Standardization, more capacity may be needed as CGTs grow

By Jennifer Rabin, Specialist Leader, Deloitte Consulting LLP

I was an apheresis nurse in 2017 when the first gene therapy was approved in the United States.¹ Because the therapy was new, regulators required anyone who might come into contact with it, or with the patient, to complete Risk Evaluation and Mitigation Strategy (REMS) training.² My hospital provided REMS training for about 500 employees; even a few people from the business office had to undergo training. Over time, those stringent requirements were loosened.³ Today, typically only the handful of people who are directly involved in the process need to go through REMS training at each facility.

Since 2017, the U.S. Food & Drug Administration has approved 19 cell and gene therapies (CGTs) for the treatment of 25 conditions.⁴ More than 1,500 other cell, gene, and RNA therapies have been registered for clinical trials.⁵ These treatments, collectively known as advanced therapies, are being evaluated for the treatment of cancers, neurologic disorders, cardiac disease, autoimmune disorders, and rare diseases.

But anticipated growth in the CGT space has fueled some concern. Health systems typically don’t have the infrastructure or resources needed to meet the increasing demand.

As more of these therapies are approved, health systems, cancer centers, and outpatient facilities will likely need to find ways to increase their capacity to accommodate an influx of patients. They might also need to develop and agree on standardized protocols and practices to help improve efficiencies.

IWG panel discusses CGT trends, challenges

It has been five years since Deloitte launched the NextGen Therapies Industry Working Group (IWG) to provide a forum for CGT leaders to share perspectives and leading practices. We recently wrapped up our 2024 IWG meeting. Invited attendees included biopharmaceutical companies, clinicians, CGT manufacturers, researchers, and health system executives. Smaller cohorts from this group get together every couple of months to work on more tactical issues.

For me, one of the most interesting sessions at this year’s IWG meeting explored some of the challenges health systems can face in delivering advanced therapies to patients—particularly capacity and scheduling. The panel, which was moderated by a CGT manufacturer, included two physicians who head transfusion medicine and apheresis collection for health systems, a representative from a laboratory that collects patient cells for infusion, and one health system administrator. One of the physicians noted that new challenges have arisen as the apheresis field (the process of taking blood from a patient and extracting cells used to manufacture the therapy) has expanded from strictly an inpatient setting to outpatient facilities.

A physician from a children’s hospital noted that apheresis can take as long as six hours. Moreover, apheresis units tend to be small and might not be able to handle multiple patients. Health systems, he said, need to determine how to conduct more than one collection at a time without overburdening the laboratories. There is also a time element to consider. Panelists suggested that allowing CGT manufacturers to contract with freestanding apheresis units might be one way to free up capacity.

The moderator explained that his organization’s CGT program is modeled after its decades-old bone-marrow transplant program. That model is now being used for CGT programs outside of oncology. He explained that the current care model needs to evolve because the existing infrastructure is not set up for advanced therapies.

CGT standards can be limited

Many patients who rely on advanced therapies for the treatment of cancer and rare diseases are often severely ill. A delay in treatment could have dire consequences. As CGTs expand into other areas (e.g., autoimmune, retinal disease, dermatology) some patients might not have a life-threatening illness but might still be severely compromised by their disease. The volume of eligible patients in these populations is larger than in the oncology space and could require therapies to be produced at a larger scale.⁶

As the CGT patient population grows, health systems should have a strategy for triaging them. The panel agreed that standardization could help improve efficiencies. While health systems sometimes share information about their CGT processes, it typically doesn’t occur frequently or in a tangible way. The most experienced CGT centers might be able to develop an organizational playbook that the rest of the country’s therapy centers could follow. That hasn’t happened yet because CGT does not appear to be a priority for most health systems—even among those that have the most experience.

Health systems should look for ways to reorganize themselves to meet anticipated demand for CGTs. One of the physician panelists noted that each patient is unique and has their own complexities and needs. Being patient-focused is important, but it can require additional coordination.

Conclusion

Biopharmaceutical manufacturers, labs, health systems, and other stakeholders should consider these two questions in preparing for the potential growth of CGTs:

• What should the role of the health system be in meeting capacity demands?
• What should the role of the CGT developers be in reducing the storage and handling processes to make these transformative therapies more accessible outside of major academic centers?

We are just scratching the surface in realizing the potential of advanced therapies. No single academic medical institution is likely to be able to meet the needs of all patients in need of CGTs. That means the entire ecosystem (physicians, administrators, manufacturers) should look for opportunities to collaborate to develop leading practices, operating models, and standards.

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Endnotes:

^{1_{FDA approval brings first gene therapy to the United States, FDA News releases, August 30, 2017}}

^{2_{Frequently Asked Questions (FAQs) about REMS, FDA, January 26, 2018}}

^{3_{Risk Evaluation and Mitigation Strategies, FDA, May 16, 2023}}

^{4_{Approved cellular and gene therapy products, FDA, April 26, 2024}}

^{5_{2023’s market outlook for cell and gene therapies, Cell and Gene, February 14, 2023}}

^{6_{Cell and gene therapy landscape in the US, GlobalData Healthcare, February 28, 2024}}

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Deloitte shall not be responsible for any loss sustained by any person who relies on this publication.

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