The next Spinraza injection is scheduled for September 6, and Dobrynya's parents, despite assurances that it will take place on schedule, cannot hide their concern: “The coach said: we will continue to build up strength. As Spinraza is not a one-off treatment it must be administered on a regular basis. Subscribe to BioPharma Dive to get the must-read news & insights in your inbox. If this happens to Spinraza, it will be nonsense, ”says Olga Germanenko. Dobryne, the son of Svetlana and Nikita Rukosuevs from the village of Boguchany, Krasnoyarsk Territory, has already given four Spinraza injections. Discover announcements from companies in your industry. Olga Germanenko, director of the SMA Family Foundation, who was present at the meeting, told RT that the discussion of the drug lasted about an hour and a half: "The discussion was extremely hot, there were many surprises, and it seems that the voting results surprised the commission itself.". Elena Maksimkina, Director of the Department of Pharmaceutical Provision and Regulation of Circulation of Medical Devices of the Ministry of Health, stressed that the conclusion of the commission is only a recommendation, the final decision is made by the Russian government. “Neither I, nor my colleagues will recall a case when, with a positive decision of the commission, the drug was eventually not included in the list. Treatment with Spinraza per quality-adjusted life-years gained cost, including caregiver expenditures, approximately 5.64 million SEK ($625,275) and 3.19 million SEK ($353,638) for infantile-onset and later-onset SMA, respectively, in comparison to the standard of care. The agreement follows closed-door meetings in which the two sides had "commercial discussions" on an agreement that would be ". “The models can be adapted to evaluate the cost-effectiveness of nusinersen in other countries,” the researchers said. It is as if we are waging a large-scale war, and we won one of the battles, although we won by a miracle. A big difference is the list price, which in the U.S. is $125,000 per vial. NICE typically recommends new technologies if their cost is below 20,000 to 30,000 pounds per QALY gained. But this is better than nothing. If the Spinraza drug, which helps with spinal muscular atrophy (SMA), is officially included in the list of essential drugs, from 2021 the manufacturer will fix the price at 5.6 million rubles per ampoule. “Dobrynya has gained strength in his hands, he can push, pull, pinch my finger, quickly raises his hands - and earlier it could take him 10 minutes. “So far, we, like many other parents, after a year of treatment as part of the early access program to Spinraz from the manufacturer, are collecting money to continue therapy,” he told RT. A year has passed since the registration of Spinraza in Russia. The models represent the societal perspective in Sweden, where the study was conducted. I think this was the decisive argument in the voting. Another problem noted by parents of SMA patients: both the updated VED list and the allocation of funds from the increase in the personal income tax rate will start earning from 2021, while children should be treated immediately, emphasizes Yakov Redko, the father of two-year-old Vera. NICE spokesman Phil Ranson said the duration of the limited-time agreement will be revealed when the agency's final appraisal of Spinraza is published. ENROLL NOW LEARN MORE. In the ENDEAR clinical trial (NCT02193074), infantile-onset patients who received Spinraza showed major advances in motor milestones, event-free survival, overall survival, motor function, and motor neuron health. Nonetheless, the difference between NICE's usual cost-effectiveness thresholds and the assessment that Spinraza cost at least 400,000 pounds per QALY suggests Biogen could have offered a lower price. However, in practice, cases when the drug could be obtained without much delay are extremely rare. Opponents of Spinraza's inclusion in the list suggested waiting until two other SMA drugs, Zolgensma and Evrisdi, were registered in Russia (the manufacturers of both submitted the relevant dossiers), compile a global list of patients and write down which drugs are suitable for whom, and also wait for the launch of the fund for financing rare diseases, which President Vladimir Putin spoke about at the end of June. British doctors will be able to prescribe Biogen's spinal muscular atrophy treatment Spinraza following an agreement between the biotech and the UK's cost-effectiveness agency NICE for a time-limited "managed access agreement" that will allow health officials to assess the drug's effectiveness. The first course of therapy costs about 40 million rubles, and about 20 million rubles for each subsequent year. on
Svetlana calls the inclusion of the drug in the VED list “incredibly wonderful and important news”. Spinraza is considered one of the most expensive registered drugs in Russia. Representatives of the manufacturing company reported this at a meeting of the Ministry of Health Commission, during which the medicine was recommended for inclusion in the list. ", "It will fix the price": how much can Spinraza cost if it is included in the list of vital drugs, "The budget should not be reduced": the state will allocate additional funds for the treatment of SMA and other diseases in children.

There is very little information on how these patients spend money on their treatment over the course of a lifetime, data gaps for disease natural history and data on the cost-effectiveness of treatment. In the CHERISH clinical trial (NCT02292537), later-onset patients treated with Spinraza showed improvements in motor abilities. Novartis has argued for a multi-million-dollar price tag for the one-time treatment based on health benefit, but NICE will likely want to give it the same type of scrutiny it's giving Spinraza. Their main measurements were for quality-adjusted life-year — a generic measure of disease burden including both the quality and the quantity of life lived used in economic evaluation to assess the value for money of medical interventions — and how much this increase cost.
The conclusion of the Biogen-NICE talks also hints at what Novartis will face should its SMA gene therapy Zolgensma (onasemnogene abeparvovec) eventually receive approval by European or British health regulators. The free newsletter covering the top industry headlines, By Jacob Bell, Ned Pagliarulo and Ben Fidler •, By Jacob Bell, Jonathan Gardner and Ned Pagliarulo •. But, according to her, there is still a lot of work to be done until 2021, including the development of a rehabilitation program for patients with SMA: "It is necessary to restore not only the musculoskeletal system, but also the respiratory system.". Nevertheless, Spinraza gained reimbursement in Sweden in 2017 for patients with SMA types I–IIIa. The Main Investigation Department of the RF IC for the Krasnoyarsk Territory and the Republic of Khakassia has opened two criminal cases under Art. - He quickly and a lot rolls for a toy, started with five laps, and now he does 16. With regulators, drugmakers and payers pulling in similar directions, real-world evidence is likely to become a more central feature of drug development despite its many shortcomings. The researchers modeled the cost-effectiveness of Spinraza over the course of 40 years in the infantile-onset model and 80 years in the later-onset model. If you wait until it starts working, then several dozen people, scampering through the courts, will irreparably lose their body functions, and some will die. This is particularly applicable to two patient populations, infantile-onset and later-onset SMA patients.

Lumis International GmbH spins off its consulting business to Lumis Life Science Consulting ... U.S. Department of Veterans Affairs Begins National Rollout of WCG’s IRBNet Research Oversig... Saama Appoints William H. Carson, M.D., to Board of Directors, Eli Lilly wins FDA emergency clearance for COVID-19 antibody drug, FDA advisers vote against Biogen's Alzheimer's drug, leaving its future in doubt, BioMarin, stung by FDA rejection, pressed on path forward for 2 key drugs. NICE, or the National Institute for Health and Care Excellence, had preliminarily rejected Spinraza on the basis of cost last year, but continued discussions with Biogen. Antisense therapeutics are used to treat certain types of genetic disorders … Topics covered: Pharma, biotech, FDA, gene therapy, clinical trials, drug pricing and much more. The Russian Ministry of Health recommended including the drug for spinal muscular atrophy (SMA) "Spinraza" (nusinersen) in the list of vital and essential drugs (VED). Twitter. The corresponding decision (nine votes "against", 11 "for") was made at a meeting of the departmental commission on the formation of lists of drugs. Estimated Retail Price* of Spinraza: 1 vial: $125,000/month *Source: Prescription Hope Price for Spinraza: 30 day supply: $50.00/month. It will be possible to reach more patients: every fourth patient will be able to receive the drug only at the expense of this discount, ”explained the director of the“ SMA Families ”foundation.