How to Decentralize Cell and Gene Therapy Treatments

The Challenge: CAGT Treatments Require Time and Lack Availability

Persons diagnosed with life threatening diseases which are now treatable with cell and gene therapies (CAGT) often needed to wait for a significant amount of time for treatment due to lack of availability.

In order to combat these challenges, companies need to address several key points:

1. How to evolve our healthcare systems to provide cell and gene therapies to patients at point-of-care facilities while maintaining or improving the quality, safety and efficacy of the treatments. The creation of an on-demand service would put the patient first.
2. Cell and gene therapies are extraordinarily complex individualized treatments which require a high degree of process understanding and careful decision making, resulting in a highly educated, labor-intensive process to achieve successful results.
3. How to manage the significant regulatory hurdles involved with cell and gene therapy treatments including treatment application, appropriate reporting, follow up and signal identification.
4. GxP processing, release, and assurance that the treatment supplied meets regulatory requirements to treat patients.
5. Raw material logistics, costing, and reimbursement challenges with unproven treatments.

The Solution: Make CAGT Treatments Available at Point-of-Care Facilities

At ProPharma, we realize this is no easy feat. There is a lot involved with the manufacturing and regulatory requirements around cell and gene therapy treatments. But, instead of accepting the status quo, where patients must travel far away to receive treatment, or the patient tissue gets shipped across the globe, made into the therapy, and then shipped back to the patient, what if there were a different way?

At ProPharma, our experts are working as teams in the RCO model to:

1. Define critical systems to establish a fully functional licensed compliant point of care facility capable of manufacturing treatments. ProPharma offers full regulatory planning to ensure that the site can treat patients with products produced at their site. ProPharma also offers business development planning to ensure that the right funding is available when needed to achieve phase appropriate milestones.
2. Build or remediate cell and gene therapy treatment labs at research hospitals and treatment centers. This includes an applicable supply chain to ensure the components including viruses, media and patient starting materials are handled in a GXP compliant and controlled way. Architectural firms will supply architectural, mechanical, electrical, plumbing layouts but they need a concept to design from. Designing the proper personnel, material, air and product flows is critical to ensure both personnel and patient product safety. ProPharma works with multiple hospitals and research institutions to develop qualified spaces for effective cell and gene manufacturing.
3. Perform the required equipment selection, qualification and process validation. For example, building the facility is a major step, but equipment selection, qualification, and process validation are required to run a 1271 cell and gene facility that intends to treat patients. All these steps are part of a regulatory filing needed to gain a license to run. Process validation is a challenge with orphan diseases and small treatment sets. Therefore, companies can take a matrix-based small sample set approach that allows bracketing of the patients and treatments to prove, with a high degree of assurance, that the process will meet pre-determined specifications in a no size fits all environment.
4. Develop safe and effective treatments requires extensive education, training, documentation, process development, studies, data and analysis to demonstrate control. For our clients, we facilitate proper development by implementing IT data control, supplying study framework for development, finding staffing requirements and performing staffing / de-staffing as needed to achieve goals. We build the algorithms to properly isolate the variability as well as the causal factors to help clinicians spend more time perfecting treatments and less time troubleshooting. ProPharma’s approved validation platform creates an environment that allows facilities to concentrate on the patient.
5. Provide auditing and inspection readiness support. All raw material suppliers require auditing, and the site will need to be inspected prior to approval. ProPharma’s industry leading team of auditors help ensure that the product being provided to patients has been properly controlled throughout the process.

Automation Solutions Can Bring Treatment Closer to Patients

For years ProPharma has been working with equipment manufacturers to develop effective automation solutions to move treatments closer to the patients who need them. Our team has worked with all major equipment suppliers in multiple roles from equipment design, production, to qualification and validation. By providing in-depth analysis of the strengths and weaknesses of each type of automation solution allows clients to have a real perspective on the best program for their application.

Overcome Regulatory Challenges by Selecting the Proper Pathway

From hospital exemptions to licensing applications, it can be overwhelming to figure out what to apply for and how to present the information. ProPharma has worked with authorities to demonstrate the science behind the innovation. This partnership allows clinicians to quickly move their treatment to the patients who need them via proper regulatory pathways.

Decentralize Your Cell and Gene Therapy Treatment with ProPharma

In the United States, US News and World Reports rank hospitals on several factors including their innovative therapies. Patient survival rates are one of the key criteria utilized, and supplying the right treatment at the right time is the best way to achieve higher survival ratios. Higher survival ratios result in higher rankings, and higher rankings lead to hospitals becoming patients’ service providers of choice.

ProPharma has supported hospitals to effectively treat patients with cell and gene therapies, guiding them through the regulatory and compliance challenges in order to bring these treatments to patients safely and successfully. If you are looking for help bringing CAGT therapies to your facility, contact us today to speak with one of our experts.

Author

Bob Beall

Vice President, Quality and Compliance