Enabling the Outcome Through Enhancing Patient Accessibility 

Cell and Gene Therapy (CGT) has emerged as one of the most transformative areas in modern medicine, shaping the future of healthcare and changing the face of next-generation therapeutic approaches. Over the years, it has gone by many names. Whether you call it cell and gene therapy, regenerative medicine, advanced therapies, or personalized medicine, the promise remains the same: what was once a distant vision is now a tangible reality. These therapies are providing new hope to patients battling diseases once thought to be incurable. Yet, despite the continued scientific progress, a critical challenge persists in ensuring these therapies are accessible to the patients who need them most. 

This challenge was the focus of the keynote address at the Advanced Therapies Congress in London, delivered by Aruna Mor, Chief Commercial Officer at Cryoport Systems, where she spoke about to the state of patient access within the field of advanced therapeutics. With more than 20 years of experience navigating the CGT landscape, Aruna’s insights offer a valuable perspective on both the strides made and the hurdles still standing in the way of widespread access. Her words called attention to the essential need for a more equitable approach to delivering these life-changing therapies to every patient who can benefit. 

 

A Tale of Two Realities 

Cell and gene therapies represent one of the most exciting frontiers in medical science. From cancer treatments to genetic disorders, the promise these therapies hold for transforming patient lives is undeniable. However, as Aruna pointed out, there exists a gap between the potential these therapies hold and the reality of patient access as it exists today. 

“We have therapies that could change the trajectory of a patient’s life,” she said. “But what value do they hold if they remain out of reach for so many?” 

This question, at the heart of her speech, prompted a reflection on a sobering reality. While these groundbreaking treatments are advancing rapidly in the lab, they are often locked behind barriers that prevent widespread patient access. Many patients, especially those in underserved regions or with complex healthcare needs, still face significant challenges when trying to access these therapies. 

 

The Accessibility Crisis: Why It Matters 

For cell and gene therapies to truly live up to their potential, patients must be able to access them. Not just in developed countries, but globally. Today, however, geographic barriers, lack of awareness, and complex supply chains often stand in the way of patients who could benefit from these life-saving treatments. 

One key issue that Aruna highlighted is the challenge posed by the complexity of these therapies themselves. Each cell and gene therapy is unique, and the processes involved in developing, manufacturing, managing, and delivering these treatments can vary significantly. The lack of standardization in production, management, and distribution adds layers of complexity, making it difficult to ensure that therapies are accessible to all who need them. 

For instance, many cell and gene therapies are highly specialized, and the logistics of transporting them can be fraught with issues. From the collection of a patient’s cells to the delivery of the final product, there are numerous variables that can cause delays or missteps in the supply chain. These delays are not just a matter of inconvenience, they can be the difference between life and death for patients relying on these therapies. 

 

The Importance of Standardization 

One of the most pressing challenges discussed in the keynote was the lack of standardization in the cell and gene therapy supply chain. Unlike more traditional pharmaceuticals, each therapy often requires a bespoke set of processes to ensure that it remains viable from start to finish. As a result, the supply chain for these therapies is highly fragmented, with various players involved in different stages of processing, production, management, and transportation. 

This lack of uniformity results in significant inefficiencies. Therapies that could be lifesaving for patients end up sitting in limbo due to inconsistent quality controls, regulatory requirements, and logistical bottlenecks. As the industry continues to grow, it is essential to develop standardized practices that streamline the supply chain, ensuring that therapies can be delivered in a timely and reliable manner. 

For patients, this means the difference between waiting for an indefinite period for treatment or receiving the care they need without delay. Addressing the variability in the supply chain could significantly reduce the time it takes for patients to access these critical therapies. 

 

A Global Challenge: Reaching the Underserved 

The global market for cell and gene therapies is expected to expand at an incredible rate, with some estimates projecting growth in the addressable market by as much as 207% by 2028. This growth presents a golden opportunity to ensure that these therapies reach patients worldwide, particularly those in low- and middle-income countries where access to advanced medical care is often limited. Yet, despite the optimism surrounding the market, the reality is that patients in these regions are still often left behind. 

In her keynote, Aruna underscored the importance of bridging the gap between high-income and low-income countries. “It’s easy to get caught up in the success stories of large pharmaceutical companies bringing therapies to wealthy markets,” she said. “But we mustn’t forget that millions of patients across the globe are still waiting for access.” 

One way to tackle this challenge is by improving infrastructure in underserved regions, ensuring that there are facilities equipped to store and transport therapies safely. Additionally, increasing awareness among healthcare providers about these therapies and their benefits is critical in overcoming the information barriers that prevent patients from getting the care they deserve. 

 

Sustained Investment and Commercialization: The Path to Widespread Adoption 

Advancing patient access in CGT takes more than just scientific breakthroughs, it requires sustainable investment and commercialization. As Aruna emphasized in her keynote, “Without a clear path to scale, these therapies will remain out of reach for most patients.” The challenge isn’t just developing treatments, but ensuring they can be delivered efficiently, affordably, and globally.  

To attract continued investment, CGT companies must prove their ability to move beyond clinical success into real-world healthcare systems. Commercial viability through streamlined manufacturing, regulatory alignment, and payer adoption is key to making these therapies accessible at scale.  

“Investors and healthcare systems need confidence that CGT isn’t just innovative, but sustainable,” Aruna noted. Without this, progress will stall. By prioritizing commercialization alongside innovation, the CGT industry can move from promise to practice, ensuring that life-changing therapies reach the patients who need them most.  

 

The Path Forward: A Call for Collective Action 

To address these challenges, Aruna called for a unified approach involving multiple stakeholders in the industry. Manufacturers, regulators, healthcare providers, and patients all play a vital role in improving accessibility. But it is clear that the road ahead requires coordinated efforts and a shared commitment to reducing the barriers to access. 

One key area is the need for increased education and training for healthcare providers. Many doctors and clinicians, despite their expertise, are still not fully familiar with the nuances of cell and gene therapies. This knowledge gap is a significant barrier to adoption, as healthcare professionals may not be equipped to navigate the complexities of these treatments. 

Additionally, there is the need for a more patient-centric approach in the development of these therapies. Understanding the unique challenges faced by patients, particularly those in rural or underserved areas, will be crucial to designing therapies that are not only effective but also accessible. By making the patient experience a central focus, the industry can work to eliminate the logistical, financial, and informational hurdles that often prevent patients from receiving life-saving treatments. 

 

A Vision for the Future 

The takeaway from Aruna’s keynote was clear: patient access is the key to unlocking the true potential of cell and gene therapies. While the industry has made tremendous strides, it is only by addressing the barriers to accessibility that these therapies can be fully integrated into the global healthcare system. 

With the market for these therapies poised to grow exponentially, now is the time to act. By standardizing supply chain processes, improving awareness and education, and focusing on patient-centered care, the industry can ensure that these revolutionary therapies are no longer just a promise for the future but a reality for all patients, no matter where they live or what their circumstances may be. 

The journey to making cell and gene therapies universally accessible is long and complex, but the future holds immense promise. As Aruna concluded her address, it became evident that the success of the industry will ultimately be measured by its ability to reach every patient waiting for treatment, transforming patient accessibility from an abstract concept to a tangible reality.