How to Navigate Cell and Gene Therapy Reimbursement: Part Two
By Ana Stojanovska |
A two-part Q&A on challenges and opportunities related to reimbursement, coding and coverage for cell and gene therapies.
Q: Data seems like it will play an increasingly important role in securing reimbursement. What can C> innovators do to ensure they have access to the right data?
Ana Stojanovska: Health outcomes data, and the ability to collect and track such data, is critically important for cell and gene innovators. Many innovative therapies are associated with high costs, small patient populations and uncertainty in long-term outcomes, all factors that cause payers to question the value of a therapy.
Whether it is having a limited data set at launch, relevant comparator data, durability of response or long-term safety data, these therapies may enter the market and launch before they have all data available. Ultimately, this can present market access challenges. Manufacturers need to have a process and plan for collecting this data pre- and post-launch in order to address the unique challenges their therapies face from an insurer/payment perspective.
For example, new cell and gene therapies that have launched in the U.S. have coupled their launch announcement with outcomes-based payment messages – while limited in detail – to proactively address some of these challenges. One gene therapy in particular, LUXTURNA, was launched by Spark Therapeutics with at least three proposed payment models to ease the financial burden and facilitate patient access, including:1
- Outcomes-based rebate arrangement to tie demonstrable patient success with product price. With Harvard Pilgrim Health Care, Spark Therapeutics agreed to pay rebates if patient outcomes fail to meet pre-specified, measurable thresholds both in the short term (30 to 90 days) and long term (30 months).
- Contracting model to minimize financial exposure of buy-and-bill process for treatment centers. The PBM buys LUXTURNA instead of hospitals. The payer then may need to agree to provide coverage for its population consistent with LUXTURNA’s FDA labeling, expedite benefits processing and cap patient out-of-pocket amounts at in-network limits. Further, the payer would negotiate payment with the treatment center that is “commensurate with the type of specialized medical care required to deliver LUXTURNA.”
- Installment payment proposal for CMS to conduct a demonstration project that would allow the manufacturer to offer public and private payers to use an installment payment option and receive greater rebates based on patient outcomes. This is an approach Spark Therapeutics maintains is “not feasible” under current government price-reporting requirements.
All of these innovative payment arrangements require manufacturers to have the means to collect and convey necessary outcomes data to affected stakeholders. Ideally, manufacturers can facilitate this process across the continuum, from the data collection method design to the analysis of the data, to make it actionable and applicable to all stakeholders.
Q: Do you anticipate there will be an industry-wide shift toward a pay-for-performance model? For example, one option in such a model would be to include a sliding scale, whereby instead of reimbursing based on response or lack thereof, factors like the depth of response and side effects could be taken into consideration with the level of reimbursement.
AS: While there has been significant and continuous growth toward pay-for-outcomes, in my opinion the current fee-for-service system is here to stay, at least in part, for the foreseeable future. There are issues that impact wholesale changes in reimbursement methodologies, and options like payer-by-payer pilots or carve-out benefits are more likely in the future.
For example, Medicaid “best price” is a big impediment to value-based payments, and any change likely needs legislation. Recently, there were additional calls for addressing best price, including:
- The National Pharmaceutical Council’s white paper, “Regulatory Barriers Impair Alignment of Biopharmaceutical Price and Value”2
- ICER, in its review of Spark’s LUXTURNA, specifically calling on CMS to “take steps to permit private payers to use innovative payment mechanisms without triggering Medicaid Best Price constraint”
Additionally, existing claims or EMR systems may not always allow adequate reporting of the types of outcomes that would/could be required.
"Manufacturers need to be able to show a detailed patient journey that encompasses multiple layers, including clinical pathways, reimbursement, financial and logistics."
Q: What’s needed to develop a long-term reimbursement model for innovative therapies?
AS: Manufacturers need to be able to show a detailed patient journey that encompasses multiple layers, including clinical pathways, reimbursement, financial and logistics that will all work together to help ensure seamless patient access. Simultaneously, stakeholders need to be flexible around potential changes to legislation and reimbursement. There will need to be an evolution in healthcare business models, because they are still too fragmented to distribute value and reimbursement appropriately. Data remains crucial, and aggregated data systems allowing sharing of data between stakeholders and use of registries to track longer-term outcomes will be important in proving product value long-term.
Q: How can innovators find support and collaboration to help them navigate potential barriers?
AS: Cell and gene therapies often start and end with the patient. However, given the large number of stakeholders that are involved, and the nuanced coordination that may be needed between them, it is easy for patients to get lost in the process and feel confused about their treatment journey. By putting the patient at the center, innovators can strategically plan for the commercial success of their therapies.
For example, multi- and cross-functional workgroups inclusive of commercialization partners, health economics, market access and reimbursement experts, COE, payer, integrated delivery network (IDN), hospital, patient advocacy and other key stakeholders, can help innovators proactively understand the patient journey from a clinical, reimbursement and care-coordination standpoint. As a result, innovators can design the types of patient experiences they want for their unique products.
Conducting such strategic planning as early as possible, often at least two to three years prior to launch, helps with the development and ultimate deployment of a successful, integrated commercialization strategy. As part of this strategy, manufacturers will need to understand the existing access and affordability landscape for each product and patient population, redefine the value of these therapies for payers and providers, collect appropriate resource utilization data to help secure new billing codes and ensure that the appropriate clinical and outcomes measures can be captured in clinical or health economics study programs. When preparing to go to market, designing and market testing risk-sharing or outcomes-based payment approaches will be critical. Ultimately, all stakeholders will need to be educated on the new, complex models and manufacturers will need to ensure they dedicate the appropriate resources to help providers navigate through insurance challenges to get appropriately reimbursed for administering these treatments.