Long before Novartis won its first approval for gene therapy Zolgensma, the drugmaker faced questions about how it would price a one-time cure for spinal muscular atrophy, a devastating rare disease.
Then, when the U.S. gave the green light, Zolgensma's $2.1 million price tag drew some criticism, but some praise too. Now, its plan to actually give away doses of the therapy beyond U.S. borders is raising eyebrows.
Novartis plans to offer up to 100 doses for free per year in countries where Zolgensma is not yet approved. But those doses would be handed out in a lottery, rather than targeted toward particularly needy patients—a fact that has patient advocates worried.
U.K. patient group TreatSMA said while it applauded the effort, it’s “yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease.” The group says it’s “highly unlikely” the approach will be legal in the U.K.
Under the program, doctors would submit requests for treatment to the company on behalf of their patients. Eligible patients would be entered into the lottery pool. If patients don’t win, they’ll remain in the program for future selections. Novartis plans to award 50 doses in the first half of 2020, TreatSMA reports.
The group said it's gathering feedback from doctors and other SMA groups to form a position on the program. But its co-founder Kacper Rucinski told the Wall Street Journal the program is "really too crude." Instead, advocates would've preferred a program that would prioritize countries and patients with the most need for treatment, he told the newspaper. Patients in some countries can access Biogen's competing SMA med Spinraza, while others don't have any treatment options.
So far, the drug is only approved in the U.S., so the program is intended for other markets around the world. When countries approve the med, patients there will no longer be eligible for Novartis’ expanded access program.
A Novartis spokesperson told Reuters the donation program is intended to be a long-term commitment. The company "designed a program anchored in principles of fairness, clinical need and global accessibility to best determine the equitable global distribution of a finite number of doses that doesn’t favor one child or country over another," he added.
Novartis won U.S. approval for Zolgensma in May and priced the med at $2.125 million. Despite the high price tag, cost watchdogs have endorsed the drug, and recent sales have come in stronger than expectations. Novartis reported $160 million in third-quarter sales, beating Wall Street estimates by 60%.
After launch, the company suffered a data manipulation scandal that didn’t end up hurting demand, execs have said. In August, the FDA said Novartis reported data manipulation from preclinical Zolgensma development. The issue didn't affect safety or efficacy, officials said.
Still, the company ran into intense criticism because it completed its internal investigation before notifying authorities, and the drug won U.S. approval during Novartis' probe.