The most expensive treatment in the world will cost 2 of $ 125,000. What is it?

A child is born. With a fatal diagnosis. And soon, one-time gene replacement cures it in the first weeks of life. Cost? You don’t know. Gene therapy will soon reach important milestones. Tomorrow pharmaceutical giant Novartis expects to receive approval to start that, he said, will be the first “blockbuster” substitutive gene therapy. Blockbuster is any drug that brings in over $ 1 billion in sales per year.

The most expensive treatment in the world

The treatment, called Zolgensma can save children born with spinal muscular atrophy (SMA) type 1, a degenerative disease that usually kills within two years. But it is shockingly expensive, ranging from 1.5 to 2 million dollars. This will be the most expensive disposable drug which is ever sold. On Friday, Novartis received the approval to use the drug and has established a price of 2,125 million dollars.

Gene therapy uses viruses for the introduction of healthy genes into cells of patients, but the growing success of the concept may shake up health care in the United States. The FDA predicts that by 2025, 10 to 20 gene or cell therapies will reach the market every year that may shake the American insurance market, if expensive treatment of hemophilia and muscular dystrophy will receive approval.

“The problem with many of these drugs is that they are expensive, but change the lives of those who need them,” says Michael Sherman, chief medical Director of the insurance company Harvard Pilgrim Health Care, which leads the negotiations with Novartis. “In the case of this and other gene therapies will come, it’s not what you need to say no and how to say “Yes, not bankrupting the system.”

Novartis executives say the launch of the CMA will include the programme of assistance to insurers in payment, including the plan for the reimbursement of the share of the cost of the drug if patients die or end up on the ventilator.

“We would like to pay gradually,” says Sherman, “and stop paying if stops working. In these ever-rising prices, I think it’s fair to ask”.

Sherman said that these new concepts of payment cannot be fully realized unless the insurers agree to accept the unpaid bills of patients at the change of plans. In addition, the rule guarantees that the state public health agencies the “best price” on all drugs, means that Novartis can’t offer a full money back guarantee.

The treatment was developed by a team from Ohio, where scientists and doctors from the National children’s hospital have tested it on children with impressive results. Children were injected trillions of virus carrying a correct copy of the gene, called SMN1. The mutated form of this gene causes loss of motor neurons in the spinal cord.

The scientist, who perfected this idea, Brian Kaspar, a startup AveXis. Last year, Novartis bought it for us $ 8.7 billion. Another scientist, Arthur Burgess from Ohio state University also played an important role in creating the mouse with the AGR. Doctor Jerry Mendell conducted the first study on humans.

Because in the US, only 400 children a year are born with SMA, the acquisition price is guaranteed an incredibly high price for the medication. No one will work for free.

When Zolgensma will have the expected green light, the treatment will be the sixth gene therapy approved in the U.S. or Europe. But some of them can’t find patients. One of the therapy — Glybera — will be removed from the market because it was bought only once. The other Strimvelis — which treats immune deficiency, was used less than a dozen paying customers, partly because it includes a bone marrow transplant. Third — Luxturna — concerns eye disease that affects very few people.

But the treatment of SMA, in contrast to these stories, is already in great demand. To date, 150 patients have undergone gene therapy in clinical trials or special access programs, says David Lennon, President of AveXis. In some cases, treatment was provided to children under the age of six weeks, underscoring how quickly after birth it is possible to correct the genes of the child.

In some States recently added to the AGR in the list of diseases that test to children in hospitals. Because other States will follow the example of Minnesota and Pennsylvania, this will probably add a constant number of new candidates to the drug.

Approval of gene replacement came only three years after another breakthrough, which used a type of genetic drug called antisense. It led to the first highly effective treatment of SMA. This drug Spinraza marketed by Biogen and is worth $ 375,000 a year, but it must be taken continuously, year after year.

It is expected that the two competing treatments will meet in the market. But insurers expect that one-time gene correction might be cheaper in the long run, despite the initial price tag. All the hopes and calculations of parents are based on the assumption that the genetic correction will last. As for now: some children who are treated, now for five years, and they feel good. But there is no guarantee that the treatment will not “fly off” or that don’t need to enter it later.

It is also unknown if it comes someday gene therapy to poor countries, health systems are not able to take care of children with SMA who can’t afford treatment. But Novartis has no specific plans to sell the treatment in the poorest parts of the world.

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