It’s a one time treatment that, at least as far as we know, provides a lifetime cure. Moving who pays the costs around doesn’t change that. According to GlobalData’s recent report, Spinal Muscular Atrophy (SMA): Opportunity Analysis and Forecasts to 2028, the SMA market is expected to grow at a compound annual growth rate (CAGR) of 16% to reach a global value of $6.6 billion in 2028. "We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time," said Novartis CEO Vas Narasimhan. Novartis’ Zolgensma was approved by the US FDA in May 2019 for treatment of spinal muscular atrophy (SMA) in pediatric patients less than two years of age.
Long-term value.. But if it costs $2 billion to develop a drug to treat 250 kids a year then that’s just what it costs to develop the drug. Zolgensma’s challenge of finding favour with a majority of the insurance companies in the US provides a sneak peek of the reimbursement challenges that are likely to face transformative gene therapies, which are expected to launch at higher price points than traditional treatment approaches. NICE (who are the UK gatekeepers in this area) reckons these things in QALYs (quality-adjusted life-years). Although specialty drugs such as Zolgensma are taken by a small portion of the population, their costs can ripple beyond an individual family or insurer. But we’ve got to allocate the total costs of being able to treat these kids to, well, to treating these kids. Though since the Indians can make a good thing out of reverse-engineering American etc drugs, the wicked Yanks insist that they can’t patent their drugs in the US. I think that would qualify as cost effective. Zolgensma is a one-time therapy that uses a virus to deliver a copy of human SMN gene to prevent the death of motor neurons. But the cost of the therapy likely will trigger debates about the escalating costs of prescription drugs and medical technology. Thanks to a French nonprofit's reasonable pricing clause, it may be much cheaper in France.
For example, while Spinraza from Biogen was approved for use in the EU in 2017, the National Institute for Health and Care Excellence (NICE) did not recommend its use through the National Health Service (NHS) for SMA patients in England, UK until 2019. About one in 11,000 babies are born with spinal muscular atrophy. It’s a one time treatment that, at least as far as we know, provides a lifetime cure. The Food and Drug Administration on Friday approved a $2.125 million gene therapy for a rare disease called spinal muscular atrophy in infants and toddlers. That $8 billion being a useful guide to how much it’s worth to buy the one that really does work instead of having to pay for the whole development process of those that don’t. In that sense, Zolgensma can expect to tread a similar path in such countries. The Institute for Clinical and Economic Review, which analyzes the cost and effectiveness of drugs, said on Friday updated its "value-based pricing" estimate for the drug to a range of $1.2 million to $2.1 million. Targets the genetic root cause of SMA. Then anyone can make this same drug and the price will plummet – because that’s the way our current system allocates the cost of the drug development. For parents of babies born with SMA, any price is worth paying to save the child’s life. The treatment of that quarter thousand of babbies each year has to carry the costs of developing the drug used to treat them. No, obviously, we’re not saying that we attach their Fisher Price mobiles with a lien until they cough up the milk phlegm to pay for it. The condition is often fatal or may require children to use breathing masks or machines. Zolgensma is expected to be the market leader in 2028 with peak year global sales of $3.1 billion. The first two years of treatment with Spinraza cost around 50% of one Zolgensma infusion, but Spinraza treatments must continue for life at a cost of $375,000 each year. Would the NHS pay for it? Novartis said the drug is priced at half of the estimated $4 million-plus cost of managing the disease with therapy for one decade. Continental Telegraph is a British news publication founded by Senior Fellow of the Adam Smith Institute, Tim Worstall in 2018. Heck, could be crowdfunding campaigns which isn’t all that different from a form of societal insurance. However we distribute that cost it’s still $1 million per kid per treatment.