There’s no denying that cell and gene therapies (CGTs) are shaping the future of medicine and healthcare as we know it. The global CGT market has already grown into the tens of billions of dollars and is projected to expand more than five‑fold over the next decade, driven by a rapidly growing pipeline and an increasing number of approved therapies across oncology, rare diseases, and genetic disorders. These therapies aren’t just transforming treatments; they’re granting patients a second chance at life.
Unlike other therapeutic approaches, cell and gene therapies have the potential to serve as a one‑time treatment, offering benefits that span a patient’s lifetime. This is due to their ability to provide the body with “immunological memory,” meaning that a person’s immune system stores information about a certain stimulus, like a cancer cell, so it can be destroyed if encountered again.For patients and families, the burden often means years of searching for answers, repeated misdiagnoses, few specialists, and even fewer approved treatments. Even when therapies exist, navigating coverage, access programs, and fragmented care teams can be overwhelming.
Chimeric antigen receptor (CAR) T-cell therapy, in particular, has demonstrated remarkable success in treating certain cancer types, but it’s not without its challenges. From diverse toxicities that can be fatal, to its ineffectiveness against all cancer types and concerns surrounding limited persistence, poor tumor infiltration, and immunosuppressive microenvironments, the hurdles are numerous. Accessibility remains a major concern, with only 20% of eligible patients able to access the treatment, largely due to the high cost and lengthy manufacturing process. Until these limitations are addressed, the full potential of CAR T-cell therapy remains untapped.
The Difficult and Complex Journey of Bringing CGTs to Commercialization
One of the biggest obstacles that companies face when developing cell and gene therapies is optimizing their manufacturing. These therapies are often customized to fit each patient’s unique needs, which makes the process complicated, resource‑intensive, and difficult to scale. With so much variability, maintaining control over cell quality is critical. Even the slightest manufacturing error could have severe consequences for patients, necessitating robust in‑process controls and advanced analytical tools to monitor critical quality attributes (CQAs) and ensure strict quality standards are met.
Beyond manufacturing, the delivery of cell-based therapies involves overcoming significant barriers, including supply chain, travel, and logistics as well as vendor and partner collaboration to ensure the product gets to the patient. Companies rely on a robust supply chain equipped with advanced capabilities to ensure the therapy reaches the patient quickly and safely. Once a CGT is produced, it must be administered to the patient safely and effectively, typically through methods like injection or infusion. To achieve this, meticulous planning and coordination are required, involving treatment centers, payers, logistics partners, and sometimes regional or decentralized manufacturing hubs.
Close Collaboration Key for Success
Despite the challenges faced, the promise of cell and gene therapies is too significant to ignore. Overcoming these difficulties requires tight, ongoing collaboration and data sharingsharing among many stakeholders. Each team plays a unique role in the process, and their effective collaboration is crucial for the success of CGT clinical trials and sustainable access after approval:
- Clinicians: Healthcare professionals are on the front lines, administering therapies and monitoring patients for side effects. They are also instrumental in gathering data about the therapy’s effectiveness and safety.
- Manufacturers: The production of CGTs is a complex process, requiring specialized facilities and trained staff. Manufacturers are responsible for producing safe, high-quality therapies.
- Market Access Teams: Once a CGT has been approved, market access teams work to ensure that it is accessible and affordable for patients. They may work with insurance companies, government agencies, and healthcare providers to negotiate pricing and reimbursement.
- Patient Services and Advocates: These individuals and organizations play a crucial role in representing the interests of patients. They ensure that patients’ needs are met and that they are informed about the trial’s progress. They also advocate for patient access to these innovative therapies, support diagnosis and referral pathways, and help patients navigate complex logistics, financial assistance, and long-term follow-up requirements.
To overcome the challenges associated with bringing CGTs to commercialization, several solutions can be applied:
- Improving Clinical Trials: The use of decentralized clinical trials, where patients can participate from their homes, can help increase patient recruitment, especially for rare diseases. Additionally, real-world evidence can be leveraged to complement clinical trial data and provide more insights about the therapy’s long-term effects.
- Optimizing the Manufacturing Process: Innovations in biotechnology are paving the way for more efficient production of CGTs. For instance, automation and closed-system manufacturing can help streamline the production process, reduce the risk of contamination, and maintain consistent quality. Digital batch records, in-line analytics, and AI‑driven process controls can further reduce variability, shorten release timelines, and support scale‑up or scale‑out across multiple sites. Novel analytical tools and technologies can also help monitor critical quality attributes (CQAs) more effectively.
- Enhancing Supply Chain and Orchestration: Advanced logistics and supply chain management solutions are crucial for ensuring the timely and safe delivery of CGTs. Cold chain logistics, which involve maintaining a constant low temperature throughout the shipment process, can help preserve the quality of these therapies. End‑to‑end orchestration platforms that coordinate scheduling, sample collection, manufacturing slots, and infusion appointments are increasingly important for minimizing vein‑to‑vein time and avoiding costly delays.
- Expanding Patient Access Post-Launch: Collaborative efforts between manufacturers, healthcare providers, and payers can help improve access to CGTs. This includes implementing patient support programs, educating healthcare providers about the new therapy, and negotiating with insurance companies for coverage.
- Rigorous Post-Marketing Surveillance: Implementing robust drug safety systems can help closely monitor the safety and efficacy of CGTs after they have been launched. This includes tracking adverse events, collecting patient feedback, and conducting post-marketing studies.
Navigating the High‑Stakes Cell and Gene Therapy Patient Journey
The patient treatment journey in cell and gene therapy is uniquely complex, with individualized products, time‑sensitive logistics, and high‑stakes access decisions.
Understanding the patient journey in CGT is especially critical for rare disease and specialty populations, where every delay or drop‑off represents a profound missed opportunity for patients, caregivers, and brands. This journey is uniquely vulnerable to access barriers—from complex benefit verification and coverage denials, to limited treatment centers and intensive logistical demands that can overwhelm patients and care teams. These challenges make it essential to connect fragmented data, anticipate obstacles before they surface, and ensure timely, coordinated support at each touchpoint in the path to therapy.
The Claritas Rx Difference
At Claritas Rx, we help teams see more of the patient journey by breaking down data silos so manufacturers and patient‑services teams can identify and resolve access barriers before they delay care. Our purpose‑built technology enables tailored support for each patient, from referral through infusion and follow‑up.
We’ve developed integrated tools and workflows specifically to improve access and remove barriers, so manufacturers can see where patients are stalling and act quickly to resolve issues. Claritas Rx helps rare disease and specialty brands remove the barriers that keep patients from accessing and staying on the treatments they need, uniting a complete view of the patient journey with technologies that predict and resolve access challenges before they disrupt care. Our intelligent solutions combine advanced analytics, real‑world data, AI, and in‑line CRM capabilities to increase start and refill rates, reduce abandonment, and strengthen both patient outcomes and brand performance.
Our Patient WatchtowerTM and Patient Services Omnichannel CRM solutions integrate specialty pharmacy, hub, and real‑world data into a single, longitudinal view of each patient’s journey, updated in near real time. Advanced reporting and AI‑driven predictive analytics highlight patients at risk of delays or discontinuation so teams can intervene sooner with targeted support. With patient journey tracking and configurable alerts, customers gain actionable visibility into where every patient is in the treatment process and why they might be stalled.
Our goal is to simplify the complexities of cell and gene therapy access through a single source of truth, enabling real‑time collaboration across manufacturers, patient‑services teams, and channel partners so patients can start and stay on treatment when and where they need it. As CGT pipelines grow and expectations for real‑world evidence increase, Claritas Rx provides the connected data, analytics, and workflow tools needed to manage individualized, high‑cost therapies at scale. With the right insights—and a collaborative partner like Claritas Rx—biopharma brands can accelerate time‑to‑treatment, close critical access gaps, and consistently deliver better outcomes for patients living with rare and complex diseases.
