The Future of Cell and Gene Therapy: Breaking Barriers to Access and Innovation

With Insights from the October 2024 Bridging the Gap webinar

In the rapidly advancing world of cell and gene therapy, we are witnessing groundbreaking innovations that have the potential to cure once-incurable diseases like cancer and sickle cell. Yet, as these transformative therapies emerge, a troubling reality persists: access to these life-saving treatments remains limited.

Despite the rapid advancements in the field, only a fraction of patients eligible for these therapies are receiving them. As the industry grapples with the logistics of delivering these treatments at scale, experts are pushing to overcome barriers that limit patient access.

The October presentation of Bridging the Gap webinar series offered a fireside chat with Tim Hunt , the CEO of the Alliance for Regenerative Medicine (ARM), and an insightful dialogue with the webinar panel.? The conversation delved into the challenges and triumphs of advancing cell and gene therapies, with a strong focus on patient access, affordability, and innovation.

Why Cell and Gene Therapy Matters More Than Ever

Cell and gene therapies offer a revolutionary approach to treating—and potentially curing—diseases that were previously managed only by treating symptoms. For many patients, especially those facing terminal or life-threatening conditions, these therapies provide the chance for a life-saving intervention. However, the complexity of these treatments, from regulatory hurdles to manufacturing costs, has made widespread access a persistent issue.

Tim Hunt explained that while the science behind these therapies is accelerating, the infrastructure to deliver them isn’t keeping pace.

“These are not your traditional medicines,” Hunt said. “They’re addressing patients with serious conditions, where the status quo represents either death or lifelong disability. We need to ensure the regulatory and healthcare systems can keep up with the pace of these advances.”

For example, CAR T-cell therapies, which re-engineer a patient’s own immune cells to attack cancer, have transformed outcomes for many patients with blood cancers. However, in many cases, only about 20% of eligible patients currently have access to these therapies. The challenge now is ensuring that this number grows as more treatments are approved.

The Cost Conundrum: Balancing Innovation and Affordability

One of the most significant barriers to wider access is cost. Gene and cell therapies often come with price tags in the range of hundreds of thousands, or even millions, of dollars. For some, this upfront cost can be a shock, even though these therapies may offer a one-time, curative treatment. Tom Whitehead, co-founder of the Emily Whitehead Foundation, whose daughter, Emily, was the first pediatric patient to be successfully treated with CAR T-cell therapy, shared personal insight on the financial hurdles families face.

“When Emily was being treated for leukemia, our insurance was billed over $3 million for her care before she received CAR T,” Whitehead said. “But after just 23 days of the CAR T-cell therapy, she was cancer-free. "

"At $473,000, that therapy might sound expensive, but when you compare it to years of failed treatments and hospitalizations, it’s life-saving and cost-effective.”

This issue isn’t unique to one treatment or one country. Health systems across the globe are grappling with how to pay for these therapies without straining public healthcare budgets. Tim Hunt pointed out that while gene therapies can cost upwards of $2-3 million, the overall impact on healthcare spending is smaller than it seems. By 2030, the total cost of all gene therapies is expected to be around $7.5 billion—just 1.3% of prescription drug spending.

But the challenge goes beyond financials. "It's not just about how much these therapies cost," Hunt noted. "It's about making sure they're accessible, sustainable, and that we're prepared for a future where these treatments are more common."

Innovative Approvals and Real-World Impact

In 2023, cell and gene therapies saw a series of breakthroughs. For example, this year’s approvals of TCR (T cell receptor) and TIL (tumor-infiltrating lymphocyte) therapies marked major steps forward in treating solid tumors, which are notoriously difficult to target with traditional therapies. These therapies are expanding the landscape of what’s possible, moving beyond blood cancers to tackle conditions like melanoma and other solid tumors.

While approvals of new therapies continue to make headlines, the process of getting these treatments into hospitals and clinics is far from simple. As Hunt explains, many patients, particularly in the U.S., rely on Medicaid and Medicare, which have struggled to adapt quickly to the demands of gene and cell therapy.

"These therapies are going to mostly impact patients who are on Medicaid and Medicare. We have to find ways to make sure those patients have access,” he said. “If we don't, these approvals won’t mean much for the majority of people who need them.”

The Human Stories Driving Change

Behind every new therapy approval, there are real patients and families who have been profoundly impacted. At the core of efforts to expand access to these treatments is the belief that the stories of patients are as important as the data. Tim Hunt, along with other advocates like Tom Whitehead and George Eastwood , Executive Director of the Emily Whitehead Foundation, emphasized that telling patient stories is essential in driving public support and regulatory progress.

One particularly powerful story is that of Victoria Gray , the first patient with sickle cell disease to receive a CRISPR gene therapy. After a lifetime of pain, she was able to regain control of her health, becoming an inspiration for millions living with the disease.

“She spent her whole life in pain, and now she can be the parent she always wanted to be,” said Whitehead. “That’s the kind of difference these therapies are making.”

These stories help fuel the push for broader access. As Hunt noted, "People make decisions with stories, not just data. We need to keep connecting these incredible breakthroughs to the lives they change."

What Lies Ahead: Access and Collaboration

Looking ahead, the field of cell and gene therapy will continue to grow, with more approvals on the horizon. But as the number of approved therapies rises, so does the urgency to address the infrastructure, regulatory, and financial barriers that stand in the way of patient access.

At the core of this effort is collaboration. Organizations like ARM are working with public and private healthcare systems, as well as policymakers, to develop frameworks that can handle the complexity of these treatments. Meanwhile, advocacy groups like the Emily Whitehead Foundation are continuing to push for better access and support for families navigating the challenges of treatment.

There is hope on the horizon, and the future of medicine is increasingly clear: personalized, life-changing therapies that could one day eliminate the most devastating diseases. But the work is far from over. As Hunt reminded us:

“The science is advancing faster than ever before, but now we need to ensure our healthcare systems can keep up.”

Key Insights from This Webinar

1. Cell and Gene Therapy Revolution: These therapies are breaking new ground, offering cures for diseases like cancer and sickle cell. But access remains limited, and increasing availability is critical.
2. The Cost of Care: While therapies can carry high upfront costs, they can also offer long-term cost savings compared to standard treatments, which can be far more expensive over time.
3. Patient Access: Only 20% of eligible patients receive CAR T-cell therapy, a problem that reflects the need for better healthcare infrastructure and payer support.
4. Patient Stories Matter: Stories like those of Emily Whitehead and Victoria Gray show the transformative power of these therapies and emphasize the importance of expanding access.
5. Collaborative Solutions: Public and private sectors must work together to make these therapies available to the patients who need them, ensuring that the benefits of scientific progress are shared equitably.

The field of cell and gene therapy has come a long way, but the road ahead is still filled with challenges. Addressing those challenges will be key to making sure that life-saving treatments are available to all, not just a fortunate few.

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The article appears on the Azenta Life Sciences blog.

About Tim Hunt

Tim Hunt is the CEO of the Alliance for Regenerative Medicine (ARM), joining in 2022 with over 20 years of biotech experience. Known for fostering corporate culture, Tim leads ARM’s team and advocates globally for the cell and gene therapy sector. Previously, he served as Chief Culture and Corporate Affairs Officer at Xilio Therapeutics and held key roles at Editas Medicine, Cubist Pharmaceuticals, and Biogen. He’s been on the board of Life Science Cares and chaired the Ethics Committee of ASGCT. Tim holds a B.A. from Boston College, a J.D. from Catholic University, and resides in Boston.

Bridging the Gap is a webinar series is produced by Azenta Life Sciences and the Emily Whitehead Foundation . The monthly co-hosts are Olga Bukatova of Azenta Life Sciences and Tom Whitehead of the Emily Whitehead Foundation, along with panelists Patrick Hanley, Ph.D. of Children's National Hospital and Albert Ribickas of Moffitt Cancer Center .

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