Restrictions on $2 Million Drug Highlight Challenge for Gene Therapies
Isaac Olthoff, 10 months old, lacks a gene crucial for muscle control. He can’t sit up and may never be able to stand independently.
A cutting-edge treatment that could prevent his disease from worsening went on sale in May. But at $2.1 million a patient, Zolgensma is the world’s most expensive drug and his insurer has refused to pay for the gene therapy. The treatment for spinal muscular atrophy, or SMA, is posing a dilemma for insurers: It promises to halt the progressive disease in one dose but isn’t yet proven to work in all cases. That uncertainty is making some wary of parting with such a steep sum.
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For Zolgensma, the two main sticking points for insurers are age and the severity of disease. The only clinical-trial evidence so far is limited to children aged six months and under with the most severe form of the disease. Novartis is still testing the treatment in older children with less severe SMA. Zolgensma works by providing a working version of a gene called SMN1 that is crucial for muscle control. Babies with SMA lack that gene, but have varying levels of a backup gene. Those with just one or two copies of the backup gene show symptoms of SMA very young and have the most severe disease. The more copies of the backup gene they have, the later their symptoms kick in and the less severe the disease.
Isaac’s insurer, Aetna Inc., restricts Zolgensma to babies under 9 months of age with the most severe form of SMA. A spokesman said in an email that criteria was “based on the most recent published science, which we continuously evaluate.” Instead, it has agreed to pay for another treatment called Spinraza that works by strengthening the backup gene. But Isaac’s mother Michelle said she would much prefer Zolgensma because it involves just a single infusion. Spinraza is given every four months over the lifetime of the patient. Mrs. Olthoff, who lives in Columbia, Mo., said she was surprised because the FDA in May gave its blessing for Zolgensma to be used in children up to 2 years old, regardless of severity.
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Novartis has defended the drug’s $2.1 million price tag partly on the grounds that it costs less than Spinraza in the long term. The competing drug, a multidose treatment made by Biogen Inc., costs $750,000 for the first year and then $375,000 for each year after that. To allay concerns over the cost, Novartis is offering insurers the option to pay for the treatment in equal annual installments over five years and has pledged to issue partial refunds if the treatment doesn’t work.
Chief Executive Vas Narasimhan said on a recent call with reporters that insurers on the whole were proving willing to pay for Zolgensma, adding that so far plans covering 40% of Americans had agreed to provide it. But he said Novartis was aware of some refusals for patients with less severe forms of the disease. “That’s something we’re going to have to work through,” he said.
https://www.wsj.com/articles/insurers-balk-at-drugs-2-million-price-tag-highlighting-challenge-for-gene-therapies-11564322506 (paid subscription)