Recap from the September 2024 Bridging the Gap webinar
Access to life-saving cell and gene therapies is one of the most critical challenges facing healthcare today. While these therapies offer remarkable hope for patients battling life-threatening conditions like cancer and rare genetic diseases, the reality is that only a fraction of eligible patients are actually receiving them.
So, why is that the case?
The answer lies in a complex web of factors - from high costs and limited treatment centers to knowledge gaps among healthcare providers and systemic inequities. This multifaceted issue was at the heart of the discussion during the recent Bridging the Gap webinar, featuring key experts Jacqueline Barry, Ph.D., Chief Clinical Officer at Cell and Gene Therapy Catapult, and Patrick Hanley, Ph.D., from Children’s National Hospital. These thought leaders shed light on the many obstacles in the way of broader access to advanced therapies and explored solutions that can drive real change.
Why Is Access So Limited?
One of the most striking revelations from the webinar was that only 20-30% of eligible patients in both the US and UK currently receive CAR T-cell therapy, a revolutionary treatment that can cure certain blood cancers. As Dr. Barry pointed out, "The barriers are multifaceted, from the limited number of treatment centers to a lack of awareness in rural and underserved communities."
In other words, it’s not just about making these therapies available - it’s about ensuring that people know they exist, know where to get them, and can afford them.
For instance, in both the US and UK, geography plays a huge role in determining whether a patient will receive advanced therapies. Rural areas often lack specialized treatment centers, meaning families must travel long distances, which is a financial and emotional burden many simply can’t manage. Dr. Barry noted that this reality is compounded by the fact that many healthcare providers in these areas aren’t fully aware of these therapies or don’t feel equipped to refer patients to clinical trials or treatment centers.
Breaking Down the Barriers
The issue of access isn't just about geography, though. It's about the entire healthcare system being "ready" for these therapies. Dr. Hanley spoke about the challenges faced by the U.S. healthcare system, where complex reimbursement processes often slow down access even after a patient has been identified as a candidate for treatment. While many community hospitals would like to offer advanced therapies, they lack the infrastructure to administer them.
This is where partnerships come in. Dr. Hanley shared that hospitals are eager to work with larger institutions or pharmaceutical companies like Kite and Pfizer to bring these therapies into more rural areas. "Community hospitals want to provide these therapies but lack the infrastructure. Building partnerships with larger centers can help bridge that gap," he said.
Meanwhile, George Eastwood, Executive Director of the Emily Whitehead Foundation, shared another challenge: the onerous long-term follow-up requirements that patients face. After undergoing a treatment like CAR-T, patients often have to return to the treatment center for regular check-ups, sometimes for as long as 15 years. This can be traumatic and costly, especially for families who live far from the treatment centers. Eastwood explained how his organization is working to decentralize the follow-up process by exploring remote monitoring and decentralized trials, making it easier for patients to stay connected with their healthcare teams.
The Socioeconomic Divide
Another significant issue that was raised during the webinar is the disparity in access for ethnic minorities and lower-income populations. Patients from disadvantaged backgrounds are less likely to be aware of or have access to clinical trials and advanced therapies. In some cases, even if they are aware, language barriers or a lack of trust in the healthcare system may prevent them from pursuing treatment.
Dr. Barry emphasized that the problem goes beyond healthcare infrastructure:
“Patients from minority groups and lower-income areas face multiple barriers - whether it’s limited access to treatment centers, a lack of knowledge about trials, or cultural and language differences.”
These factors contribute to a system where the most vulnerable populations are left out of cutting-edge treatments.
Driving Down Costs
Perhaps one of the most urgent challenges is the cost of cell and gene therapies. While these therapies are often described as "one-time" treatments, their upfront costs can be staggering. Dr. Barry explained that while the value of these therapies is undeniable - especially when you consider their long-term benefits - manufacturing them remains costly. And this cost gets passed on to patients.
But there’s hope. Dr. Barry and her colleagues at CGT Catapult are working to drive down the cost of goods for these therapies by improving manufacturing processes and standardizing certain aspects of production. The goal is to make these treatments not only more affordable but also more accessible on a global scale.
“Driving down the cost of manufacture is critical if we want to make these therapies more accessible globally,” Dr. Barry noted.
Collaboration: The Key to Progress
Ultimately, the solution to the access problem lies in collaboration. Industry, healthcare providers, and policymakers must come together to break down the barriers preventing patients from receiving these life-saving therapies. As Dr. Hanley pointed out, many of these challenges - whether it’s cost, healthcare provider knowledge, or geographic access - are interrelated. Solving one will help solve the others.
This sentiment was echoed throughout the Bridging the Gap webinar, which highlighted that while the field of cell and gene therapy has made tremendous strides, more work needs to be done to ensure that these advances reach the people who need them most.
Key Takeaways
- Limited access: Only 20-30% of eligible patients in the US and UK receive CAR T-cell therapy, highlighting the need for more treatment centers and better referral systems.
- Healthcare provider knowledge: A lack of awareness among healthcare providers, especially in rural areas, limits patient referrals for advanced therapies.
- Patient follow-up: Long-term follow-up requirements can be costly and burdensome for patients, with decentralized trials and remote monitoring as potential solutions.
- Socioeconomic disparities: Patients from lower-income and minority communities face additional barriers, including language and cultural challenges, in accessing advanced therapies.
- Cost of goods: Reducing the high costs of manufacturing is crucial for making cell and gene therapies more affordable and accessible globally.
Future Considerations
If you’re working in the field of cell and gene therapy, or if you’re simply passionate about advancing healthcare, now is the time to focus on equity of access. As the experts in the Bridging the Gap webinar emphasized, addressing these challenges requires not just technological breakthroughs but systemic changes across the entire healthcare ecosystem.
With the right focus, we can move closer to a world where everyone - not just a fortunate few - can benefit from the incredible promise of cell and gene therapies.
Stay updated on the latest challenges and ideas in cell and gene therapy. Register to watch the recorded webinar and be invited to future sessions.
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About Jacqueline Barry, Ph.D.
Jacqueline Barry is a senior business leader with over 20 years of experience in biologics and advanced therapies. Her career has focused on supporting the translation of research from the laboratory bench through to gaining regulatory approval and then clinical delivery. She possesses a blend of expertise across the areas of regulation, quality, Good Manufacturing Practice, science, strategy, commercial development and management.
Jacqueline holds an executive position at the Cell and Gene Therapy Catapult, where she leads a specialist team that is dedicated to developing regulatory and translational strategies for advanced therapy products, as well as working to ensure that the wider ecosystem supports the development and adoption of these therapies in the UK and beyond. She also leads a network of advanced therapy treatment centers in the UK that aim to accelerate patient access to these transformative therapies.
Jacqueline sits on a number of national and international advisory boards. She feels passionate about making advanced therapies more widely available for patients and over her career she has worked closely with industry partners and policy makers to support the adoption of these therapies by healthcare systems.
Prior her time at Cell and Gene Therapy Catapult, Jacqueline worked at the Scottish National Blood Transfusion Service where amongst other activities she designed the Regulatory strategy for the Cellular Therapies for the Blood Transfusion Service and acted as Qualified Person for their release.