CELL & GENE THERAPY - It’s Time to Build Infrastructure to Handle the Coming Surge

INTRODUCTION

Cell and gene therapies are being developed faster than ever before, and they represent some of the most exciting devel­opments in all of medicine. Cell and gene therapies are person­alized medicines designed specifically to an individual patient. Although often grouped together because of their extraordinary impact and place on the cutting edge of medical tech, they’re quite different.

Cell therapies modify existing human cells from a patient or donor and infuse them back into the patient. The cells become therapeutic, and they’re designed to attack specific targets or dis­eases like multiple myeloma or leukemia. On the other hand, gene therapies directly impact a gene that is causing a condition or disease. These therapies can correct, eliminate, or even re­place a targeted gene using vectors to deliver new genetic mate­rial. Gene therapies can treat complex and rare diseases like hemophilia or spinal muscular atrophy. Cell and gene therapies can even be used in conjunction and combined into a single ther­apy.

These treatments are saving lives and giving second chances to patients who once thought they had no other options. Called “living drugs,” they are not only our best chance at finding treat­ments for rare genetic diseases that currently have no cure, but also offer greater potential to treat widespread diseases like can­cer, rheumatoid arthritis, or heart disease.

The complexities and innovations associated with the pro­duction of cell and gene therapies also necessitates a shift in in­frastructure, which will affect manufacturers, distributors, and providers. From development, manufacturing, storage, and de­livery to patients; each step in the process requires forging a new path.

THE TRAJECTORY

Since their introduction, cell and gene therapies have been among the fastest evolving therapies in medicine. Early CAR-T cell therapies saved the lives of children with late-stage pediatric acute lymphocytic leukemia. Additionally, the US Food and Drug Administration (FDA) approved a gene therapy to treat hemo­philia, a welcome advancement for patients who have historically faced a high risk of complications and who required ongoing treatment with very expensive medications.

These therapies significantly improve patient outcomes and reduce the ongoing burden of care. More than 6,000 diseases could potentially be treated by cell and gene therapies, affecting more than 350 million people worldwide.¹

Today, only a small number of these therapies have been ap­proved by the FDA, but a flood of new treatments is coming. More than 3,000 new therapies are in development globally, indicating a sign of their clinical potential.² In 2021, the US cell and gene therapy market was valued at around $5 billion. By 2027, it’s ex­pected to grow to about $37 billion, an increase of nearly 640%.³

When we look at the future of cell and gene therapies, clin­ical trials can give us a good idea of what may come next. Phase 3 trials are underway for therapies aimed at treating pulmonary hypertension, diabetic neuropathy, Parkinson’s disease, chronic heart failure, vision loss disorders, and some cancers. Although not yet approved, trials across such a broad range of conditions are proof of their potential to have a criti­cal impact on the standard of patient care.

These treatments have the potential to save untold lives; however, this potential is paired with some significant challenges.

THE CHALLENGE

Patients will only benefit from treat­ments if they have access to them. Patient access to cell and gene therapies is limited for many reasons, beginning with product development.

Cell and gene therapies are often de­veloped to treat very rare conditions. It is challenging to identify specific conditions likely to be responsive to these therapies and then to find enough patients suitable for participation in clinical trials from among some of the smallest patient pop­ulations in the world. Not only do manu­facturers of these therapies need to design and enroll patients in clinical trials from among small groups of medically fragile patients, but they must also find the right sites and clinicians to administer these treatments.

The necessary patient-by-patient manufacturing process for cell and gene therapies is yet another barrier. These ther­apies are among the most complex spe­cialty medications available; they require tailored manufacturing processes that cre­ate a completely individualized product for a single patient’s treatment. After manu­facturing, the product is volatile and must be handled with extreme care. This re­quires very sophisticated logistics, storage and tracking capabilities.

Cell and gene therapies are often stored at extremely cold temperatures, with temperature requirements as low as neg­ative 190 degrees Celsius or below. These temperatures necessitate special handling and safety precautions for the logistical and clinical teams handling them, with de­livery and on-site storage being particu­larly complex. Most provider sites are not currently equipped with cold chain storage for these products.

Upon receiving the product, providers administer it to a specific patient according to stringent processes and carefully chore­ographed timing. Once administered, tracking patient response and outcomes over time is extremely important in build­ing the body of knowledge in this emerg­ing field. This requires sophisticated chain of identity and custody solutions.

Upon completion of the requisite clin­ical trials, the process of securing FDA ap­proval is lengthy, expensive, and complex. However, regulatory agencies are already working to improve these efforts.

In 2019, FDA Commissioner Scott Gottlieb acknowledged the upcoming surge in these therapies, and predicted that by 2025, the department would be approving 10 to 20 cell and gene thera­pies per year. Even then, he seemed to un­derstand that changes would be necessary to meet the coming demand.

“We’re working to expand our review group dedicated to the evaluation of these applications to keep pace with the rapid expansion in new product development,” he said at the time. “Our eventual goal is to add about 50 additional clinical review­ers to the group charged with overseeing the clinical investigation, development, and review of these products. The activity reflects a turning point in the development of these technologies and their application to human health. It’s like the period mark­ing an acceleration in the development of antibody drugs in the late 1990s, and the mainstreaming of monoclonal antibodies as the backbone of modern treatment reg­imens.”

This expansion is a welcome develop­ment in our fight to save and improve lives, but a major challenge remains.

Cell and gene therapies are very ex­pensive — in fact, they’re some of the most expensive treatments available. The average cost is around $1.5 million per dose, with some treatment costs rising into the multimillions. In instances in which a therapy is administered a single time, this may be lower than the cost of ongoing life­time care.⁴ But for most patients and pay­ors, the cost of these treatments may be prohibitive and could limit their availability to the patients who need them most. This is a challenge we must address if we want to expand access to these treatments.

In addition to all the logistical and costly expenses, there are also a variety of challenges in the development and avail­ability of these therapies.

The first challenge starts at the begin­ning of the process with the identification of patients. It’s challenging to identify a very small population of people with a very rare disease, which can be an issue when that population is the target demographic for many of the cell and gene therapies that are on the market today. Manufactur­ers of these therapies need to design and enroll patients in clinical trials, and identi­fying patients for those trials is a significant lift.

Also, the necessary patient-by-patient manufacturing process for cell and gene therapies represents another barrier to broader patient reach, requiring single-dose preparation and extreme diligence to produce.

THE FIXES

At Cardinal Health, we work to widen access to life-saving treatments like cell and gene therapies, and we’re doing everything we can to move the industry forward. We have been working to build advanced infrastructure to support the de­velopment, manufacturing, distribution, and delivery of these products.

As the leading provider of compre­hensive services for cell and gene thera­pies, we are proud of the clinical, regulatory, logistical, and patient-focused value we offer to healthcare providers and pharmaceutical manufacturers. We have been here from the field’s inception, sup­porting the first CAR-T products, providing expertise in regulatory support, and con­sulting for more than 40 advanced thera­pies in 12 therapeutic areas. To date, we’ve processed thousands of cell and gene therapy orders.

We also have a partnership with Trak­Cel, the first provider of integrated cell and gene therapy software solutions for the precise management, control, and track­ing of cell and gene therapy products. Our partnership with TrakCel allows us to have information on chain of identity, chain of custody, location, and patient journey throughout the process of product delivery and administration. As partners, Cardinal Health and TrakCel offer industry-leading visibility and transparency to healthcare providers, manufacturers, and case man­agers working with these important med­ications. However, we can’t stop there.

Evidence-based medicine is critical to the delivery of better patient care. In the case of treatments that can cost millions of dollars, tracking and demonstrating the impact of these treatments on patient out­comes is key to increasing access. We must improve data collection before and after the patient receives treatment, which can be especially difficult when tracking pa­tients from cell and gene clinical studies. Patients are located all over the world, but the treatment centers providing these ther­apies are often limited to centers of excel­lence or large hospital systems, often in urban or populated areas. This makes long-term patient evaluation difficult and data collection inconsistent or incomplete. Because of this challenge, it’s difficult to create a complete data set around patient outcomes.

At Cardinal Health, we recognize these challenges, and we see them as op­portunities. Opportunities to partner with treatment centers, providers, and pharma­ceutical manufacturers to do better every day for patients. We continue to innovate beyond what we believe is possible, and we will strive to build a sustainable foun­dation upon which cell and gene therapies can thrive. We are committed to providing cell and gene therapy services that meet the needs of all stakeholders that will ulti­mately lead to better patient outcomes.

REFERENCES

1. https://med.stanford.edu/cdcm/CGT.
2. https://pubmed.ncbi.nlm.nih.gov/ 37927037/.
3. https://www.businesswire.com/ news/home/20220201006057/en/36.92-Billion-Cell-Gene-Therapy-Market—Global-Outlook-Forecasts-2022-2027—ResearchAndMar­kets.com.
4. https://icer.org/wp-content/up loads/2020/11/ICER-Gene-Therapy-White-Paper-030317.pdf.

Fran Gregory is responsible for cell & gene therapies and biosimilars at Cardinal Health. Her background includes extensive clinical, health economics and outcomes research, trade and commercial experience in the payer/ PBM, specialty pharmacy and biotech manufacturer spaces. Her career reflects a consistent focus on complex, innovative, high-value pharmaceuticals and a passion for breaking down barriers to access and care.