Imagine a cancer patient who has exhausted all conventional treatments, their hope dwindling as each option fails to deliver results. Their doctor mentions how CAR-T, a cutting-edge gene therapy, has shown remarkable success in targeting and destroying cancer cells. But the cost of the treatment is staggering—starting at $373,000, with some therapies made from patients’ own cells exceeding $3 million.
For most people, such astronomical costs put these potentially life-saving treatments far out of reach, leaving them with little hope of accessing the care they desperately need.
Fortunately, a new model is emerging that could significantly lower costs, making these life-saving treatments more affordable and accessible.
Bridging the Gap: Expanding Access to Life-Saving Cellular Gene Therapies
In the rapidly advancing field of medical treatments, cellular gene therapies (CGT) such as CAR-T cell treatments are at the forefront. CGT offers hope to patients with severe conditions, particularly various forms of cancer and genetic disorders, where traditional treatments have failed. However, the challenge remains: how to make these cutting-edge therapies accessible and affordable to all, especially those in rural and economically disadvantaged areas.
CGT are advanced treatments that reprogram a patient’s own cells to fight diseases. For example, CAR-T cell therapy involves modifying T cells, a type of immune cell, to target and destroy cancer cells.
Treatments like Kymriah and Lyfgenia have shown remarkable success in treating certain types of leukemia and lymphoma. Kymriah, approved by the FDA, is used to treat acute lymphoblastic leukemia (ALL) and certain types of large B-cell lymphoma. Lyfgenia, on the other hand, targets rare genetic disorders. Despite their efficacy, these treatments come with astronomical costs—$475,000 per dose for Kymriah and $3.1 million per dose for Lyfgenia—making them inaccessible to many patients and healthcare systems.
This inaccessibility is further exacerbated by the traditional model of centralized CGT manufacturing, which is often slow, costly, and logistically challenging. The current centralized approach involves extensive personnel and complex logistics, leading to significant delays and reduced treatment efficacy. Consequently, decentralizing the manufacturing process is a critical solution to overcome these barriers.
Challenges Faced by Patients Under the Traditional Model
Patients in rural or economically disadvantaged areas who need CAR-T therapy face similar challenges under the traditional centralized model. The therapy must be produced in a distant, specialized facility, requiring complex logistics for cell transportation. This process can take several months, during which the patient’s condition might deteriorate, reducing the treatment’s efficacy.
With prices reaching hundreds of thousands to millions of dollars per dose, many patients find these treatments financially out of reach, even with insurance. Additionally, the necessity to travel long distances for treatment can impose further financial and physical burdens on patients and their families.
Point-of-Care Manufacturing: A New Paradigm
A revolutionary approach, known as Point-of-Care (POC) manufacturing, is emerging as a solution to the high costs and logistical challenges associated with CGT. This model proposes producing therapies at local clinical centers that already have the necessary infrastructure and expertise, significantly reducing costs and improving access.
“POC manufacturing has the potential to cut costs to one tenth of the cost compared to centralized methods,” explains Boro Dropulić, Executive Director of Caring Cross. “By localizing production, we can also eliminate issues like cross-border payments and make treatments more accessible to patients who cannot travel long distances.”
As Executive Director of Caring Cross, Dropulić is at the forefront of this movement. Caring Cross, a nonprofit based in Gaithersburg, Maryland, is dedicated to accelerating the development of advanced medicines and enabling access to these therapies for all patients, regardless of their location or financial status. Dropulić’s leadership has been pivotal in promoting the adoption of POC manufacturing, which he believes is essential to overcome the financial and logistical barriers that currently limit the availability of life-saving CGT.
“Our goal at Caring Cross is to transform how these therapies are produced and delivered,” says Dropulić. “By implementing POC manufacturing, we can give patients in rural or underserved areas the same access to cutting-edge treatments as those in major urban centers.”
Real-World Success Stories
The feasibility of POC manufacturing is not just theoretical. In Spain, a network has successfully produced CAR-T therapies for approximately $95,000 per treatment, aligning with national reimbursement limits and demonstrating sustainability. Similarly, an Indian clinical center has achieved remarkable cost efficiencies, producing CAR-T therapies for around $35,000, thus expanding access in low- and middle-income countries.
A new collaboration between Caring Cross and Fiocruz, a Brazilian medical foundation supported by the Ministry of Health, aims to establish Brazil as a regional hub for POC CGT production, leveraging technology transfer to enhance affordability and accessibility.
“Our partnership with Fiocruz marks a significant step towards making CGT accessible within the Brazilian public health system,” says Boro Dropulić, Executive Director of Caring Cross. “By localizing production, we aim to reduce costs significantly, benefiting patients across Latin America.”
Addressing Healthcare Disparities
In the United States, disparities in healthcare access persist, particularly among marginalized populations such as Medicare and Medicaid recipients. Current reimbursement limits often fall short of covering the high costs of commercial CGT, hindering access for vulnerable groups. POC manufacturing could bridge these gaps.
“Aligning treatment costs with reimbursement limits under Medicare and Medicaid can make therapies financially viable for these populations,” Dropulić notes. “This approach both enhances access and supports the long-term sustainability of healthcare delivery.”
Building Momentum for Change
As the industry gathers at biotech conferences, discussions are increasingly focused on advancing POC manufacturing, regulatory frameworks, and equitable reimbursement models. These efforts aim to catalyze collaborative initiatives that will shape the future of CGT accessibility.
Recently, Caring Cross announced a partnership with GermFree, a leader in modular cleanroom infrastructure. This collaboration aims to support the development of decentralized POC manufacturing, addressing the exorbitant prices, complex logistics, and long timelines associated with current gene therapies.
A Vision for Equitable Healthcare
The push for POC manufacturing represents a transformative vision for equitable healthcare access. By decentralizing the production of advanced therapies, the goal is to ensure that lifesaving treatments are available to all who need them, regardless of their financial situation or geographic location.
“We are committed to creating a future where cutting-edge medicine is within reach for everyone,” emphasizes Dropulić. “This is about making advanced therapies accessible to all, ensuring that no one is left behind in the quest for better health.”
Boro Dropulić will be discussing these groundbreaking initiatives and the future of accessible gene therapies at several key events in New York this September, including:
Social Innovation Summit – https://unga.
Monday, September 23
5:00pm – 7:00pm ET
Devex Summit – https://pages.devex.com/
September 25 – 1pm – 3pm EST
These platforms will provide an opportunity to explore further the transformative potential of POC manufacturing and its role in revolutionizing healthcare access.
