- An FDA decision is due within weeks on Novartis' gene therapy Zolgensma, which has proved in clinical trials to extend the lives of babies with the rare condition called spinal muscular atrophy.
- An April 3 report from the Institute for Clinical and Economic Review estimated the one-time treatment may be cost-effective at prices of up to $1.5 million, depending on the measure used. The Switzerland-based big pharma has not set a price but has argued the therapy would offer clinical value at $4 million given the lack of options and severity of the condition.
- The durability of Zolgensma's benefit and its ability to prevent disability is still subject to question. Payers have said they could ask for a refund if patients treated with Zolgensma decline and need another expensive drug to arrest progression, according to industry commentary included in ICER's report.
Novartis last year spent $8.7 billion to buy AveXis and acquire what is likely to be only the second gene therapy approved in the United States.
The treatment targets the most severe form of SMA, Type 1, which affects roughly 300 babies a year in the U.S. and usually results in death less than two years after birth.
ICER, a closely followed cost watchdog, has argued for Novartis to set a lower price, between $900,000 and $1.5 million, than what the pharma has said it could reasonably charge for Zolgensma (onasemnogene abeparvovec).
In keeping with standard industry practice, Novartis has not disclosed what Zolgensma will cost, and probably won't until the FDA and other regulators approve the therapy. Given how the company has advocated for use of higher cost-effectiveness thresholds, it would be a surprise if its list price did not exceed ICER's mark by some measure.
On Wednesday, ICER put out its final report on Zolgensma and a competing therapy, Biogen's Spinraza (nusinersen), containing its cost-effectiveness calculation. This report, formally approved by an expert panel convened by ICER, incorporated commentary from pharmaceutical manufacturers, insurers and patient advocates about the need to base payment for such expensive therapies on how well patients fare post-treatment.
These outcomes-based contracts have become more common with pricey new drugs that have uncertain long-term benefits, and the commentary contained clues about what events would trigger a discount. A clear example cited in the report are patients who, after receiving Zolgensma, then require further treatment with Spinraza, which as a maintenance therapy requires an infusion every four months at a list price of $125,000.
Spinraza's price also came under criticism, with ICER stating that a cut of up to 90% would be necessary to meet its preferred value-based price benchmarks.
Clinical data included in the report showed that at two years after treatment with Zolgensma, one of 12 patients was unable to achieve head control and two were unable to sit unassisted. These are motor control milestones that could be reached with additional Spinraza doses, so patients progressing from one to the other is a very real possibility.
In a statement, Biogen noted reports have indicated seven of the 15 patients treated with Zolgensma went on to receive Spinraza.
ICER's report acknowledges that use of combination or sequential therapy with Zolgensma and Spinraza remains "an open question" and hasn't been well studied.
Other clinically validated ways of measuring physical development could also be used to measure outcomes for purposes of determining reimbursement, according to discussions from a policy roundtable cited in the ICER report. These include a scale called the Children's Hospital of Philadelphia infant test of neuromuscular disorders, which measures arm and leg movement, rolling and sitting.
In an emailed statement, AveXis criticized the cost-effectiveness standards contained in the report as "designed around the status quo of chronic care management and cannot possibly capture the full benefits of disease-modifying treatments delivered as a one-time administration."
But the statement acknowledged that the company is exploring risk-sharing and installment payment models that can enable patients to receive Zolgensma.