Sickle Cell Disease Cell & Gene Therapy Model

Erin Lopata | PharmD, MPH

The growing number of approvals for cell and gene therapies (CGTs) has raised concerns about affordability across stakeholders. These groundbreaking products offer an opportunity to change the course of many chronic conditions but do so at a price that can reach up to several million dollars per treatment. Health care payers and employers have recognized the need for innovative approaches to payment for these therapies and have explored various models that mitigate the uncertainty in clinical benefit and durability.¹ Uptake of these models has often been limited by operational complexities and the need for alignment on outcomes metrics and thresholds across stakeholders. As a result, many payers are continuing to manage CGTs via cost containment and utilization management strategies, which can limit patient access.²

The approval of two CGTs for the treatment of sickle cell disease (SCD) places particular pressure on Medicaid payers. Patients with rare genetic diseases, especially pediatric patients, are often covered under Medicaid, and SCD is no exception. It is estimated that about half of patients with SCD are enrolled in Medicaid, with 11% enrolled in Medicare (typically as a dual-eligible).³ Furthermore, certain states have significantly higher numbers of patients with SCD, with 85% of the SCD population residing in 17 states.⁴

With a goal of overcoming many of the current barriers to CGT access, the Centers for Medicare & Medicaid Services (CMS) and the CMS Innovation Center have developed a voluntary Cell and Gene Therapy Access Model to evaluate whether a CMS-led approach to developing and administering outcomes-based agreements (OBAs) for CGTs can improve Medicaid beneficiary access to treatment, health outcomes, and reduce health care expenditures. This model will initially focus on CGTs indicated for SCD, because of the high proportion of patients with SCD enrolled in Medicaid, the high costs associated with SCD treatment, and health care disparities and racial bias that have impacted access to care for patients with SCD.⁵ The model also includes a safe harbor for manufacturers to support fertility preservation services for patients who receive a SCD CGT through the model. This support is critical, as the treatment process for both SCD CGTs involves a course of chemotherapy, which can impair future male and female fertility. Furthermore, the Department of Health and Human Services (HHS) Office of the Inspector General (OIG), recently issued an opinion that while CGT manufacturers are permitted to provide financial aid to patients for treatment-related travel, they would not be permitted provide to financial aid for fertility support. The OIG noted that this ruling does not apply to the CMS model, which highlights the importance of this model in supporting patient access to these services.⁶

While the CMS Cell and Gene Therapy Access Model is launching with a focus on SCD CGTs, it will be important for all CGT manufacturers to closely follow the uptake and outcomes of the model. This is particularly important for CGTs that impact larger patient populations. To date, many payers have been able to absorb the cost of CGTs that are indicated for the rare or ultra-rare populations, but as the number of eligible patients continues to climb across products and indications, many insurers and employers will be looking to limit their exposure to the high cost of these treatments. Centralized models such as the CMS Cell and Gene Therapy Access Model and risk carve-outs will likely become critical tools in maintaining access to CGTs across populations but also could potentially shift the decision-making around CGTs away from the traditional payer access decision-maker. It will be critical for manufacturers to monitor this evolution and to keep a constant pulse on the value drivers for the key decision-makers for CGTs.

In addition to streamlining the OBA process for CGTs, the CMS Cell and Gene Therapy Access Model also seeks to maximize equitable access to gene therapies through funding available for initiatives that target care delivery gaps and access barriers through the patient journey. For manufacturers of CGTs, patient identification and connection to treatment is a critical but complex undertaking. The patient identification starts with ensuring appropriate coordination of care for patients with chronic conditions amenable to gene therapy. Even for patients who are identified as appropriate clinical candidates for gene therapy, the need to travel to a qualified SCD center or lack of resources for fertility preservation present barriers to treatment. It is critical for CGT manufacturers to think holistically about the patient experience and to partner with payer and provider stakeholders to optimize the process. The CMS Cell and Gene Therapy Access Model may provide a roadmap to an approach that can be extrapolated to other CGTs.

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References

1. Phares S, Trusheim M, Emond SK, Pearson SD. Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform. Institute for Clinical and Economic Review/NEWDIGS at Tufts Medical Center; 2024. Accessed September 10, 2024. https://newdigs.tuftsmedicalcenter.org/wp-content/uploads/2024/04/Managing-the-Challenges-of-Paying-for-Gene-Therapy-_-ICER-NEWDIGS-White-Paper-2024_final.pdf
2. Lopata E, Terrone C, Gopalan A. Opportunities and challenges surrounding financial models for high-investment medications: a survey of access decision-makers and employers. J Manag Care Spec Pharm. 2023;29(7):782-790.
3. Centers for Medicare & Medicaid Services. CMS Sickle Cell Disease Action Plan. Centers for Medicare & Medicaid Services. Accessed September 10, 2024. https://www.cms.gov/files/document/sickle-cell-disease-action-plan.pdf
4. NEWDIGS at Tufts Medical Center FoCUS Project. Research Brief 2024F202v058: Emerging Market Solutions for Financing and Reimbursement of Gene Therapy for Sickle Cell Disease: Why Do Payment Innovation? NEWDIGS at Tufts Medical Center; 2024. Accessed September 10, 2024. https://newdigs.tuftsmedicalcenter.org/wp-content/uploads/2024/03/NEWDIGS-SCD-Payment-Innovation-1-2024F202v058.pdf
5. Centers for Medicare & Medicaid Services, Center for Medicare & Medicaid Innovation. Cell & Gene Therapy Access Model: Request for Applications for States. Centers for Medicare & Medicaid Services, Center for Medicare & Medicaid Innovation; 2024. Accessed September 10, 2024. https://www.cms.gov/files/document/cgt-model-rfa.pdf
6. Arnall Golden Gregory LLP, Blank D. OIG issues split decision: travel assistance approved, fertility support under scrutiny. JD Supra. August 1, 2024. Accessed September 10, 2024. https://www.jdsupra.com/legalnews/oig-issues-split-decision-travel-9039451/