The cell therapy market continues to grow. Estimated at $101 billion in 2021, the global cell therapy market is projected to reach $247 billion by 2028, a CAGR of 13.64%.1 The revenue generation is being driven by technological advancements in manufacturing processes and developments in CAR T-cell therapy for solid tumors, and the approvals and clinical success of Novartis’ Kymriah and Gilead Sciences’ Yescarta are giving encouragement to other developers of cellular immunotherapies.2 In the U.S. this year, the cell and gene industry is anticipated to see its highest annual number of regulatory approvals.3
Recent approvals include Bristol Myers Squibb’s Breyanzi cancer cell therapy, which the FDA cleared for an earlier line of treatment. The product was initially approved last year to treat diffuse large B-cell lymphoma (DLBCL) as a third-line treatment following chemotherapy plus Rituximab and an autologous stem cell transplant. With the new expanded approval, Breyanzi becomes a second-line therapy for patients whose disease has relapsed within 12 months of the initial chemotherapy regimen; — this matches the expanded approval of Yescarta.4
There is no question about the incredible clinical impact cell therapies can have on the lives of patients. However, this impact is the result of innovation that does not equate to a simple, straightforward commercialization process. The commercialization and management requirements of these treatments are just as complex as their innovative science.
Cell therapy manufacturers must think differently about their approach to and corresponding capability investments in commercialization and market readiness. This will require leveraging the latest thinking and best practices from traditional big pharma while also thoughtfully crafting novel approaches and keys to success. The list of “keys to value” is ever growing, and they will require creative thinking along with the application of disciplines from industries not necessarily associated with pharma or healthcare. Below we highlight a few standout keys to value that cell therapy leadership teams will have to navigate in a best-in-class manner despite an arena with a high degree of complexity and uncertainty.
Talk of the promise and clinical success of cell therapies is accompanied by debate and thought leader discussions around the pricing and financing of these novel (and often expensive) treatments. The average price range of currently commercialized therapies is $370,000 to $450,000. When accounting for the total cost of care associated with delivering and managing these treatments, the costs can increase to $750,000 to $1 million per patient.
Cell therapy manufacturers are building the case for these price ranges based on the curative nature of these products and the severe unmet need these treatments are addressing. Demonstrating true value in a real-world setting for these treatments will be critical to provide sufficient evidence that not only supports the pricing expectations, but also provides further clarity on specific patient populations that will respond disproportionately better than other populations. Enhanced real-world monitoring and analytics of cell therapy patients is required to draw the correlative and causative factors that will drive clinical performance. However, this will necessitate technology investments that will span manufacturer, provider, patient, and payer stakeholders.
Another aspect of the pricing and financing discussion relates to how these therapies will be funded and how the expenses will be distributed across payers and providers. Financing mechanisms such as outcomes-based contracts, amortization schedules, re-insurance, and payment pools are in consideration to create an appropriate financial system of payment. Naturally, cell therapy manufacturers will have to collaborate with private insurance companies, hospitals, and state and federal agencies to craft a set of financial approaches that provide the appropriate incentives for all parties while minimizing any financial burden on patients.
To further address patient financial burden, it will also be important for cell therapy companies to thoughtfully craft their patient support and assistance programs in addition to their drug pricing models. One major point of nontechnical differentiation among CAR-T manufacturers is their patient assistance and support offerings, which bring down the overall patient out-of-pocket and payer costs. The OIG offered an opinion indicating cell therapy companies will be exempt from certain anti-kickback regulations to allow manufacturers to pay for or offer expanded patient support to increase patient access by alleviating the financial burden of utilizing their treatments.5
Provider Engagement (But Not What You Think)
Stating that provider engagement is another key to value is a significant understatement in the context of cell therapies. Unlike when dealing with traditional pharmaceutical products, providers need a much greater level of support in order to successfully operationalize and provide cell therapies, and they look to manufacturers for help.
One example of this major difference is onboarding hospitals to be cell-therapy ready. First, a great deal of effort will be required to assess the current capabilities of hospitals interested in using cell therapies by developing a deep understanding of how providers will use each unique therapy. Next, an onboarding process will need to be designed that assists hospitals in preparing to safely steward patients through a complex journey. This journey involves collecting and shipping patients’ cells to the manufacturer; tracking the manufacturing process; receiving, storing, and administering the therapies; and finally, providing post-infusion patient monitoring. It cannot be overstated how important and challenging it will be for cell therapy manufacturers to develop partnerships with providers and deliver the appropriate resources and training across multiple channels (e.g., in person, digital) to support care via cell therapies.
Supply Chain and Operations
Cell therapy manufacturers will need to bring a very different lens and set of capabilities to the supply chain and operations in order to successfully commercialize their products. Orchestrating activities across various stakeholders throughout the process of patient identification, product development, manufacturing, distribution, etc., will require drawing from the leading practices of other industries such as shipping, retail, and consumer packaged goods. The stakes are even higher because there is truly no room for error on late shipments, sub-quality products, or poor execution of treatment protocols.
The answer to these challenges is not simply for each manufacturer to create a comprehensive software/workflow management tool and standard operating procedures their customers can access. Hospitals have already expressed concerns about using different software applications and resources tailored for specific cell therapy manufacturers. The market is signaling a desire for a unified platform of technologies, tools, and operational processes that can be a ubiquitous resource for any cell therapy manufacturer, hospital provider, payer, and patient to access in order to maximize the value and care of using these treatments.
The three keys to value outlined above represent just a few of the requirements for realizing the true potential of cell therapies. Though the challenges are intimidating, they are not insurmountable. Leadership teams of cell therapy manufacturers need to revisit their approach to these issues, including how they define the keys to value; collaborate, design, and construct solutions with all stakeholders in the process; and implement with capabilities that will frequently need to be developed from scratch. Solving these issues will help patients realize the full value of the promise of cell therapy.
1Market Growth Reports. Global Cell Therapy Market Research Report 2022. Available at https://www.marketgrowthreports.com/global-cell-therapy-market-21146132. Accessed July 19, 2022.
2BioSpace. T-cell Therapy Market: Increase in Investment in Adoptive T-cell Transfer Approaches of Disease Treatment Drives the Market. Available at https://www.biospace.com/article/t-cell-therapy-market-increase-in-investment-in-adoptive-t-cell-transfer-approaches-of-disease-treatment-drives-the-market/. Accessed July 19, 2022.
3Genetic Engineering and Biotechnology News. Cell & Gene Therapy Growth Predicts Demand for Biomanufacturing Space. Available at https://www.genengnews.com/topics/bioprocessing/cell-gene-therapy-growth-predicts-demand-for-biomanufacturing-space/. Accessed July 19, 2022
4MedCityNews. BMS cancer drug’s expanded approval gives it an edge over a Gilead cell therapy. Available at https://medcitynews.com/2022/06/bms-cancer-drugs-expanded-approval-gives-it-an-edge-over-a-gilead-cell-therapy/. Accessed July 19, 2022.
5Office of Inspector General. OIG Advisory Opinion No. 19-02. Available at https://oig.hhs.gov/fraud/docs/advisoryopinions/2019/AdvOpn19-02.pdf. Accessed July 19, 2022.