Following an announcement by the FDA that it is investigating ‘data manipulation’ of Novartis’ Zolgensma, the Institute for Clinical and Economic Review (ICER) has commented that this could lead to a re-evaluation of its initial cost-effectiveness assessment of the drug.  

Novartis’ gene therapy Zolgensma was approved by the FDA to treat spinal muscular atrophy (SMA) in children under two years of age earlier this year. However, in a statement issued earlier this week the FDA said it was made aware of a “data manipulation issue” of initial product testing performed on animals.

ICER has since responded, and said that it is “deeply concerned about issues related to data integrity, and we are monitoring this situation closely”. Although the animal data which was manipulated did not factor into ICER’s assessment of the cost-effectiveness of Zolgensma, the institute has said it will issue an update of this if necessary. The FDA is currently investigating the situation and if this does reveal compromised data from human trials, ICER could update its pricing of the novel gene therapy.

The initial assessment, which was last updated in May 2019, reviewed data from early results of Novartis’ phase 3 single arm trial SPR1NT. In light of the data, ICER used a cost-effective threshold of $100,000 to $150,000 per quality-adjusted life year (QALY) gained. It determined a value-based price benchmark for Zolgensma would be between $1.1m to $1.9m per treatment.

ICER also determined that for Zolgensma to reach the alternative thresholds of $100,000 to $150,000 per life year gained (LYG), the value-based price benchmark for Zolgensma would be between $1.2m to $2.1m per treatment.

The independent institute urged Novartis to price the gene therapy fairly, to ensure Zolgensma could be accessible for patients. Novartis then set Zolgensma’s price at the top end of ICER’s assessment at $2.12m per treatment, making it the most expensive medicine across the globe.

When this initial assessment was published, ICER president Steven Pearson commented that “insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5m”.

“It is a positive outcome for patients and the entire health system that Novartis instead chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families,” he added.

If the FDA does find any additional data manipulation, especially within that which was used in ICER’s assessment, Novartis could face significant consequences. Not only could the Swiss pharma company see the price of Zolgensma re-evaluated, but the FDA has also stated the it could face civil or criminal penalties.