Cell and Gene Therapy: Key Payer Considerations

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Truveris’ recent webinar, Payer Strategies for Anticipating and Managing Gene Therapy, outlined the recent development and availability of cell and gene therapies that have created advanced treatment options for patients to manage and even cure rare or previously untreated diseases. However, with costs that can reach millions of dollars, these life-changing therapies have implications that require solution-oriented planning by plan sponsors.

Given the burgeoning pipeline of new gene therapies, it is essential that plan sponsors and their broker partners understand the key clinical, operational, and financial implications of these medical innovations to optimize access and value for both members and organization.

Cell and Gene Therapy Provides Life-Changing Medicines

According to the National Institute of Health, a “rare disease” is one that affects fewer than two hundred thousand people in the United States. Approximately 7,000 rare diseases currently meet this definition, impacting about 30 million Americans.

5,600 of those 7,000 rare diseases have been identified as having a genetic origin. Therefore, the potential exists where gene therapy treatments could directly impact the lives of as many as 24 million people in the United States.

Where previously a patient may have had few or no options to manage or cure their rare genetic disease, extensive development of new gene therapies could provide symptom relief and pathways to cures for millions of people. Dozens of new therapies are currently in development, and the expansion of gene therapy as a category of treatment is here to stay.

Here are the seven gene therapy treatments that are FDA-approved and currently available in the market:


Cell and Gene Therapy


As gene therapies are developed to address a wider variety of conditions, this will not only provide important care for patients, but it will also increase the usage of high-cost medicines. In turn, plan sponsors and their broker partners will need to determine an effective strategy to provide patient access while managing cost.

Assess Your Benefit Program’s Gene Therapy Readiness

Gene therapy is life-changing for patients, but it is also complex and expensive. Proactivity is the best approach for plan sponsors and broker partners as they review their current benefits and look ahead to possible shifts in their coverage needs.

Truveris has identified six areas that will help plan sponsors identify their plan’s potential for gene therapy usage:

    1. Benefit Assessment (Medical vs. Pharmacy)
    2. Contract Assessment (Cost Reimbursements, Rates)
    3. Clinical Assessment (Prior Authorization Use, Ancillary Services Scope)
    4. Utilization Notification Process (Gene Therapy Usage Communication)
    5. Management and Monitoring (Ongoing Treatment and Outcomes)
    6. Affordability/Risk Assessment (Utilization Likelihood, Financial Stability)

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The first five areas for consideration in the checklist above help set the stage for the sixth, which focuses on financial stability and approaches to payment for the gene therapies in question.

Payers have proposed various alternative payment strategies to spread costs over time, including supplemental stop-loss strategies, outcomes-based contracts, installment payments, performance-based annuities, among others.

However, the cost concerns extend beyond the high costs of the therapeutic agents themselves, as additional obstacles can stand in the way of making value-based coverage decisions. These include uncertainty about how long the gene modifications will last, and a limited evidence base to assess the dollar value of gene therapies relative to their clinical benefits.

Given the high stakes decisions around gene therapy for patients as payers, it is key to have an industry partner with clinical expertise and robust data access to help achieve member-centricity and promote access to novel therapies.

Truveris is a leading digital health company focused on delivering truth and clarity in pharmacy. Truveris’ proprietary technology, coupled with deep pharmacy expertise, helps to build a more efficient market that maximizes choice, accessibility and prescription drug affordability. Our solutions provide the insight and knowledge to help people lead healthier and more productive lives.