In recent years, gene therapies, gene-modified cell therapies and cell therapies, collectively known as ‘Advanced Therapies’, have rapidly become an integral part of the R&D programs of many Biopharmaceutical companies.
This is illustrated by some of the key trends and metrics published by the Alliance for Regenerative Medicine (ARM) in their most recent Quarterly Global Regenerative Medicine Sector Report. For example, there are now in excess of 900 companies citing Advanced Therapies as part of their R&D portfolios. In addition to several recent FDA approvals, there are currently over a thousand trials for Advanced Therapies underway worldwide across a range of disease areas, many of which are showing promising clinical data.
Despite this phenomenal growth in interest in Advanced Therapies, there are still significant technical and financial challenges associated with their development and manufacturing. You may have seen our article in European Pharmaceutical Manufacturer which described the pressing need for faster and more effective process development and how technology can help. In this blog, we’ll be expanding on this and describing how better use of informatics can underpin development activities and outlining the key attributes an informatics platform must possess in order to address the complex needs of any Advanced Therapy R&D organization.
Timely, accessible information
The development of a novel advanced therapeutic requires an increasingly diverse range of activities, performed by multiple specialist teams. While this results in an increasingly complex picture, collaboration both within and between teams can be significantly simplified when everyone uses the same platform to manage their data. This eliminates manual hand-offs of data between groups and streamlines communication between them.
When process variables, in-process analytics and QC data are all managed in one place, it becomes simple to collate all information and context relating to a given batch of product. It is also easier to determine the impact of changing a process parameter, such as incubation conditions, on the quality attributes of the product, such as the titer or purity.
A single platform also enables organizations to use a common language, such as terminology and units of measurement and facilitates a recipe-based approach to process data capture. This not only facilitates technology transfer and scale-up, but also reduces ambiguity and ensures that processes are repeatable and reproducible.
When all product and process data is managed centrally, there is a single point of truth – scientists don’t need to look in multiple systems to find the data they need. This in turn enables a ‘self-service’ approach to data – being able to access critical information in a timely manner, rather than having to chase experts for information that will help inform development decisions.
Supporting process execution and improving quality
While managing all process and quality data centrally will be of benefit to any organization, it doesn’t have to stop there. Systems that not only capture data but also support scientists as they conduct their work present an opportunity for significant gains in capacity and quality. For example, laboratory workflows can be streamlined by directly connecting instruments, such as cell processing devices and analytical equipment, which saves time and also eliminates error-prone manual transcription.
When data is captured at the point of process execution, users can also be alerted to potential issues, such as a parameter or result that is outside of the expected range or a reagent that is out of date – all contributing to increased quality.
Flexibility and adaptability
One of the defining characteristics of all Advanced Therapies is the fast-moving nature of their development. With this comes the challenge of supporting processes and technologies that are both innovative and complex.
Many of the methods employed in the development of advanced therapies are evolving quickly, so it’s essential that an informatics platform is sufficiently adaptable to incorporate new workflows and adapt existing ones.
Similarly, advanced therapies involve the creation and management of a range of different materials, such as DNA constructs and cells. As such, an informatics platform must be able to manage a variety of types of molecular and cellular entities, each with different properties, and have the flexibility to incorporate completely new entities whilst adhering to standards which describe macromolecules, such as HELM.
When processes are evolving, it’s important to link a batch of material to the version of a process was used to generate it. But the need for traceability goes much further than this. In particular for autologous therapies, where a patient’s own cells form the basis of the treatment involving a process that effectively produces a ‘batch of one’, the ability to trace the location of material and its chain of custody is essential.
More generally, it’s critical to be able to manage and query the genealogy of materials, from their creation through to their use, and the relationships between them, such as between a cell line and the DNA construct used to generate it.
Advanced Therapies present an exciting opportunity for both the pharmaceutical industry and patients. To be able to support their development effectively, an informatics system must be flexible enough to adapt to an environment characterized by evolving techniques and processes, whilst ensuring traceability of both materials and data. It should also help scientists by not only streamlining their workflows, but also by providing them with access to all the relevant data they need to make timely, impactful decisions.