CGT, The Value Chain, And The Central Role Of The Patients

By Kelly Kester, MBA, MS, RD, Director of Product Management, Advanced Therapy Solutions and Shannon Williams, Director of Commercial Development, Advanced Therapy Solutions, Cardinal Health

As health care advances, the world continues to move toward individual and personalized treatments. In fact, the current drug pipeline consists of more than 250 cell and gene therapies in various stages of development and the market is anticipated to reach more than $36 billion by 2026.

Until this point, CGTs have spurned immense excitement across pharma, providers, patients, and payers alike, primarily due to the future potential of the therapies. In this sense, CGTs offer the opportunity to progress from symptom management to potentially curative therapies. But with the CGT commercialization boom approaching in the U.S., it is clear how critical the individual patient is in the development of these therapies — not just as the end user of the treatment, but as part of the actual product, specifically for gene therapies and autologous cell therapies.

With this understanding, the central role of patients and caregivers in the CGT space is further elevated; their voices are critical in the space as it is built from the ground up.

Here are three ways patients’ perspective can be incorporated across the CGT value chain:

Bringing Visibility to Patients and Caregivers

Understanding a product’s exact position in the supply chain is not a new goal for worldwide pharmaceutical supply chains, but with the arrival of precision therapies comes a growing need and appreciation for tracking products in real-time. The continuous monitoring of these products during storage and transit is vital not only for cost management, but also to ensure product quality and patient safety. In fact, real-time tracking is largely considered one of the most pressing challenges affecting the pharmaceutical industry, especially when paired with complex product characteristics like patient-specific donor materials (when applicable) and cryogenic temperature monitoring, among others.

However, while the current mindset surrounding tracking visibility is limited to biopharma manufacturers and providers, there is an emerging school of thought that patients and caregivers are among those with the right to visibility as well, especially in the case of therapies that include patient-specific donor materials. Across CGTs, visibility into the status of their therapy can provide comfort and assurance while waiting for therapy arrival and administration. In an age where patients can track their retail shipments and restaurant deliveries, CGT manufacturers can set a new standard for the patient and caregiver experience in health care. Although privacy concerns and other challenges have emerged, CGTs have a unique opportunity to introduce patient visibility into the supply chain.

Patient Access as a CGT Success Measurement

Although access is often defined traditionally by patient support hubs and narrowing in on the patient as the product, that idea is beginning to shift to include the supply chain as a critical component. A variety of complexities exist today that limit patient access to CGTs, including the commercial cost of therapy, travel and lodging required to reach administration sites (primarily academic medical centers) and a non-linear pathway to therapy, among others.

As we move from therapies focused on symptom management to potentially curative therapies that may result in one treatment in a lifetime, the cost constraints change dramatically, often leaving patients struggling to access these medications. Without the active involvement of patients and donors, CGTs will not enter clinical development nor progress to commercially available products.

In spite of the significant emphasis on patient access, patients are often still struggling to receive treatments, which is ultimately affecting the results and future of the products.

We must continue to put patient access at the core of CGT development and commercialization as the world shifts to a new approach to patient care.

Building a Patient-Focused Data Ecosystem

Just as CGTs challenge the traditional physical and digital supply chain footprints, a patient-specific focus is warranted when it comes to post-administration considerations. In 2020, the FDA released guidance recommending the design of long-term follow-up studies for gene therapy products due to the nature of the therapies and the increased risk of undesirable and unpredictable outcomes that may present as delayed adverse events. 

The extensive LTFU requirements present particular complications for biopharma manufacturers and providers, as multi-year patient follow-up in the real world is not commonplace within today’s health care system. The ability of manufacturers to collect patient data long after treatment requires the coordination and cooperation of payers, providers and most importantly patients.

While the push to obtain patient-reported outcomes measurements is not new to health care, it often creates challenges for providers, already struggling to keep up with administrative work, because of the challenges of collecting and using this information in a way that does not disrupt their workflows. From the patient perspective, the goal will be to find a way to ensure they keep up with outcomes reporting, whether through the gamification of data or other incentives.

As more of these precision therapies come to market, understanding how patients fit in the supply chain both as a patient and part of the product will be vital to ensuring success.

Kelly Kester is the Director of Product Management for Advanced Therapy Solutions at Cardinal Health. Over the last decade, Kelly has spent her career tackling complex problems across healthcare and life sciences. Beginning her days in healthcare as a Registered Dietitian and Clinical Research Associate at Rush University Medical Center, Kelly specialized in neurointensive care, gastroenterology, and sports nutrition. Soon, she translated her clinical expertise into marketing and strategy consulting for contract research organization (CRO) and genomics clients before joining Cardinal Health’s corporate strategy and business development team in 2019. Kelly holds a master’s degree in Clinical Nutrition from Rush University and an MBA in Strategy and Finance from DePaul University.

Shannon Williams is the Director, Commercial Development for Advanced Therapy Solutions at Cardinal Health.  Shannon has 15+ years of healthcare experience (serving 13 of those years at Cardinal Health), holding roles in finance, strategic sourcing, data & analytics, strategy, innovation, and commercial operations.  She holds dual bachelor’s degrees in Finance and Marketing from Ohio University.