The FDA’s approval of Zolgensma marks the first approval in a new wave of potentially curative gene therapies to reach the market soon, and represents a bellwether case for whether payers can adapt to the challenges of potentially transformative drugs with high up-front costs, through the use of innovative payment models.
Approved on Friday by the US Food and Drug Administration (FDA), Novartis’ product was unveiled at a price of $2.12m per patient. This price was recognised as within the bounds of cost-effectiveness by the Institute of Clinical and Economic Review (ICER).
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ICER is an independent US medicines cost-effectiveness think-tank which has drawn attention for criticising medicine prices in the US. Its broadly positive assessment in this case, despite the highest ever upfront list price for a medicine in the US, represents encouraging news not just for Zolgensma, but for the wider gene therapy field.
In its press release, Novartis says that through a combination of pricing approaches, including pay-over-time and outcomes-based agreements, it has been able to achieve cost-effectiveness compared to existing treatments.
Steven Pearson, President of ICER, commented:
“Zolgensma is dramatically transforming the lives of families affected by this devastating disease, and given the new efficacy data for the presymptomatic population, the price announced today falls within the upper bound of ICER’s value-based price benchmark range.”
This case shows that companies may be able to successfully illustrate the cost-effectiveness of treatments in value-based frameworks even when a high price draws press attention. Novartis hopes that one-time therapy Zolgensma can provide a lasting cure to infants with spinal muscular atrophy (SMA) through this new payment model.
Marketing Authorisation from the European Medicines Agency (EMA) is expected to follow the FDA approval in the coming months.
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