Drugmaker Novartis recently launched a lottery to help families access the novel gene therapy Zolgensma outside of the United States—the only country where it's currently on the market, but some have questioned whether a lottery is the most appropriate way to distribute the drug, Andrew Joseph and Ed Silverman report for STAT News.

About the life-saving drug

FDA last year approved Zolgensma to treat a spinal muscular atrophy (SMA), a genetic neuromuscular disease.

Patients with SMA produce an insufficient amount of SMN protein, which breaks down motor neurons that send messages to the muscles. In turn, those with the disease develop muscle weakness, which can lead to death. Without any treatment, children with Type 1 SMA, which is the most serious form and affects 1 in 15,000 babies, typically die by the age of two, mostly from respiratory problems, Joseph and Silverman report.

Zolgensma treats the condition by inserting a healthy copy of an SMN gene into the patient's motor neurons, which heals the neurons and restarts production of the SMN protein. The treatment is only needed once. In the United States, the drug is only approved for patients under age two.

However, access to the drug is currently limited.

The United States is the only country where regulators have approved the drug. According to Joseph and Silverman, European regulators are expected make an approval to decision this quarter, while officials in Canada and Australia may take until next year to make a decision.

In addition, the supply of Zolgensma is limited, according to Novartis, and the therapy is complicated to manufacture. On top of that, Novartis has just one facility making Zolgensma, Joseph and Silverman report. The company said it intends to open two more facilities to make this year.

The drug also has a $2.1 million price tag, which makes it the most expensive drug in the world, according to Joseph and Silverman. In countries where the drug is not approved, families have tried fundraising to buy the drug and get it injected by U.S. doctors, Joseph and Silverman report.

Novartis has said it will allow health insurers to pay for Zolgensma over the course of five years via annual installments of $425,000, but critics say the price is too high.

How Novartis' lottery works

To help patients in countries where the drug is not yet approved, Novartis devised a lottery that will give some patients access to the drug for free through a compassionate use program, which enables patients to get unapproved medications. The company will give away 50 doses in the first six months of 2020 and plans to give away up to 100 doses total this year.

Some ethicists have said lotteries can be a fair way to dole out limited supplies of a resource, as they create an even playing field and keep those with more money or better connections from having an advantage over others. However, others questioned whether the lottery is truly fair, saying the company should favor the sickest children, as healthier children may be able to wait longer.

Holly Fernandez Lynch, a bioethicst at University of Pennsylvania's Perelman School of Medicine, said, "If it is really not possible to help all who are in need of help, then a lottery with priority to patients who are worst off is not a bad approach—and definitely fairer than other things a company could do." She added, "The key is to first do everything possible to minimize the need for a lottery at all—and it's not obvious to me that Novartis has done that here."

Genevieve Kanter, another bioethicist from the Perelman School, said she understands why some people feel uneasy about the lottery. "[I]t does become a zero-sum game, which is what bothers some people about the mechanism, even if at the end of the day, more kids get treated than in the alternate scenario where there's no lottery," Kanter said.

Dave Lennon, president of AveXis, the unit within Novartis that developed Zolgensma, said that Novartis thought about giving priority to the sickest patients, but after consulting with ethicists, the company decided they didn't want to influence who would receive the treatment in any way.

"It's the only fair way to allocate," Lennon said, acknowledging that it was "not an ideal situation." He added, "The alternative is not do anything, which we didn't feel like was a good option." According to Lennon, if supply allowed, Novartis wanted to expand the program (Joseph/Silverman, STAT News, 2/7).