Economic Burden of SMA

EP: 1.SMA Prevalence and Disease Burden

EP: 2.Disease-Modifying Therapies Across SMA Treatment Landscape

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EP: 3.Economic Burden of SMA

EP: 4.Approach to Treatment Selection for Patients with SMA

EP: 5.Optimizing Treatment of SMA With Disease-Modifying Agents

EP: 6.The Role of Switching and Combination Therapy in SMA Regimens

EP: 7.Considerations for Payers Covering Combination Treatment of SMA and the Impact of Utilization Management

EP: 8.Evolving Landscape of SMA Treatment

Julie Parsons, M.D.: It’s a bit difficult to discuss the economic burden of spinal muscular atrophy [SMA]. I’m a pediatric neurologist, so I have taken care of babies and children. When I started, about 95% of children diagnosed with spinal muscular atrophy under the age of 2 died. Their lifespans were very short and costly in terms of the medical care provided to them during that time. Now we have the ability to screen and diagnose spinal muscular atrophy at birth.

But the disease-modifying therapies aren’t inexpensive. With Zolgensma being over $2 million for a single IV [intravenous] dose, if a child who’s treated early on would have died or had significant medical needs — multiple hospitalizations, ICU [intensive care unit] stays, oxygen, gastrostomy tube for feeding — and they no longer need that kind of therapy, can we amortize that cost over a child’s life in which they’re able to run, walk, and be fairly typical kids who don’t need the amount of medical intervention that they did prior?

This is true of the other disease-modifying therapies, as well. The initial up-front cost isn’t as high. A standard cost for a patient on risdiplam would be about $340,000 a year. For nusinersen, it’s about $375,000 after their initial loading dose that first year. We’re still looking at a significant ongoing medical cost, but we’ve seen that children who were treated earlier don’t need the extensive medical care that they did when they were declining and had respiratory and nutritional failure. There’s a bit of a disproportionate financial risk on pediatric hospitals, which are charged with treating patients with these very costly medications up front in initiating the therapies.

John Brandsema, M.D.: There’s a significant amount of expertise that goes into attempting to quantify this in a measurable way, and the perspectives on it are valid. But it’s challenging to try to counterbalance something that’s countable — days in hospital, a lumbar puncture procedure that requires procedural support to deliver for a young person, or the cost of frequent visits and laboratory testing post-gene transfer to assess whether there’s a safety event — versus things that are harder to quantify, such as the impact on the overall ability of someone living with SMA to contribute to society, let alone their caregivers and all of the other people involved in the experience of living with this disease. Regardless of the form of SMA that you have, it’s going to limit your ability to function in the world.

The spectrum of severity is also something to consider in this decision-making process. Some people with SMA are still walking and have no breathing problems or difficulties with their eating. Other people are very dependent in all aspects of their care on other people, machines and medical devices to be able to sustain life. You have that whole spectrum of SMA severity to consider when you think about the impact of the treatments. That makes it very complicated. Also, a lot of modeling went into the development of the therapies and where the price points were set for them, and the different aspects of medical care involved in delivering them. At the end of the day, according to their labels, the therapies are indicated for anybody living with a diagnosis of SMA.

Julie Parsons, M.D.: [Our knowledge comes from] the five years that we’ve been able to treat patients with disease-modifying therapies, but this is a very short time, so we can’t always anticipate the future or patients’ additional needs. We need to be cautious about doing a lot of forecasting and predicting as the disease-modifying therapies unfold and we get more experience. The story will be told in terms of other issues that we might not have anticipated that will become more clear over time.

Transript edited for clarity.