The expensive new drug is finally available for rare illness – but he fights for two years to get it


When Annie Wilson was only six months old, he was diagnosed with spinal muscular atrophy, a rare condition that causes progressive muscle weakness and impaired physical activity – and told him that he would not be over 3 years old. Over 30 years later, the first treatment of his condition was approved by Biogen Inc.

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and Ionis Pharmaceuticals Inc

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Spinraza. Spinraza has attracted attention to innovation, but it also has a high price tag: $ 750,000 in the first year and $ 375,000 a year. At the time of approval, Wilson had difficulty breathing, talking and even riding his wheelchair and was excited about the possibility of new treatment. But when he first told him he was a good candidate, his doctor, who worked for the California healthcare system Kaiser Permanente, said he was too weak and should return later, Wilson told MarketWatch. Wilson has challenged Kaiser's decision through the Medicare appeal process. Nevertheless, the two-year battle is over, Wilson told MarketWatch in an email. He doubted that stress affects his health, and even considered giving up. "I did not want to die to fight drugs that stop the disease progress," Wilson said. "I feel like denying someone, it's like Kaiser says that your life does not matter to us, but the dollar does." "If I got Spinraza, I'm not expecting a miracle," he said. "Stopping the progression of the disease is what I mean. Maintaining a small movement is most important to our quality of life."

Annie Wilson, 36, from Alameda, California, first received a rare illness from the spine of the spine muscles when she was six months old. He has fought Kaiser Permanent's health system over the past two years to get expensive treatment that can stop the disease progression. See more: Very expensive orphan drugs may "call a storm" Kaiser has "immense empathy" for Wilson and he wants to have support and care, but "we can not recommend a practice that jeopardizes the patient's lack of clinical evidence of patient efficacy "said Dr. Sameer Awsare, Managing Director of Permanente Medical Group. "There are currently no clinical studies that show that Spinraza is effective in respiratory-dependent adults with Spinal Muscular Atrophy, and in some cases there are significant, potentially fatal risks." In recent years, increasingly rare medicines have been adopted, but Wilson's experience speaks of complications of complications that may be promising but expensive treatments far away from patients. Related Articles: Why Health Insurance Do Not Cover This $ 300,000 a Year In Rare Disease? There are about 10,000 to 25,000 individuals in the United States who think SMA is an inherited disorder in the SMA Foundation. Biogean estimates that most of the total population, or about 60%, are adults. Read This: This pricey medication has become Biogen's most viewed product – and could soon be a puzzle game Spinraza was approved in late 2016 for children and adults, but some health insurance companies including Kaiser had to cover adults because Spinraza's only clinical trials had only tested it in children . More adults SMA now has treatment with Spinraza, including Kaiser, Wilson Patient Adviser, Joahn Ginsberg said. "They have to treat her and they have to pay for that care because she is paying every month for Medicare," Ginsberg said. The guidelines, however, vary, and large health insurers such as UnitedHealthcare

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and Cigna Corp.

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which do not have people who require the continuous use of fans. The price is especially a key factor affecting the restrictions on Spinraza access, perhaps because of the rareness of SMA is relatively common. See: The ALS patient group is dissatisfied with the use of $ 115 million by Ice Bucket Challenge. But SMA's adults are treated in the United States, including fans. For example, Stanford University Hospital had had 32 adults since mid-2018, of which 22 were unable to walk, and three of them had respiratory support, and no serious adverse reactions or medical concerns emerged in the letter. a neuromuscular team sent Wilson's doctor to Kaiser earlier this year. The Stanford team also recommends that " [Annie] Bioge has been trying to expand the use of adults and estimates at the end of October that it has reached about 15% in adult patients, compared to about half of all young children and children, and told the MarketWatch that the chairman had told him that he would stop the disease progress and improve his current activities. that Spinraza has "demonstrated significant and clinically significant efficacy … in the broader scope of SMA populations." There are a number of untreated adult patients we believe could benefit from Spinraza. "Jeffrey Capello, CFO of Biogen, said at a telephone conference in late October. But questions about price tags, patient population and side effects are likely to be delayed, and Wilson has time to do it all. "Will she marvelously admire the SMA? No," Ginsberg said. "But we think it can help her?"

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