The Role of Digital/Automated Technology in Delivering Cell and Gene Therapies
The Advanced Therapy Drugs (ATMP) industry is growing rapidly in terms of both therapy diversity and investment, with the Alliance for Regenerative Medicine reporting nearly 200 new therapy developers in 2021 and more. 2600 active clinical trials. This is largely due to the success of the first approved treatments such as Provenge (2010) in the treatment of prostate cancer and the subsequent successful ATMPs that treat conditions such as pediatric leukemia with long-term success for patients, which show the potential not only to treat but to cure what would otherwise be. deadly diseases. Scientists are studying the potential of these therapies to treat cancers, autoimmune diseases, rare genetic diseases, as well as help with tissue growth.
What does it mean to bring advanced therapies to patients?
The potential of these therapies is huge and could have a positive impact on many lives, but the successful delivery of these therapies depends not only on the innovative science behind them, but on the infrastructure and supply chain. more complex than initially apparent. This is because many of these therapies are truly personalized, derived from samples of the patient’s blood, marrow, tumor, or tissue, where cells can be extracted and altered or genetically mapped to create a specific treatment for the patient. original patient. The economic cost of each therapy is high, but an error leading to the administration of the wrong therapy would result in a potentially fatal cell rejection in the patient.
This process is complex and involves multiple specialized partners within a single therapeutic journey that begins and ends with the patient. The need to “orchestrate” supply chain components and partners is compounded by the critical requirement for temperature-controlled transport and the viable life of cells. If during clinical trials, developers cannot demonstrate that they can safely transport patient samples to laboratories, fabricate individual therapy, and transport them back to the patient under critical conditions and deadlines, then science is not selected and the product will not be approved.
Challenges of traditional supply chain management
Traditional ATMP patient journey tracking solutions have been paper-based and labor-intensive, requiring manual reconciliation of patient registration forms, shipping details, and patient information. manufacturing, batch records and infusion with patient details, product labels and quality records. The resulting paper traces can be geographically diverse, difficult to report and audit, and require extensive resources to manage. As therapies progress through the clinical trial phases, the number of patients increases making management cumbersome and highly error-prone. This was stated by the FDA during the Provenge approval process, and stated that the orchestration of the supply chain had to be considered in order for the product to remain safe after approval.
In addition, much of the above information must be reviewed and recorded to ensure that the quality and condition of all items have been acceptable throughout the process. Manual review of this data for each treatment is difficult to maintain and even more difficult to perform quickly. Advanced therapies such as CAR-T have a short viable life, even when handled properly, cell cultures can take weeks to grow, and early cultured cells age throughout the process. This transfers pressure to the supply chain to minimize delays, ensure that therapies pass quality controls, and are delivered to patients before they lose their effectiveness.
In addition, the role of the patient in the supply chain means that, in addition to the need to register all the partners who have managed the therapy and the samples (chain of custody), what is known as the identity chain mapping therapy to the right patient) creates a data protection consideration that does not exist in a traditional pharmaceutical supply chain.
Using digitization and automation
It is in response to the above challenges that the distinctions between simple digitization and an automated digital solution become important. While switching to electronic records solves part of the challenge of paper tracing, there is still considerable effort and margin of error in consolidating, storing, auditing, and preserving documents. ATMP test records may need to be kept for up to 15 years after the end of a trial, and for a trial to lead to a successful commercial release depends not only on medical success but also 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 leads to a more automated system where pre-planned workflows can quickly analyze massive data volumes and mark instances that require actions or decisions on critical decision makers quickly. When exceptions are quickly identified, the loss of product (the estimated cost for the biopharmaceutical sector of the product lost only by temperature excursions was more than $ 15 million in 2014) can be avoided, reminders are minimized, and the chances of dangerous errors are greatly reduced. In addition, the details of this process are collected, electronically signed and the date is set for a future audit or review.
Integration between supply chain partners also plays a very important role in this image, as it enables the automated flow of accurate real-time information so that critical stakeholders throughout the process can make informed decisions quickly. Obtaining third-party information such as messengers or manufacturing partners means that patient data remains within the developer-controlled system and is combined with the anonymous patient identifier, ensuring that the identity string can be validated without compromise. data security or privacy.
Design solutions that last
As an industry at the forefront of science, change is rapid 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, request and enforce these processes improves security for to patients, the management capacity for healthcare providers and ultimately reduces the cost of delivering therapy. In 2021, 55% of The FDA recorded records of organic products was the result of process failures and 39% more inadequate records or labeling. Memories in this case are likely to return to the patient at the beginning of the cycle, a delay that could be devastating for a critically ill patient. Avoiding these memories translates into a smoother and more cost-effective patient journey and paves the way for better treatment outcomes.
As a result, orchestration solutions must be on platforms that can be properly maintained in terms of security and connectivity and that can enforce the critical processes and 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 that developers are still determining best practice methodologies to facilitate access to therapies for patients. As a result, cloud-hosted multi-lease solutions have features that lend themselves particularly well to ATMP orchestration, especially when the vendor can support user support and software validation. Such solutions typically provide secure and robust connectivity, as well as being quick to set up and deploy, even when processes are highly tailored. The multi-lease environment shares the cost of keeping the platform secure and current and facilitates the exchange of best practices between therapies or even collaborating developers.
While both the supply chain and digitization may seem like small gears in the mechanism of using cutting-edge science to advance the fight against previously incurable conditions; there is a vital role to play in ensuring that patients can access these therapies safely and cost-effectively. As such, partners in the supply chain continue to collaborate in orchestrating therapy to improve patient access and the safety of these life-changing ATMPs.