Saturday , July 24 2021

New expensive medication is finally available for its rare disease – but she has been fighting for two years to get



When Annie Wilson was just six months old, a diagnosis of back muscular atrophy, a rare illness that caused serious muscle weakness and severe movement – and said she would not live over age 3. More 30 years later, the first therapy for his condition was approved at Biogen Inc..

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a Ionis Pharmaceuticals Inc. & s

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Spinraza. Spinraza has attracted attention for being innovative, but so he has a high price tag: up to $ 750,000 in the first year, and $ 375,000 a year later. By the time of approval, Wilson was having trouble breathing, talking and even driving his wheelchair, and the new treatment was exciting. But after she told her that she was a good candidate, her doctor, who works for the Kaiser Permanente health system, California, said she was too weak and she should come back later, Wilson told MarketWatch. Wilson has challenged the Kaiser decision through the Medicare appeals process. However, the two-year battle has been completely, Wilson told MarketWatch in an email. She suspects that the stress affects her health, and she even considered giving her the best. "I did not want to die fighting for a drug that will stop the progression of my disease," said Wilson. "I feel, in refusing someone, I'm just like Kaiser who says your life is not important to us, but the dollar does that." "If I had Spinraza, I'm not expecting a miracle," he said. "What I aim to give is best to follow my disease. Keeping the small movement that I have left is most important to my life of life."

Annie Wilson, 36, from Alameda, California, was first diagnosed with the muscular backache of the rare disease when she was six months old. She has been fighting with the Kaiser Permanente health system over the last two years to access expensive therapy that could prevent the development of the disease. See more: A very rare drug drug can invite a storm of criticism & # 39; Kaiser has a huge "empathy" for Wilson and she wants her help and care but "we can not recommend a treatment course that puts a patient at risk and no clinical studies show her effectiveness to the patient, "said Dr Sameer Awsare, associate executive director of The Permanente Medical Group, in a statement. "There are currently no clinical studies that Spinraza is effective in adults who are dependent on the aircraft with Spin Muscle Atrophy, and in some cases there are potentially fatal risks." More and more rare illness medications have been approved in recent years, but Wilson's experience is talking to the complexities of complications that can keep promising but expensive therapies from patients. Relevant: Why health insurers will not cover this drug of a rare disease of $ 300,000 a year. Around 10,000 to 25,000 individuals in the United States, they are believed to have SMA, who is an inherited disorder, according to the SMA Foundation. Biogen estimates that most of the total population, or about 60%, are adults. Reading: This busy drug has become Biogen's spectacular product – and it could soon be a spectacular trigger being approved at the end of 2016 for children and adults, but some health insurers – including Kaiser – have It has fallen on adults, as Spinraza's clinical trials have just been approved in children. More adults with SMA are now being treated with Spinraza, including Kaiser, advocate Wilson's advocate, says Joahn Ginsberg. "They must treat it, and they need to pay for that treatment, because that's what she is paying in Medicare every month," said Ginsberg. The guides vary, however, and there are large health insurers like UnitedHealthcare

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a Cigna Corp.

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You have policies that will not cover individuals who need continuous use of ventilation. Price, in particular, is a key factor that influences restrictions on Spinraza access, perhaps because rare disease, SMA, is relatively common. See: ALS patient group unhappy with how $ 115 million raised by the Ice Bucket Challenge is spent But adults with SMA are being treated in the US though, including those who are in the United States. depending on aircraft. Stanford University Hospital, for example, had treated 32 adults from mid-2018, of whom 22 were unable to walk and three of them were on respiratory support, and there were no serious side effects or medical concerns, according to a letter that he did Neurosurial team sent Wilson's doctor at Kaiser earlier this year. The Stanford team also recommends "treatment [Annie] Spinraza was reasonable and it would be helpful for them to prevent disease prevention and improve the current function, "according to the letter. Biogen has tried to expand adult use, estimated at the end of October that he had reached around 15% of patients with adults, compared with around half of all babies and pediatric patients. A MarketWatch spokesman said that Spinraza had "demonstrated significant and clinical significance … across a wide range of SMA populations. "We have a large number of patients who are untreated adults who believe that Spinraza could benefit us," Jeffrey Capello, chief financial officer of Biogen, on the late October conference call. But it's like there will be questions about price doc, patient population and side effects similar, and for Wilson, time is everything. "Is it going to improve its SMA miraculously? No, "says Ginsberg." But do we believe he can help him? Yes. "

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