Pharma USA 2024

Mar 26, 2024 - Mar 27, 2024, Philadelphia

North America’s ultimate gathering for pharma changemakers

The complexities of designing a digital business model for seamless CGT commercialization

In navigating the era of cell and gene therapy, pharmaceutical firms must strategically engage stakeholders digitally while preserving space for personalized patient care amidst the digital landscape.

With the new era of cell and gene therapy (CGT) upon us, the major challenge facing pharma companies is how to successfully and effectively commercialize their products. To do so, companies should start the commercialization process early, digitally connecting and collaborating with a variety of stakeholders, including treatment centers, health care providers, advocacy groups, regulatory bodies, and importantly, the patients themselves.

But while much of the process will take place in a digital environment, which will help ensure success toward the best patient care, there must still be room for personalization, which many patients want.

Early challenges

First, a big challenge in the ultra-rare disease space involves finding the patients, according to Marie Muniz, Senior Director, Marketing – Gene Therapy, PTC Therapeutics. She suggests using ICD-10 codes to find the patients needing treatment, properly diagnose them, examine their health journeys, and understand any misdiagnosis they may have experienced.

“We can develop algorithms around concomitant diagnoses or misdiagnoses and also different referral patterns within the health care community. So we can understand exactly how these patients might be moving within their health care system,” she explains. “We can target communications and education to both health care professionals (HCPs) and patients themselves around diseases that may be of interest.”

It is possible to identify interventional behavior points where either the HCP or the patients themselves can be educated to learn more about a specific disease. “We're learning a lot from census data, and understanding differences in populations. You can see different trends in different ethnicities or demographic groups. That’s another way we can use data to understand where these patients might be. As a pharmaceutical executive, your goal is to help them as quickly as possible get the treatment that they need,” Muniz adds.

Muniz says when trying to learn more about “previously underrepresented populations,” Big Data can be harnessed to understand patients’ needs, where they are in their health journey, and to learn about the health systems themselves. “When you work with different diseases, you need to understand who is affected by these diseases. You need to go in into the data, understand what's really happening in those communities, understand how to best support these patients, their caregivers, and the GPs,” she says, explaining how it is possible to use data to target a patient down to a street address, and send meaningful communications.  

Part of those communications should include any available options that could help patients and their families pay for a treatment. It’s important that patients and their families understand the financial burden of a disease. Muniz indicates pharma companies should reach out to community HCPs and patient advocacy groups to help communicate with and educate the patients.

Identifying target patients and locating where they are in their health journey are key steps in the commercialization process that should be brought in early in the drug development program to help streamline the interactions that occur in the organization and drive the interconnectedness that needs to be present at launch, according to according to Robert Sexton, Vice President, Program and Alliance Leadership, Mustang Bio. 

He indicates it is possible to use data to help a company establish its market and geographic footprint for a new gene therapy or CAR-T program. Companies also should consider embedding themselves within the local community to improve their understanding of local HCP practices and patients’ lives, he believes.  

Pharma companies should try to determine what type of capabilities, skills, and infrastructure are needed to be built to find patients. “When in the paradigm of drug development do you begin bringing in people for commercialization? When do you start asking the questions? When do you gather insights? It’s really dependent on the operating environment of the organization, and the existing capabilities that you have,” says Sexton.

“How do you plug into this new market that you haven’t interacted with before? That’s a big lift on an operational front. It's a big lift on your sales and account management front. It's a big lift on your supply chain and manufacturing teams because you have individuals working together in a way that they've never worked before,” he adds.

Different business model

“This is a completely different business model,” cautions Muniz. “Many of these treatments are one-time only. So it's imperative that we completely understand the data early on. It’s important that you understand your data sets and how to best support the patient and health care professional for real world outcomes to be as optimized as possible. Digital is the way to do it to ensure success for everybody for the best patient care.”

With cell therapy, a made-to-order patient centric unique manufacturing and delivery process takes place over several weeks, unlike a traditional biologic which occurs over a few years. This requires a change in engagement strategy for commercialization, according to Marissa Cullen, Director of CAR-T Strategic Planning and Commercial Operations for Oncology for Janssen, now Johnson & Johnson Innovative Medicine.

“There is a continuous and frequent flow of information, products, and material going across processes and systems,” says Cullen, who includes manufacturers, treatment centers, and patients involved in a digital approach. “You need to think differently about the people you need sitting at the table with all of the different stakeholders at the treatment sites. And that may seem very simple and intuitive, but I think among large manufacturers, we're not necessarily set up organizationally to do that. How to engage with treatment centers and create a great experience right out of the gate is certainly a consideration,” she adds.

The treatment centers will require significant training and education. “You’re going to have to meet the centers where they want to be. It’s not going to be a one-size-fits-all approach,” she notes.

Optimal interactions

When it comes to digital, says Sexton, it’s important to have a single point-of-contact so that the customer – treatment center – doesn’t have to search for information and can have an optimal interaction. Electronic exchange would streamline the workflow, he believes. Muniz adds that digitization can speed cross-functional communication and facilitate commercialization.

And when determining the type of digital portal a company wants to have in place, management must determine its strategy and how much money it wants to spend. “You have to look at each component that you need to build. You have to look at your capabilities and match your capabilities to the right and best currently available technology,” notes Sexton.

But a company should know whether its customers only want to communicate digitally, or if they might want a more personalized disease management approach, Sexton adds. Cullen agrees, indicating that some people desire to talk on the phone. “They want to have a conversation and hear a human voice at the other end.”

Notes Muniz, “We need to remember that the human connection is very important, and it influences product choice. If you're in a potential competitive environment, that could be something that you consider.”

Keep it simple

Cullen also stresses the need to keep communications, including portals, between manufacturers and the treatment centers simple, as the centers will be busy with other tasks. “I think the centers ideally would love to have one portal for all parties. That’s not a reality right now. For each portal that they log into, they want the functionality and the experience to be simple and intuitive,” she says.

A partnership

Sexton says the relationship between pharma and treatment centers should be looked at as a partnership, not a transaction – a way to learn how they operate and a way they can learn about a product. “We’re moving into a space where they're (the treatment centers) not just ordering a product from you. They’re inviting you into the treatment of their patient. They expect you to be there with them to make sure that patient ends up with a good outcome. That’s a big paradigm shift for us.”

Muniz emphasizes the importance of understanding the nuances between different treatment centers and their individual processes. She suggests making a comparable diagram of what could actually occur with a manufacturer’s base process at each treatment center. “But it's going to be nuanced for each one,” she notes.


For Cullen and the others, just as important for a successful launch is determining how much of a minimal viable product (MVP) is needed, while retaining some flexibility, and figuring out the challenges that will occur in production as well as at the treatment centers, including the enhancements needed to address a treatment center’s external plans and to maintain patient support services. Sexton also emphasizes that manufacturers must have the know-how to closely connect external demand, such as from treatment centers, with internal production capacity. 

Moreover, when planning globally outside of the US, continuous communication is important with colleagues in different geographical areas, Cullen maintains, to help improve the business model and enable it to meet regional specifications and regulations, as well as better communicate with targeted local patient populations.  

Essentially, developing a digitally underpinned business model that makes possible seamless CGT commercialization is a complicated undertaking that requires attention to the smallest of details and connecting with key stakeholders in the process, including the patients themselves.

Pharma USA 2024

Mar 26, 2024 - Mar 27, 2024, Philadelphia

North America’s ultimate gathering for pharma changemakers