A revolutionary drug that could treat a rare and devastating disease is prohibitively expensive. But one state has a plan to pay for its potential $5 million price tag.


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A one-time treatment for a devastating rare disease could be paid for with an installment plan as soon as this summer in Massachusetts.
Novartis’s AveXis unit is involved in the discussions. Its gene therapy could cost up to $5 million per treatment.
Organizers hope the plan will ensure patients can access a potentially life-changing treatment.
Business Insider is the first to report on the discussions and the interest from AveXis.

In recent years, no treatments were even available for the rare, devastating disease known as spinal muscular atrophy.

Now, in a matter of months, an experimental one-time therapy designed to address the disease’s underlying genetic cause could treat the disorder. First though, someone has to pay for its potential multimillion-dollar pricetag.

A new effort is underway in Massachusetts to figure out how to do that. The idea is to let health insurers pay for the treatment over several years. If it succeeds, organizers hope that it could prove to be a viable model for the entire US.

Novartis’s AveXis unit, which makes the gene therapy, Zolgensma, and has suggested a price tag of up to $5 million could be appropriate, is in talks to participate. Business Insider is the first to report both the plan and interest from Novartis’s AveXis.

Americans have long paid for big-ticket items like houses and cars in a similar manner. But the plan — if it is finalized — would mark one of the first such approaches for a medicine. And Novartis would only receive each of its payment if the treatment is effective.

Paying for drugs on an installment plan

“Think of it as installment plan that’s then tied to how well the therapy works. This would be a car loan but you’ve still got to see if the car is going to work,” Mark Trusheim, strategic director of the MIT Center for Biomedical Innovation’s NEWDIGS program, told Business Insider.

NEWDIGS brings organizations together to discuss how the US health system will be able to pay for costly cures, and the Massachusetts initiative came out of that, Trusheim said.

That work has become increasingly important as more gene therapies are likely to become available in coming years for different diseases, according to experts interviewed for this story. Gene therapies are typically administered in a single treatment and can have very high price tags compared to other types of pharmaceuticals. That could impose massive costs and challenges for an unprepared health system

Read more: From the gene therapy that spurred a $9 billion acquisition to a CBD medication for rare types of childhood epilepsy, here are the 12 promising drugs to watch in 2019

Doing the unthinkable, at an exceptional price

Gene therapy is a cutting-edge technology with the potential to cure diseases by tinkering with the body’s genetic material. Drugmakers have cited the value these new products could bring to patients and the medical system to justify their high prices.

Spinal muscular atrophy is a rare genetic condition that affects muscle movement in children and is the leading …read more

Source:: Business Insider

      

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