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Opportunities and pain points for CGT innovation and market access

By Lung-I Cheng

Cell and gene therapy commercialization
Pulling every lever to advance product development, manufacturing and reimbursement  
Cell and gene therapy commercialization

After a difficult funding year for cell and gene therapies in 2023, optimism is the resounding sentiment among industry stakeholders at the start of the new year. Significant regulatory successes and a healthy pipeline of CGTs from pre-clinical to pre-registration stages pave the way for a promising 2024i.

While there is much optimism, there remains caution over how best to address industry pain points in the evolving landscape. These considerations were reflected at Advanced Therapies Week 2024, with a discussion about initiatives to expand patient access to CGTs by overcoming logistic, regulatory strategy, and commercialization challenges being a central focus. 

The conference – held in Miami from January 16 to 19 -- brought together leaders from all sectors to discuss the various opportunities and pain points with cell and gene therapies across product development, regulatory hurdles, commercialization, and market and patient access. 
Cell and gene therapy commercialization

This article captures some of the key insights colleagues across the industry shared during the event and analyzes what these mean in the broader context of successfully bringing CGTs to patients.

Addressing access limitations 

More initiatives to expand patient access to CGTs are being investigated, such as ways to streamline CGT process development and manufacturing, for example, deploying configurable and customizable solutions to support production or flexible platforms to scale development.  

These initiatives will become of greater importance if the industry is to address challenges around the commercial viability of gene therapy manufacturing. Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the FDA, has warned that without “pulling every lever” with regard to manufacturing advances, these products will become increasingly inaccessible to patientsii.  

The Miami meeting also heard from CAR-T developers, who are trying to enable more CAR-T eligible patients to receive those products. These steps may help to address some reported challenges, such as a survey of CAR-T centers in the US showing only 25 percent of patients receiving the therapy, with patients on wait lists for a median time of 6 monthsiii. Finding ways to address access barriers remains a key priority for industry.  

Data-driven decision-making 

The role of data management cannot be overlooked when discussing CGTs. While the sheer volume of data associated with CGTs poses significant challenges, the ability to harness the data generated throughout the development and delivery processes is crucial for success. 

Data management involves collecting and storing information while ensuring its integrity, accessibility, and usability. Robust systems and infrastructure are necessary to handle diverse datasets such as genomic sequencing, patient demographics, treatment protocols, and long-term follow-up. 

Integration of these diverse datasets is equally important. By consolidating data into a unified platform or ecosystem, comprehensive insights can be gained to drive informed decision-making. Integration allows for trend analysis, pattern identification, treatment evaluation, and real-time feedback optimization. 

Leveraging digital technologies like artificial intelligence, machine learning, and predictive analytics enhances data analysis capabilities. These tools efficiently extract meaningful insights from complex datasets and identify potential optimization opportunities. For example, AI can be used for target identification with CGTs to identify the right patients for clinical trials, and to track long-term patient outcomesiv

By effectively managing and integrating data throughout the CGT lifecycle, innovation can be accelerated while maintaining patient safety, and thus support decision-making.  

Demonstrating value to payers 

Determining an appropriate price for CGTs involves balancing affordability for patients with ensuring sustainable business models for manufacturers. It requires a deep understanding of the value these therapies bring to patients' lives, as well as an ability to demonstrate that value to payers. 

Integral to this value proposition is demonstrating the safety, efficacy, and cost-effectiveness of CGTs through robust clinical data. However, capturing this evidence can be complex due to factors such as limited patient populations and long-term follow-up requirements. 

Value demonstration goes beyond clinical outcomes and involves quantifying the broader impact of CGTs on healthcare systems, patient quality of life, and societal benefits. Communicating this value effectively to payers is vital in gaining their support for reimbursement. 

Recent developments in Europe could pave the way for a more streamlined process for bringing innovative medicinal products to patients and achieving equal access. The Joint Clinical Assessment (JCA) centralizes the health technology assessment of clinical evidence of new products, potentially addressing issues such as the access delay caused by differing evidence requirements across the EU. This will soon be relevant to CGT innovators, with the JCA process becoming mandatory for oncology drugs and advanced therapy medicinal products (ATMPs) from January 2025. 

Working with providers 

Additionally, successful commercialization of CGTs relies heavily on strong collaboration with healthcare providers, starting from the early stages of development. Building trust and establishing collaborative partnerships early on can enhance patient access to these life-changing therapies.  

By keeping the patient at the center of every decision and involving providers early on, biotech and pharma companies can tap into their expertise and insights, ensuring that CGTs are developed to meet the needs of both patients and healthcare systems. 

Providers play a vital role in patient access to CGTs, from diagnosis to treatment delivery and follow-up care. By collaborating with these crucial stakeholders, industry can streamline processes, address logistical challenges, and ultimately enhance patient access to these life-changing therapies. Indeed, ensuring patients have access to treatments and are properly informed about CGTs means prioritizing their support services, including healthcare providers.   

Furthermore, provider collaboration offers an opportunity for valuable feedback and real-world data collection. It gives manufacturers insights into the practical implementation of CGTs in different clinical settings, helping to refine treatment protocols and optimize patient outcomes. 

Patients at the center 

CGT is an increasingly competitive field, and while only 3 percent of CGTs being researched have been approved, nearly 4,000 clinical trials are ongoing as of the fourth quarter of 2023.  

With so many CGTs being investigated for underserved patient populations, there is a need for collaboration across the industry to tackle the challenges of optimizing market access to these innovative products. Moving the needle forward in this exciting area will take a village, with all stakeholders working together to bring more therapies into commercialization and the hands of patients.  

There are also demonstrable efforts across the industry to raise the bar in all areas of CGT innovation. That was reflected in the Advanced Therapies Week awards ceremony, with a growing number of categories and more participantsv. We were honored to be nominated in two categories. The first is the Sustainability Initiative of the Year Award, which recognizes sustainable development in the CGT industry through innovative and sustainable use of resources and prioritizes the needs of the wider environment and society. The second is the Patient Advocacy Award, which awards organizations that foster awareness for CGTs and support those going through treatment – an award that goes to the heart of our commitment to helping developers overcome challenges and finding ways to simplify the path to patient access.  

While the industry has faced many hurdles with development and market access, the Advanced Therapies Week meeting sounded a hopeful note for 2024. There have been bumps along the way, but there is growing consensus that the science is strong. CGT research is moving into more modalities, including autoimmune diseases, and gene therapies are set to reach a larger patient population with approvals for new treatments for rare diseasesvi.  Indeed, in 2023, the FDA approved seven CGTs, including the first to treat sickle cell disease, providing more reason for optimismvii.  


The contents of this article may contain marketing statements and do not contain any legal advice. Cencora strongly encourages readers to review the references provided with this article and all available information related to the topics mentioned herein and to rely on their own experience and expertise in making decisions related thereto.

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About The Author

Lung-I Cheng
Vice President, Cell and Gene Therapy
Biopharma Services
View Bio