Current Treatment Landscape for Spinal Muscular Atrophy

Ed Pezalla, MD, MPH, chief executive officer of Enlightenment Bioconsult, LLC, provides insight on the drugs currently approved for the treatment of spinal muscular atrophy in the United States.

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Welcome back to PopHealth Perspectives, a conversation with the Population Health Learning Network, where we combine expert commentary and exclusive insight into key issues in population health management, and more. In this episode, Dr Ed Pezalla discusses the current treatment landscape for patients with spinal muscular atrophy.

Hello, I'm Dr Ed Pezalla, I'm CEO of Enlightenment Bioconsult, LLC. I work full-time as a payer strategy and market access consultant to pharma and biotech firms. And prior to that, I was vice president for pharmaceutical policy and strategy at Aetna, where I was responsible for how drugs are reviewed, development of clinical policy, and input from the P&T committee to create formularies and cover decisions for both medical and pharmacy benefits, as well as for Medicare, Medicaid, and commercial business. Prior to that, I was vice president for Clinical Services at Prescription Solutions, a wholly owned subsidiary pharmacy benefit manager of Pacific Care. My training is in pediatrics, public health, and health policy.

At high level, can you describe the current treatment landscape for spinal muscular atrophy?

So, there are currently 3 drugs approved in the United States for spinal muscular atrophy. Nusinersen also known as Spinraza, which was the first drug approved, and is given intrathecally. Zolgensma, which is a gene therapy, and is commonly covered by health plans. And Evrysdi, risdiplam, which is a drug that's given on a regular basis to patients. We do sometimes see combinations of the drugs, including a combination of gene therapy followed by one of the other therapies.

These are all covered by the majority of health plans in the US, of course, there are always outliers for those sorts of things. The health plans generally require a diagnosis of spinal muscular atrophy that has been confirmed with appropriate genetic testing.

And also the type of disease, most patients who are being treated especially with gene therapy, have the earlier onset or type 1, perhaps type 2 features of the disease. There are other features of SMA, which may indicate later disease and those patients generally do not receive all of these therapies, including the gene therapy. The therapies also importantly are covered by Medicaid, which is important because close to 40% of children in the United States will have Medicaid or other public pay programs at some time.

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