NewYork-Presbyterian Morgan Stanley Children's Hospital is one of only a handful of New York State–designated SCID Specialty Care Centers. Both NewYork-Presbyterian Morgan Stanley Children's Hospital and NewYork-Presbyterian Komansky Children's Hospital have specialists on staff who are experienced in making referrals for newborns who test positive for SCID. However, many newborns who test positive during screening do not actually have the disorder, and must undergo advanced testing to confirm the diagnosis. About Our ProgramĪll infants born in New York State are screened for SCID. NewYork-Presbyterian Morgan Stanley Children's Hospital has a designated SCID Specialty Care Center where children with this disorder can receive all the care they need through one convenient, coordinated program. They often experience recurrent illnesses and fail to thrive. Because they're born with little or no immunity, children with SCID are susceptible to potentially life-threatening infections. “We hope that Strimvelis will be the first of a number of innovative gene-therapy medicines that we will bring to patients.” The company recognizes, she adds, “that the industry will need to adapt the way in which medicines are priced and funded.Children with the rare genetic disorder severe combined immunodeficiency (SCID) have few or no T cells, the white blood cells needed to fight infections. “We do not expect to recover all of the costs of building a platform to deliver gene and cell therapy from Strimvelis alone,” says Anna Padula, a spokesperson for GSK’s rare-diseases group. It’s also working with a small company, Adaptimmune, to genetically alter immune cells to battle cancer. Instead, it sees the treatment as a way to help patients and gain experience with treatments involving cells and genes. GSK says it won’t make much money off Strimvelis. We need a new model for ultra-rare disorders, because we are going to develop these treatments.” Reilly says money-back guarantees and pay-as-you-go schemes are two ways to make high sticker prices palatable.
“But there are hundreds if not thousands of disorders that fall into this category. “Treating 12 kids a year-it’s just not commercially viable, at any price,” says Phil Reilly, a partner at Third Rock Ventures in Boston, who invests in gene-therapy companies. Treating all of them would generate about $8 million in revenue-barely a blip for GSK, which sells $30 billion worth of drugs a year. Only about a dozen children are born with ADA-SCID each year in Europe. Based on experience gained so far, GSK might end up refunding about one in six treatments.īut the big question isn’t whether gene therapy costs too much-it’s whether companies can make any money at it, especially treating ultra-rare diseases. Patients receiving GSK’s gene therapy will also be tracked in a registry, the company confirmed. It maintains 135 patient registries to track how well they work and Pani says Italy has collected more than 250 million euros in refunds. The Italian agency is unusual in that it already imposes pay-for-performance rules on some cancer drugs. But potential cures for hemophilia, a rare eye disease, and a fatal brain illness could reach the market next, and they could be similarly expensive. Strimvelis is the first treatment to be commercialized that lives up to the promise. The idea behind gene therapy is that a one-time correction to a patient’s DNA will lead to a lifelong cure. “It’s a symbol of the future, absolutely.” “It’s really difficult, because everyone’s models involve regular drugs and these are not regular drugs,” says Pani. GSK declined to comment on the negotiations. But AIFA was in a strong negotiating position because the therapy had been developed in Italy with charitable donations. “My initial gut feeling was ‘Hmmm, that’s really surprising.’”Īccording to Pani, GSK approached the Italian agency with a price “nearly double” what was eventually settled on-or of about $1 million.
“I was expecting a higher price,” says Christian Hill, managing director with Map Biopharma, a consultancy in Cambridge, U.K. The expense of these drugs and the care needed for a sick child quickly add up to millions. Some other patients get treated with enzyme injections that cost $250,000 a year. The cost of a bone marrow transplant from another person-the established way to treat ADA-SCID-can reach $1 million. By some measures, Strimvelis’s price counts as a bargain.
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