Novartis launches lottery for the world’s most expensive drug

Novartis is offering 100 doses of its gene therapy Zolgensma, the most expensive drug in the world, for free on a lottery basis.

John Miller had the news for Reuters:

Novartis aims to give away 100 doses of its $2.1 million-per-patient Zolgensma for spinal muscular atrophy (SMA) in 2020 in a free-drug program that one patient group worried was a “health lottery” that could neglect some babies.

Starting Jan. 2, Novartis’s AveXis unit which developed Zolgensma will allocate 50 doses of the world’s costliest single-dose treatment through June for babies under 2 years old, Novartis said on Thursday, with up to 100 total doses to be distributed through 2020.

The program applies to countries where the medicine is not yet approved for the rare genetic disorder affecting 1 in 10,000 live births, but which can lead to death and profound physical disabilities.

Zolgensma, with sales of $175 million through September, won U.S. approval in May and has been touted as potentially curative for babies treated before symptoms begin.

Eric Sagonowsky from Fierce Pharma wrote:

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.

Scarlett Kuang from Karma noted:

Novartis told the WSJ that bioethics experts advised them and the plan addresses concerns about discrimination toward some patients. “Unfortunately, there is no perfect solution,” a spokesman said.

The program focuses on countries where the medicine is pending approval, and Novartis cited low manufacturing capacity as a the main reason for its approach. It currently has only one facility in the U.S., and expects to build two more plants next year. 

Spinal muscular atrophy is a rare genetic disease leading to muscle weakness and paralysis. It’s caused by a defective or missing SMN1 gene, affecting one in 10,000 newborn babies. If left untreated, 90% of patients of Type 1 SMA will die by age of 2 years old. 

U.S. pharma Biogen’s Spinraza is the only SMA treatment available on the market. Spinraza costs $750,000 the first year, and must continue for life at $375,000 each year.