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The Role of Digital/Automated Technology in Delivering Cell and Gene Therapies

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The advanced therapy medicinal product (ATMP) sector is growing rapidly both in terms of therapy diversity and investment, with the Alliance for Regenerative Medicine reporting almost 200 new therapy developers in 2021 and over 2600 active clinical trials. This is largely down to the success of early approved treatments such as Provenge (2010) in treating prostate cancer and subsequent successful ATMPs treating conditions like pediatric leukemia with long term success for patients, showing the potential to not only treat but cure what would otherwise be fatal illnesses. Scientists are now looking at the potential for these therapies to treat cancers, auto-immune diseases, rare genetic conditions as well as assisting with tissue regrowth.

What is involved in bringing advanced therapies to patients?

The potential of these therapies is massive and could positively impact many lives, but delivering these therapies successfully relies not only on the innovative science behind them, but an infrastructure and supply chain more complex than initially apparent. This is because many of these therapies are truly personalized, derived from patient blood, marrow, tumor or tissue samples, where cells may be extracted and altered or genetically mapped to create a treatment specific to the originating patient. The financial cost of each therapy is high, but an error leading to the wrong therapy being administered would trigger a potentially fatal cell rejection in the patient.

This process is complex and involves multiple specialist partners within a unique therapy journey that begins and ends with the patient. The need to “orchestrate” the components and partners in the supply chain is compounded by the critical requirement for temperature-controlled transportation and the viable life of the cells. If during clinical trials, developers cannot show they can safely transport samples from patients to labs, manufacture the individual therapy, and transport back to the patient within critical conditions and timescales, then the science is unpicked, and the product will not be approved.

Challenges of traditional supply chain management

Traditional solutions to track the ATMP patient journey have typically been paper based and very labor intensive, requiring manual reconciliation of patient registration forms, shipping details, manufacturing information, batch and infusion records with patient details, product labels and quality records. The resulting paper-trails can be geographically diverse, difficult to report and audit and require extensive resource to manage. As therapies progress through clinical trial phases, patient numbers increase making management cumbersome and highly prone to error. This was noted by the FDA during the Provenge approval process, stating that consideration needed to be made of supply chain orchestration for the product to remain safe post-approval.

Additionally, much of the above information needs to be reviewed as well as recorded to ensure the quality and condition of all elements have been acceptable throughout the process. Manually reviewing this data for every treatment is difficult to sustain and even harder to perform quickly. Advanced therapies such as CAR-T have a short viable life, even when handled correctly, cell cultures can take weeks to grow, with the early cultivated cells aging throughout the process. This transfers pressure onto the supply chain to minimize delays, ensure therapies pass quality checks and are delivered to patients before they lose their efficacy.

Furthermore, the patient’s role in the supply chain means that in addition to the need to record all partners that have handled the therapy and samples (chain of custody), what is known as the chain of identity (mapping the therapy to the right patient) creates a data protection consideration that does not exist in a traditional pharmaceutical supply chain.

Utilizing digitization and automation

It is in answer to the above challenges that the distinctions between simple digitization and an automated digital solution become important. Whilst switching to electronic records solves part of the paper-trail challenge, there is still considerable effort and margin for error in consolidating, storing, auditing and retaining records. ATMP trial records may have to be kept for up to 15 years after a trial ends and for a trial to lead to successful commercial release hinges not only on medical success but on successful audits (with FDA records suggesting that up to a third of audit failures are the result of inaccurate records).

The speed required in the decision-making process also drives towards a more automated system where pre-planned workflows can analyze the massive data volumes quickly and flag instances requiring action or decision to critical decision makers quickly. Where exceptions are identified quickly, product loss (estimated cost to the biopharma sector of product lost to temperature excursions alone was over $15b in 2014) may be avoided, recalls minimized and the chances of dangerous errors massively reduced. In addition, the detail behind this process is captured, electronically signed, and date stamped for future audit or review.

Integration between supply chain partners also has a huge part to play in this picture as it allows the automated flow of accurate information in real-time so critical stakeholders throughout the process can quickly make informed decisions. Pulling information in from third parties like couriers or manufacturing partners means patient data remains within the developer-controlled system and married with the anonymized patient ID, ensuring chain of identity can be validated without compromising data security or privacy.

Designing solutions that last

As an industry on the cutting edge of science, change is fast-paced and the therapies that are being developed vary significantly in their production steps. In a scenario where healthcare professionals and supply chain partners are being asked to follow different processes for multiple therapies, a system that can help automate, prompt and enforce these processes improves safety for patients, manageability for healthcare providers and ultimately reduces the cost of delivering the therapy. In 2021, 55% of FDA recorded recalls of biologics resulted from process failure and a further 39% from inadequate records or labelling. Recalls in this instance are likely to return the patient to the start of the cycle, a delay that could be devastating to a critically ill patient. Avoiding these recalls results in a smoother, more cost-effective patient journey and paves the way to better treatment outcomes.

As a result, orchestration solutions must be on platforms that can be adequately maintained in terms of security and connectivity and can both enforce the processes and critical pathways that govern each therapy supply chain and can flex and grow as therapies, process steps, geographies and languages are added.

The relatively “new” nature of the ATMP industry means developers are still determining the best practice methodologies to facilitate getting the therapies to patients. As a result, cloud hosted, multi-tenancy solutions have characteristics that lend themselves particularly well to ATMP orchestration, particularly when user support and software validation can be supported by the supplier. These types of solution typically facilitate secure and robust connectivity as well as being quick to configure and deploy, even when processes are highly tailored. The multi-tenancy environment shares the cost of keeping the platform secure and current and facilitates the sharing of best practice between therapies or even collaborating developers.

Whilst both supply chain and digitization may seem like small cogs in the mechanism of utilizing cutting edge science to progress the fight against previously untreatable conditions; there is a vital role to play in ensuring that patients can safely and cost-effectively gain access to these therapies. As such, the partners involved in the supply chain continue to collaborate on therapy orchestration to improve patient access to and safety of these life changing ATMPs.