A Decentralized Manufacturing Future

May 8, 2024 by Tom Lynch

Regulation, Development and Effects to Consider

When you answer questions, it can generate more questions, which can be a good thing if you’re working on a new development process. In my post, The Potential of Decentralization, I discussed the broad outlines of decentralized manufacturing, and as we do a deeper dive into this manufacturing approach, we need to address topics such as regulatory concerns, infrastructure needs and how we build in scalability.

Now, the goal of decentralization is to make cell and gene therapies more accessible in terms of cost, time, and availability to a wide range of patients. However, to achieve this objective, it will require significant time and development, along with direction from regulatory agencies around the world. Regulatory agencies are open to the idea of local or decentralized manufacturing, indicating that this approach makes sense for certain cellular therapies.

What’s the regulatory outlook?
In the U.S., the FDA released a draft guidance document titled, "Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products," in March 2022. This document addresses decentralized manufacturing for the first time. In this guidance, developers of CAR-T cell products are required to address the potential for product variability when manufacturing is distributed across multiple sites. They must demonstrate that products manufactured at each site and the associated analytical assays are consistent and meet regulatory expectations.

In the UK, the Medicines and Healthcare Products Regulatory Agency (MHRA) is also considering a regulatory framework for decentralized manufacturing of innovative medicinal products. This further indicates that regulatory agencies are recognizing the importance of adapting regulations to accommodate the changing landscape of drug manufacturing, including decentralized approaches.

In nearly all instances, developers are being encouraged to adopt measures such as automation and standardized processes to meet regulatory expectations and ensure the safety and efficacy of these advanced medicinal products. Regulatory agencies are actively seeking input and feedback from industry stakeholders to shape these evolving regulatory frameworks.

Building out the infrastructure
As manufacturing infrastructure matures, it's anticipated that elite medical centers and their manufacturing partners will become increasingly influential in the development and commercialization of cell and gene therapies. This shift may reduce the dominance of traditional big pharmaceutical companies and centralized manufacturing in the cell and gene therapy space. The effects could include:

Decentralized Clinical Trials

• Remote patient monitoring and data collection
• These have already become essential, especially considering the COVID-19 pandemic
• Virtual trial platforms to help ensure safe and effective treatment options
• Collaborative networks and partnerships as a crucial role in expediting treatments.

Benefits and Potential Impacts of Cell and Gene Therapy Decentralization

• Enhanced Patient Outcomes
• Streamlined treatments and patient-physician connections lead to better outcomes.
• Streamlined Regulatory Processes
• Decentralization simplifies regulatory approvals once a platform is established.
• Accelerated Innovation and Commercialization
• Standardized platforms enable innovation and commercialization.
• Economic Implications
• Decentralization can reduce costs through economies of scale.
• Ethical Considerations
• Empowering patients to take control of their health is an ethical responsibility.

Scaling for viability
Decentralization in cell and gene therapy has the potential to transform healthcare by improving patient outcomes, empowering the patient and clinicians, streamlining processes, and accelerating innovation. While innovation is essential in healthcare, demonstrating a sustainable business model is a critical step in the process. It not only ensures the long-term viability of the innovation but also facilitates its broader adoption, ultimately leading to improved patient care and outcomes.

Regulatory agencies are working to keep up with rapidly advancing technology, and emerging trends including artificial intelligence, as well as advances in automation and logistics could help catapult this manufacturing model into a successful practice.

Once a sustainable business model is established and proven, it can lead to a transformation in the healthcare market. Other players are more likely to follow suit, expanding the availability of the innovative approach and benefiting a larger number of patients.

• Learn more about Aldevron’s custom manufacturing services
• Have questions on this topic? Contact Tom Lynch
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