A baby is born. With a fatal diagnosis. And soon a one-time gene replacement treats it in the first weeks of life. Cost? You better not know. Gene therapy will soon reach an important milestone. Tomorrow, the pharmaceutical giant Novartis expects to receive approval for the launch of what, according to him, will become the first "blockbuster" of gene replacement therapy. A blockbuster is any drug that brings more than $ 1 billion in sales per year.
The most expensive treatment in the world
A treatment called Zolgensma can save children born with spinal muscular atrophy (SMA) type 1, a degenerative disease thatusually kills within two years. But it is also shockingly expensive: from 1.5 to 2 million dollars. It will be the most expensive single-use drug ever sold. On Friday, Novartis received approval for the use of the drug and set the price at 2.125 million dollars.
Gene therapy uses viruses to injecthealthy genes in patient cells, but the growing success of the concept may shake the US healthcare industry. The FDA predicts that by 2025, between 10 and 20 gene or cell therapies will enter the market each year, which could shake the American insurance market if expensive treatments for hemophilia and muscular dystrophy are approved.
"The problem with many of these drugs isthat they are expensive, but they are changing the lives of those who need them, ”says Michael Sherman, chief medical director of the insurance company Harvard Pilgrim Health Care, which is negotiating with Novartis. “In the case of this and other gene therapies that will come, the point is not what to say no, but how to say yes, it does not bankrupt the system.”
Novartis executives say launchthe medication from AGR will include programs to help insurers pay, including a plan to reimburse a fraction of the cost of the drug, if patients die or end up on the ventilator.
“We would like to pay out gradually,” says Sherman, “and stop paying if it stops working. With such constantly rising prices, I think it’s fair to ask. ”
Sherman says such new payment conceptscannot be fully implemented until the insurers agree to accept unpaid patient bills when changing plans. In addition, the rule that guarantees the "best price" for all drugs to state health agencies means that Novartis cannot 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 tested it on children with impressive results. Children were injected with trillions of viruses carrying the correct copy of a gene called SMN1. The mutated form of this gene causes a loss of motor neurons in the spinal cord.
The scientist who refined this idea, BrianKaspar, created a startup AveXis. Last year, Novartis bought it for $ 8.7 billion. Another scientist, Arthur Burges of Ohio State University, also played an important role by creating a mouse with AGR. Doctor Gerry Mendell conducted the first studies in humans.
Since in the US only 400 children per year are born with AGR, the purchase price guaranteed an incredibly high price for the drug. No one will work for free.
When Zolgensma receives the expected green light,treatment will be the sixth gene therapy approved in the USA or Europe. But some of them cannot find patients. One of the therapies - Glybera - will be removed from the market because it was bought only once. The other, Strimvelis, which treats immunodeficiency, has been used by less than a dozen paying customers, in part because it involves a bone marrow transplant. The third, Luxturna, is an eye disease that affects very few people.
But the treatment of SMA, unlike these stories, is alreadyis in great demand. To date, 150 patients have undergone gene therapy in clinical trials or under special access programs, says David Lennon, president of AveXis. In some cases, treatment is given to children as young as six weeks, which emphasizes how quickly the genes of the child can be corrected after birth.
Some states have recently added SMA to the list.diseases that check children in maternity homes. As other states follow the lead of Minnesota and Pennsylvania, this will likely add a constant number of new candidates for the drug.
Gene replacement approval came in only threeyears after another breakthrough, in which a type of genetic drug called antisense was used. He led to the first highly effective treatment of SMA. This drug Spinraza is sold by Biogen and costs $ 375,000 a year, but it must be taken all the time, year after year.
It is expected that two competing procedureswill meet in the market. But insurers expect that a one-time gene correction may be cheaper in the long run, despite the initial price tag. All hopes and calculations of the parents are based on the assumption that the genetic correction will last for a long time. So far this is true: some children who have been treated are now five years old, and they feel good. But there are no guarantees that the treatment will not “fly off” or that it will not be necessary to administer the drug later.
It is also not known whether gene will evertherapy to poor countries whose medical systems are unable to care for children with SMA who cannot afford treatment. But Novartis has no specific plans to sell treatment in the poorest parts of the world.
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