Increasing Access to Gene Therapies Requires Innovative Payment Approaches

Joe Nolan

Joe Nolan, general manager of U.S. operations at Bannockburn, Ill.-based AveXis, describes gene therapy as “truly transformational.” It’s on par with medical breakthroughs such as immunotherapies for cancer, he says.

“Gene therapy is actually helping to redefine what’s possible for families affected by rare and devastating diseases, like spinal muscular atrophy,” added Nolan, who co-presented the session "Gene Therapy, Curatives, and Ultra Orphans – How Access and Cost will be Managed," on September 23 at the Pharmaceutical Care Management Association Annual Meeting, in Scottsdale, Arizona.

Specifically, he pointed to Zolgensma (onasemnogene abeparvovec-xioi), which allows AveXis to impact the survival of patients with spinal muscular atrophy (SMA) and the achievement of motor milestones, including sitting and walking.

The National Human Genome Research Institute describes SMA as a group of inherited disorders that cause progressive muscle degeneration and weakness. The most rare incidents of the rare, genetic neuromuscular disease occur among infants and young children. Approximately 500 new SMA cases are diagnosed each year.

Nolan called on the healthcare industry to ensure access to gene therapies “while shifting from the traditional model of life-long, chronic medicines to one-time, potentially curative therapies.”

Managed Healthcare Executive (MHE) recently discussed with Nolan ways to manage the access and cost of gene therapies. 

MHE: Why is it so important to figure out cost and access issues related to gene therapies?

Nolan: It’s essential to determine how access and cost are managed. This will ensure patients who can benefit from gene therapies, such as Zolgensma, are able to access them. It’s equally important to look at gene therapies as a class of new medicines that will require all of us-from the pharmaceutical industry to payers to physicians to hospital systems-to rethink healthcare delivery. We also need to continue to evaluate each new therapy individually and in the context of the medical condition it’s treating.

For example, SMA leads to progressive muscle weakness, paralysis, and, when left untreated in its most severe form (Type 1), death. The disease is caused by a lack of a functional survival motor neuron (SMN1) gene, resulting in the rapid and irreversible loss of motor neurons, affecting all muscle functions, including breathing, swallowing, and basic movement.

As a one-time infusion, Zolgensma provides a functional copy of the human SMN gene and is designed to halt disease progression and provide long-term benefit.

MHE: Tell me about your approach to pricing Zolgensma and why you’ve taken this approach.

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Nolan: We’ve used value-based pricing frameworks to price Zolgensma at approximately 50% less than multiple established benchmarks, including the 10-year current cost of chronic SMA therapy. The current 10-year cost of chronic therapy, which is given over the patient’s lifetime, can often exceed $4 million in just the first ten years of a young child’s life. In addition, that therapy stops working if treatment is stopped. Many insurers currently cover the costs of chronic SMA therapy and would, therefore, realize cost savings over time with Zolgensma.

MHE: Talk about specific efforts you’ve made to increase access and cost.

Nolan: We’ve partnered with the specialty pharmacy Accredo to offer health plans a pay-over-time option up to five years to help ease possible short-term budget constraints, especially for states, small payers, and self-insured employers. We’ve had many payers inquire about this option and nearly every commercial and Medicaid contract currently in place includes pay-over-time as an option.

In addition, because of our belief in the long-term value of Zolgensma, we’re offering payers outcomes-based agreements up to five years to help share the risk of providing coverage for this one-time treatment. We’re including the outcomes-based agreements in most of our commercial and Medicaid contracts.

MHE: Explain what AveXis’ outcome-based approach looks like.

Nolan: The outcome-based offering is grounded in our agreed-upon outcome with the payer. If a patient has a negative outcome during the five-year period, AveXis reimburses a percentage of the cost of the product, all within “best price.” We’re also working with CMS to change the way we look at best price implications going forward. 

MHE: What do you need from payer and provider executives as you manage access to gene therapies and their cost?

Nolan: We need continued partnership to accelerate coverage decisions that will support access for eligible patients. We recognize that FDA approval doesn’t guarantee immediate access to a new treatment and it takes time for plans to put policies in place.

Overall, we’ve been pleased with the response from payers and that most of the insurance policies developed to date are reflective of our label. We continue to work with those that have restrictive policies and those without coverage policies. To facilitate this, we’re educating payers about Zolgensma and our clinical data, as well as the impact of SMA on patients and their families.

Since FDA approval, a wide range of patients with different SMA types, weights, and ages up to two years old, have been approved and treated across many insurance plans, including Medicaid. In just the first two months following FDA approval, more than 20 commercial plans, representing 40% of people with commercial insurance, as well as four Medicaid plans, have coverage policies in place and we’ve made significant progress since then.

Aine Cryts is a writer based in Boston.