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What manufacturers need to know about reimbursement and patient support for cell and gene therapies

By Ana Stojanovska, Myra Reinhardt

An Executive Q&A with Ana Stojanovska and Myra Reinhardt

What will it take to meet the surge of cell and gene therapies (CGTs) with real solutions for reimbursement and patient support? Experts from our commercialization businesses weigh in on what therapy owners need to know as they bring these innovative products to market.

Q: What do manufacturers need to consider regarding reimbursement and payer strategy for cell and gene therapies?

Ana Stojanovska, Vice President, Commercial Consulting, Xcenda: Engage payers and stakeholders early. While their familiarity with CGTs grows as more treatments reach the marketplace, payers as a whole are still new to understanding these unique types of advanced therapies.

Not only is this education needed post-approval, but manufacturers need to collaborate with payers about ultimate coverage and payment decisions much earlier in the development life cycle. They should be specifically asking, and then subsequently building into their phase 2 and phase 3 clinical trials, endpoints that are meaningful to payers and are specifically designed to be used to support the value of their products.

Additionally, current payment systems and reimbursement paradigms are not designed to capture the therapy's value over time. It takes time to evolve current thinking around reimbursement. Payers and government entities may not be ready to do this fast enough to accommodate timing as more of these therapies become commercially available. And, securing an accelerated development designation means there may be even less time for manufacturers to discuss, negotiate, and coordinate payment for the treatment with providers and payers.

This means manufacturers may need to advocate for system changes while working in existing reimbursement paradigms and at the same time pushing for incremental change to maximize patient access.

Q: How are payers currently managing the cost of/reimbursement for cell and gene therapies?

AS: In a survey of payers in Xcenda's Managed Care Network, we discovered that most US payers (62%) currently use traditional techniques, like formulary or utilization management tools. Over the next year, the majority of payers (66%) plan to leverage reinsurance. Payers also said they anticipated increasing their use of value-/outcomes-based contracting (47%) and installment payments (28%) over the next 12 months as techniques to manage CGTs.

Q: How can stakeholders work together to make sure cell and gene therapies reach their full potential?

AS: Manufacturers and payers need to be flexible, agile, and open-minded in considering arrangements that may support improved patient access to these therapies. We anticipate new and traditional payment mechanisms will likely co-exist for quite some time. That said, there are opportunities to advocate for change to modify existing payment and reimbursement methodologies to better account for the long-term value and clinical outcomes over the term of the patient's policy. Ultimately, the evolution and increased use of these alternative payment models may help expand access to these therapies and drive product success.

Xcenda's proprietary market research in the CGT space confirms the need for many stakeholders (payers, providers, manufacturers, patient advocates) to stay well-coordinated in order for a therapy to be most successful. We continue to leverage our unparalleled reach in the US healthcare market to help shape how pharmaceutical-led care is delivered in the CGT space.

And that reach is vast. Xcenda is able to leverage both our partnerships with all manufacturers who have commercialized CGTs and many more owners of pipeline assets, as well as our payer and provider reach via the Global Provider Insights and Managed Care Network. We also rely on industry knowledge from Lash Group's 15 million patients reached and AmerisourceBergen's service to 50,000+ healthcare facilities daily.

Q: Describe the patient journey for CGT. How does patient support differ from traditional therapies/specialty drugs?

Myra Reinhardt, Vice President, Innovation & Analytics, Lash Group: The patient journey for cell and gene therapies can have multiple steps for treatment and can be confusing for those families navigating the process. That's why it's so critical to have a strong case management approach. When the provider identifies the patient as a best fit for these therapies, the patient journey becomes a literal one as they may need to physically travel to an approved site of care. This is where the path diverges for those patients being treated with autologous cell and gene therapies because only certain US centers are trained on the process, preparation, treatment, and ongoing care for these therapies.

At Lash Group, we're focused on the holistic patient approach, but we also plan for how they will gain access to and pay for these therapies. We're working toward understanding the different steps in the manufacturing and treatment process: When is apheresis? How long does it take for a therapy to be ready for the patient? What type of administrative prep work has to happen to prepare them to receive the therapy? When the patient is part of the manufacturing process, as it is with 1:1 cell and gene therapies, the patient support solution must follow the steps to ensure access and timely patient treatment.

Q: Describe the importance of patient support in cell and gene therapies. How/where do you see this evolving?

MR: Select cell and gene therapies have only been approved by the FDA over the past five years, and the industry is learning more about how to support patients every day. I think patient support has a greater role to play as more therapies are approved in this space because there is likely to be a high level of patient interaction throughout the process of delivering therapy. In addition, the treatment administration process can be much longer than a traditional therapy. There are typically weeks or months between when a patient first provides a tissue sample to when the therapy is ready for the patient to receive. Having touchpoints in between and ensuring that if something, like insurance coverage or payer updates, has changed for the patient is critical to their treatment timeline.

Q: What trends are you monitoring in this space? Anything manufacturers should be aware of? Is there anything particularly surprising/shocking?

MR: With so many of these therapies coming to market, standardization and collaboration are going to be critical. We're hearing about standardization from key stakeholders in the process, from the logistics to just how we reduce the degrees of complexity that exist to get patients on therapy. One way pharmaceutical manufacturers of these CGTs will be able to standardize and streamline the process is how they design patient support. You can use a patient support program to provide high-touch care coordination where it may not otherwise exist. And even though what you're seeking is a way to make the processes feel standard and simplified, working with a partner that can put together a tailored, patient-centric program that removes as many barriers as possible for the patient is likely to be essential for these treatments.

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About The Authors

Ana Stojanovska
Vice President, Commercialization Strategy & Access Solutions
Biopharma Services
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Myra Reinhardt
Vice President, Product Innovation
Biopharma Services
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