Gene Therapy, Pharma

Novartis’ SMA gene-therapy, if priced at $2M, could be more cost-effective than Biogen’s Spinraza — ICER

Biogen’s Spinraza was approved by the FDA amidst much fanfare in 2016 as the first and only disease-modifying treatment for SMA, a rare and often fatal genetic muscular disorder. But the price tag of $750,000 for the first year of therapy (and a lower price thereafter) prompted heavy criticism, although many payers eventually agreed to reimburse the treatment. However, a report by the Institute for Clinical and Economic Review (ICER) on Thursday has suggested Novartis’ experimental SMA gene therapy, Zolgensma, could be more cost-effective in the long run versus Spinraza.

Zolgensma is currently under FDA review and the agency is expected to announce its decision on the drug in May 2019. In its review, ICER has assumed Zolgensma will carry a list price of $2 million, although the Swiss drugmaker has suggested a price of $4 million for the curative therapy, which it acquired via its $8.7 billion takeover of AveXis, may be justified.

Despite the lack of long-term data on either therapy, the non-profit estimated incremental cost-effectiveness of Spinraza is $728,000 per QALY in presymptomatic SMA patients, while Zolgensma has an incremental cost-effectiveness of $247,000 per QALY in patients with symptomatic Type I SMA (on the basis of a placeholder price of $2 million).

QALYs, or quality-adjusted life-years, are a measure of the state of health of a person or group in which the benefits — in terms of length of life — are adjusted to reflect the quality of life. Essentially, one QALY is equal to one year of life in perfect health.

“Novartis has estimated that Zolgensma would yield 13.3 QALYs in SMA Type I patients, and could be priced to $4-5 million based on the relationship of 10-year cost of approved drugs for ultra-rare diseases and their corresponding incremental QALYs gained. ICER estimates Zolgensma provides 11.33 QALYs, and uses a placeholder price of $2 million. While ICER undercut both of Novartis’ estimates, the high cost/QALY of Spinraza in Type I SMA patients offers Novartis bargaining power with payers if the company can argue Spinraza use could be reduced or eliminated to offset total costs,” Leerink analysts wrote in a note on Friday.

Currently available data on Spinraza and Zolgensma show prolonged survival and improved motor function compared with historical controls or placebo, but “there remains considerable uncertainty in the generalizability of the results and in the long-term durability and tolerability of treatment. In particular, for both interventions, the narrow eligibility criteria of trials and the limited sample size (especially for Zolgensma) raises concerns about generalizability of results to the wider population of patients with SMA,” the report added.

Biogen underscored this difference in sample size in its response to the report. “The draft ICER report is an incomplete representation of Spinraza’s value to patients and health care systems. Furthermore, to compare a treatment that has helped nearly 6,000 patients worldwide against an investigational treatment on data from 15 patients with an artificially selected price is misleading and wrong,” a spokesperson told Endpoints News.

The ICER report is not final and has been opened to the public for comment, which will be incorporated into an evidence report in February 2019. This report will be subject to further deliberation by one of ICER’s independent evidence appraisal committees in early March, after which a final report will be released by the end of the month.

“If confirmed in a final report…these findings could offer support for Novartis in pricing and access negotiations for Zolgensma ahead of 2019 approval,” Leerink analysts added.

In response to the report, Novartis said that the findings had affirmed the company’s initial assessment of the value of their product. “While we have not yet determined the price of our investigational product…once we determine a price, we are committed to flexibly partnering with healthcare stakeholders to ensure access,” a spokesperson told Endpoints News. 

Like NICE in the UK, ICER analyzes the evidence on the effectiveness and value of drugs and other medical services in the United States, however unlike NICE, it is not a government-affiliated body.

In August, NICE refused to recommend the use of Spinraza in the UK — despite Biogen discounting its price — on the basis that the long-term impact of the drug was unclear for patients, some of whom die by the age of 2. Without that data, the price of the drug couldn’t be justified, they said.


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