Seeking to provide its pricey gene therapy to patients in countries where the drug is not yet approved, Novartis (NVS) has taken the unusual step of creating a lottery, a move that drew criticism from patient groups over concerns the approach would unfairly forsake some babies.
At issue is access to Zolgensma, which costs $2.1 million per patient and is used to combat spinal muscular atrophy, a rare inherited disease that afflicts children up to 2 years old and often leads to death. The treatment is available in the U.S., but not yet in Europe or Japan, where approval is expected next year. Meanwhile, parents have been clamoring for the medicine.
In recent months, stories have popped up in countries such as Belgium and Hungary about families using crowdfunding to pay for the unapproved treatment. Novartis may have worsened their frustration by countering that supplying Zolgensma through compassionate use programs was not an option under European Union laws because another treatment from Biogen could be obtained.