alphanumeric datasets, are well understood in other adjacent healthcare industries – for example, the wider genome sequencing industry has established cloud compute standards for handling the ingress & egress challenges of very large scale data; while healthcare image manipulation is standardised through Digital Imaging and Communications in Medicine (DICOM).
Integration is challenging in many levels. Within a particular development phase, it is common that seamless integration between unit operations or groups does not exist. For example, pre-clinical development involves multiple data sources, including data from process development, animal studies, in vitro assays and others. These data sources come in different formats or use different data standards. In addition, the data collection practices can be quite manual especially in early development phases in academic labs. To increase the complexity around the integration challenge, it is not limited to connectivity problems within a development phase, but rather spans over the various development phases. For instance, the systems used to log information during manufacturing are vastly different to those used in pre-clinical. The systems, security and governance for data collected in healthcare centres is once again vastly different to that in a manufacturing facility. It can boil down to trivial issues such as things like some healthcare centres not allowing operators to use USBs and software providers needing those users to utilise a USB. Lack of automated data collection and lack of alignment or harmonisation on best practices are key challenges for integration.
For example, cross-industry healthcare bodies like medical societies regularly integrate with diverse datasets from across healthcare systems in multiple countries. These bodies have invested in standards development and have been active members of the Health Level 7 (HL7) community.
The CGT industry continues to capture significant amounts of data in inaccessible and disconnected forms – in bench logbooks, paper records, and on stand-alone machines. Compounding this, the data being captured is increasingly sensitive. Data is no longer purely anonymous biochemical research in early-stage lab work, it now includes highly detailed, protected patient data alongside commercially sensitive corporate information.
As the industry matures it must make provision for appropriate management of the data and records it holds. This includes appropriate measures to understand which jurisdictions data can be held within; the legal needs to be able to access and identify what information is being held, on who; and also a strategy for safe and appropriate record disposal at the point information is no longer required – both to comply with legislation but also to reduce unnecessary corporate overheads.
For example, with the introduction of European GDPR legislation over a decade ago led to all large pharmaceutical organisations having to take a revised look at records management; where information was stored; how research data was held and in what jurisdictions.
Organisations need to have the right set of digital skills and competence to appropriately select and use the digital tools and approaches. This enables the development and deployment of processes to capture, store and analyse data, as well as ensure data security.
Organisations in the CGT space is often strong in science and engineering but often lack deep digital competence. We’ve seen examples within the broader biotech industry where “CIO skills” can be dropped into startup organisations to support on the early stages of their growth journey.
Security again is a multi-faceted challenge. There are concerns associated with the potential for leaking or cyber-attacks of sensitive patient data which is often being handled by more than one person or party. With autologous therapies, there can be multiple hand-off points, exposing multiple systems and stakeholders to this sensitive data – an end-to-end solution is only as secure as the weakest link. It is therefore important to have strong solutions around chain of identity and chain of custody, two critical records used to ensure and monitor product safety, spanning from creation of the therapeutic material, through to administration into a patient and any follow-up activities. Importantly, there needs to be an audit trace of data to ensure legitimacy and that the data has not been corrupted or manipulated.
Completeness, timeliness, and accuracy of attributable data is critical across the entire product lifecycle. Poor-quality data can lead to incorrect conclusions, decisions and lack of regulatory compliance. Quality issues can arise due to factors such as human error, equipment failure, improper data handling or insufficient collection of data.
For example, front line hospital systems have been forced to tackle data quality issues for decades. This is to manage individual care pathways effectively, ensure clinical safety incidents don’t occur, and create efficient operations across healthcare systems. The data quality routines such as up-front data entry controls, regular data cleansing activities, and shared data quality teams can all support improved quality management in the CGT industry.
The overall process may require working and interfacing with a variety of products or systems that may input/output data in a variety of formats, be intermittent in providing data, or not provide any data at all. Being able to cope with this variability is important for successful and efficient implementation.
With various therapy developers using different systems, this can lead to various clinical adoption problems downstream. For instance, various companies using different supply chain solutions mean hospitals offering different therapies may have to be trained on multiple supply chain solutions, wasting valuable healthcare professional time and resources.
For example, the recent global mass-vaccination initiatives surrounding COVID-19 has shown how interoperability of systems is both important, and during times of crisis or urgency, traditional barriers of interoperability are broken down – whether through formal or informal routes to ensure that data can flow and action can be taken where it needs to be taken.
As the workshop came to an end, the attendees reflect on the insightful discussions and algin on three key outputs as conclusions:
- The industry understands the benefits of digital adoption and most intend to pursue it in varying degrees.
- The magnitude and complexity around implementation, however, together with the lack of access to digital skills makes leadership buy-in (and therefore investment) challenging.
- A degree of industry-wide harmonisation would help reduce the time and cost of adoption as well as streamline connectivity.
However, questions remain around: (1) how to harmonise?; (2) how to achieve critical mass to encourage solutions to adopt these standards?; (3) can we leverage learnings from other industries such as the smart home applications where smart devices can quickly and easily connect to a platform to automate operations in a household (from grocery shopping to vacuuming to turning on lights and even opening curtains)? (4) can we achieve this harmonisation in a highly regulated industry?
An industry-wide consensus on how to harmonise would go a long way in removing a key barrier to digital innovation and adoption. Various industry trade bodies and academic groupings are already investigating ways to replace the requirement for the current costly, time-consuming tailored connections. As a sector, if we can ensure that there is no compromise on flexibility when it comes to deciding which are the best systems and/or vendors to use for any given activity/unit operation, then we will see an increase in leadership buy-in and start to harvest the true benefits from digitalisation from much earlier in the product development lifecycle, realising the vision of Industry 4.0.
If you are interested in harmonisation for wider digital adoption, please let us know by contacting us via the links below, and we will keep you informed of future consensus-building activities.
[i] Precedence Research, 2022