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5 barriers to access for sickle cell disease gene therapies

By Jen Klarer

Partner and head of cell & gene therapy

Sickle cell disease (SCD) is a genetic blood disorder that affects the lives of 100,000 Americans1.

SCD causes abnormal hemoglobin molecules in the red blood cells, resulting in cells forming in a crescent or sickle shape, which can block blood vessels and cause pain, organ damage, and other complications.

This cellular-level disfunction is addressable with gene therapy—a cutting-edge treatment modality that, at the most basic level, corrects how a patient’s cells work. The gene therapy treatment process can involve harvesting a patient’s cells, delivering genetic material so the cells can function properly, and reintroducing them to the patient so that they graft in the bone marrow, for example, and become part of how the body works.

Gene therapy is a promising and potentially life-changing treatment option for SCD; however, patients are likely to face significant barriers to receiving treatment.

Accessing appropriate medical care for rare conditions is often a long and complex journey. Challenges are exacerbated for a gene therapy, and particularly for a Medicaid-dominant patient population characterized by limited resources, such as patient transportation to specialized centers, and insufficient provider site reimbursement. Nearly 66% of the United States sickle cell population obtains medical coverage through Medicaid2. To ensure equitable access, pharmaceutical developers and healthcare providers are presented with the challenge of addressing uniquely steep hurdles to reaching patients in need. As such, gene therapy developers often begin commercial model design and launch planning 4-5 years pre-launch. The first step is understanding what a therapy is up against and how access barriers have or could be addressed.

Traditional treatment methods vs. gene therapies

The main issues with traditional treatment approaches in SCD are adherence, adverse effects of treatment, and ultimately dependence. Traditional SCD treatments involve taking oral pills daily or returning to a treatment center regularly to receive therapeutic infusions. Adhering to oral medication is difficult and infusion-based treatment is often not feasible due to transportation limitations or financial constraints. In turn, many sickle cell patients experience significant pain and acute medical crises (e.g., vaso-occlusion). In response, many patients are prescribed opioids, leading to further health risks.

A transplant may also be considered, but it is not a viable option for most patients. It is difficult to find a donor, and this treatment approach comes with significant risks such as graft vs. host disease and even death.

A treatment alternative with fewer side effects, better outcomes, and less day-to-day effort makes gene therapy an attractive option, both medically and socioeconomically. Therefore, it is important to understand the barriers to gene therapy access to drive appropriate change in healthcare policy and inform strategic commercial model design.

5 barriers to patients accessing gene therapy

1. Payer management with high treatment costs

One major challenge is that gene therapies are high-cost treatment options relative to the standard of care. Due to these high costs, payers look to narrow coverage or limit how much they pay providers offering this product. One example of how this can manifest is payers integrating clinical trial inclusion and exclusion criteria in a coverage policy that exceeds eligibility criteria in the FDA label.

Clinical trials are often designed to be narrower than the potential eligible population to limit variability in outcomes, understand the optimal efficacy of a product, or limit trial participation to patients without alternative options. When the FDA approves a product, the label usually differs from clinical trial inclusion and exclusion criteria because real world situations are much more nuanced. Consequently, payer inclusion of clinical trial criteria set in a coverage policy unnecessarily limits potentially eligible patients covered for treatment. This can challenge or deter prescribing for otherwise eligible patients (e.g., aligned to broader FDA label) who would benefit from treatment, adding burden and delays to the provider workflow.

To minimize this risk, pharmaceutical companies can educate payers on appropriate coverage criteria and payers can leverage provider and publicly available clinical data to develop coverage criteria enabling access to the complete set of patients that would benefit from treatment based on the most recently available data.

2. Lengthy time to coverage

While patients with health insurance through their employer or a commercial insurer have historically received coverage for ex-vivo gene therapies more quickly, Medicaid-covered patients typically have a much longer waiting period before receiving the same treatments: days to weeks for commercially insured vs. weeks to months for Medicaid patients.

Since every state has domain over its Medicaid patient coverage, these patients often experience a fragmented and lengthy process to gain coverage, exacerbated by limited Medicaid resources addressing a wide range of people and population health issues. For example, Medicaid release of coverage criteria can take 6 to 12 months following FDA approval and varies at the state level.

In addition, both commercial and government payers must contract with provider sites individually and agree upon how much they will reimburse for products and services associated with care. This adds an extra step in the coverage and care journey for patients seeking gene therapy and is often a primary driver of delays between patient identification and treatment.

To address coverage process delays, pharmaceutical companies can help provider sites access payer policies and educate on patient coverage criteria to facilitate sharing of complete patient case information with the initial coverage request, while payers can increase clarity of coverage criteria. Additionally, pharmaceutical companies can educate payers and providers on reimbursable steps in the patient journey to include in payer – provider contracts to streamline discussions.

3. Insufficient reimbursement structures

Due to low fixed payment rates of government reimbursement structures, providers can lose money administering cell & gene therapies (CGT) to FFS Medicaid and Medicare patients, particularly in the inpatient setting. Fixed rates for an episode of care are a bundled payment for products and services rendered based on historical standard of care costs (e.g., MS-DRG, AP-DRG). The bundle can take at least 2 years to update in policy. In the interim, New Technology Add-On Payments (NTAP) may be available to close the gap, but support is limited by delays to approval post-launch, brief duration of availability, and unpredictability in CMS perception of new product differentiation.

Bundled payment updates have remained tremendously insufficient and rely on hospitals to request added payments (e.g., outlier payments, NTAP if available) and inflate cost-to-charge ratios to break even. For example, outlier payments have complex request structures and are designed to only reimburse 80% of losses beyond $40,000. Variable hospital experience, sophistication, and comfort with such billing / coding practices can propagate continued financial risk with CGT administration.

In an indication with high government fee-for-service patient volume like SCD and new pricing thresholds, potential for associated payment gaps are likely to be particularly challenging to accommodate. To the provider site, reimbursement gaps can indicate a lack of full coverage, even if a payer publishes product coverage criteria and appears to cover product in a policy. Reimbursement sustainability for a novel product is often most challenging in the first few years on market prior to broader, repeat experience, and can slow adoption & utilization for more financially sensitive provider sites. As such, CGTs are often adopted earliest at large, specialized hospitals with more experience and sophistication managing complex reimbursement.

To address insufficiencies in reimbursement structures, CMS should update government inpatient reimbursement structures to support sufficient reimbursement for this novel class of therapies (e.g., adequately increased DRG multiplier) or mandate carve-out of CGT reimbursement from standard Medicare and Medicaid structures to ensure provider site sustainability of care and equity in patient access. To minimize associated potential for inequities in access, pharmaceutical companies can educate provider sites on billing/coding best practices to support sustainably offering novel products, as well as continue to comment on healthcare policy to support appropriate updates to government reimbursement structures.

4. High logistical costs associated with receiving care

The logistical costs of receiving care are a limiting factor for many SCD patients seeking gene therapies. For example, if a patient does not live near the handful of provider sites that offer an ex-vivo gene therapy, they may need to miss a significant amount of work, fly to the center, stay in a hotel, travel to and from the center regularly, etc. These resource needs (e.g., ~1-2 months away from home, time for complex travel/lodging coordination) can pose extensive financial burden to individuals and families, often making the patient journey and ultimately access feel unattainable. Sickle cell patients are estimated to pay $44,000 in out-of-pocket medical expenses spread over their lifetime3 with the current standard of care, so upfront costs that could total ~$20,000 require alternative sources of affordability support.

Some options exist for patients to receive affordability support through pharmaceutical companies, government payers, and other foundations and advocacy groups today; however, patient and referring provider awareness of available support remains limited. Increased messaging on availability of support and contact information for support can help the treatment journey feel more accessible and less overwhelming.

5. Lack of resources

Competing infrastructure priorities and hospital operational capacity challenges make it increasingly difficult for patients to access gene therapies. Many hospitals are strapped for resources. When an SCD patient receiving gene therapy treatment needs to stay in an inpatient bed for an extended period or needs a specially trained nurse to care for them, they’re not always available.

While there is limited room for external intervention here, provider sites may be able to leverage pharmaceutical company information sharing on target patient population size and anticipated resource needs per patient journey based on clinical trial learnings. These learnings could be shared with provider site leadership to inform infrastructure investment decision-making.

How can the pharmaceutical industry make inroads for access?

One of the first things the pharmaceutical industry can do to enable access to gene therapies is to raise awareness of the structural issues in resourcing, coverage and reimbursement that exist throughout the SCD journey and continue policy commenting to invoke change.

Another way to improve access to care is through strategic commercial model design and launch planning. Given our current healthcare infrastructure does not adequately support these products (e.g., one-time vs. chronic care budgeting and payment) and the coverage journey for gene therapies is uniquely challenging, building pan-stakeholder fluency is essential to getting the novel treatments to the patients who need them the most.

Norstella’s dedicated Cell & Gene Therapy Practice of specialized industry experts can provide critical data and strategic direction to current and emerging pharmaceuticals and biotechs. Our deep expertise in pipeline investment decision-making, go-to-market commercial planning, and lifecycle management strategy, including commercial opportunity assessment, infrastructure needs, order fulfillment operations, and roadmap development, make us the partner pharmaceuticals and biotechs rely on for answers:

•  Learn the access paint points in the treatment journey, support needs, adoption value drivers, and engagement approaches.

• Get precise payer product positioning and messaging.

• Predict payer coverage and reimbursement.

• Profile, prioritize, and activate provider sites with a tailored education strategy.

• Understand stakeholder financial flows and sensitivities to ensure treatment sustainability.

• Optimize distribution and dispensing models, vendor opportunities, and business rules.

• Access the patient services program strategy to address pain points, cross-functional team interdependencies for coordination, and key resources.

It’s time to move your treatment from pipeline to patient. We can help.
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SCD Gene Therapy Access Barriers summary provided by Jen Klarer, Partner and Head of the Cell & Gene Therapy business unit at The Dedham Group, a Norstella Company.


Sources:
1. Data & Statistics on Sickle Cell Disease. Centers for Disease Control and Prevention. Published May 2, 2022. Accessed May 23, 2023. https://www.cdc.gov/ncbddd/sicklecell/data.html
2. Grady A, Fiori A, Patel D, Nysenbaum J. Profile of Medicaid enrollees with sickle cell disease: A high need, high cost population. PLoS One. 2021;16(10):e0257796. doi:10.1371/journal.pone.0257796
3. Researchers identify the high costs of living with sickle cell disease. National Institutes of Health. Published May 16, 2022. Accessed May 23, 2023. https://www.nih.gov/news-events/news-releases/researchers-identify-high-costs-living-sickle-cell-disease

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