Skip to main content

Ricardo Batista and Jessica Batista with their four-month-old daughter Eva in their home in Toronto, on Jan. 4, 2020. Eva was diagnosed with spinal muscular atrophy, a disease that targets the motor nerve cells.

Marta Iwanek/ The Globe and Mail/The Globe and Mail

Canadian babies with a rare and often fatal muscle-wasting disease may soon have access to a cutting-edge treatment that could change their lives – but only if they’re lucky enough to win a high-stakes lottery.

The Swiss pharmaceutical company that makes Zolgensma, a US$2.1-million gene therapy, is planning to give away as many as 100 doses of the one-time treatment this year in countries where the drug is not yet approved, including Canada.

Novartis began accepting applications for the lottery, which it calls a “managed access program,” on Jan. 2. The company intends to select one baby or toddler with spinal muscular atrophy (SMA) at random every two weeks.

Story continues below advertisement

The Zolgensma lottery is an extreme solution to the kind of ethical dilemma that is bound to arise more often in the age of astoundingly expensive gene therapies, a new class of drugs designed to correct underlying genetic defects, usually with a single treatment.

Deciding which patients get gene therapies won’t be easy, especially when governments and private-insurance plans are only beginning to grapple with the eye-watering prices of the new treatments.

Yet some bioethicists say that in a scenario such as this one – where all babies with SMA are equally medically needy and the company says it does not have the production capacity to give away more than a limited supply – a lottery may be the best of the bad options for divvying up free Zolgensma.

“It’s a difficult situation,” said Ricardo Batista, whose five-month-old daughter, Eva, has SMA. “It’s a lottery where we’re leaving children’s lives up to chance. I don’t think it’s a game that any of us want to play.”

The Zolgensma lottery is an extreme solution to the kind of ethical dilemma that is bound to arise more often in the age of astoundingly expensive gene therapies.

Marta Iwanek/ The Globe and Mail/The Globe and Mail

Despite his queasiness about the concept, Mr. Batista said he and his wife, Jessica, will likely put Eva’s name forward for the lottery. Time is not on their daughter’s side.

The Toronto family has already been on a media blitz, trying desperately to raise enough money through GoFundMe to pay for Zolgensma in the United States, the only country where the treatment is currently approved.

SMA is a progressive neuromuscular disorder believed to affect about one in 11,000 babies.

Story continues below advertisement

Patients are usually missing both copies of a gene called SMN1 whose job it is to instruct all the cells in the body to make survival motor neuron, a protein essential for nurturing specialized nerve cells in the spinal cord called motor neurons.

Motor neurons, in turn, transmit signals telling the muscles to move.

If the survival motor neuron protein is in critically short supply, babies are born floppy, often unable to feed or swallow their own saliva because of their low muscle tone. In the worst cases, untreated babies die before their second birthdays.

There is one Health Canada-approved drug for SMA called Spinraza, which has a sticker price of $708,000 in the first year and $354,000 every year thereafter, and has to be taken for life.

Unlike Spinraza, Zolgensma is given as a one-time intravenous infusion. A new, healthy copy of the SMN1 gene is placed inside a harmless virus, which acts as a delivery mechanism for the treatment.

Zolgensma is not a cure, but it gave more than half of the 36 patients enrolled in the two pivotal trials on which the U.S. Food and Drug Administration based its approval the strength to sit on their own.

Story continues below advertisement

As far as the lottery goes, Julie Schneiderman, a spokeswoman for Novartis Canada, said the company designed a managed-access program “anchored in principles of fairness, clinical need and global accessibility – meaning a program that serves all markets irrespective of where we have submitted or plan to commercialize.”

She said the company intends to apply later this year for Health Canada approval of AVXS-101, as Zolgensma is known outside the U.S.

An independent bioethics committee advised AveXis, the Novartis unit developing Zolgensma, on how to craft the program, Ms. Schneiderman said by e-mail.

The names of the bioethicists are not being publicly revealed to protect their independence, she added.

Vardit Ravitsky, a bioethics professor at the University of Montreal’s school of public health, said she had overarching ethical concerns about the high price of Zolgensma and about Novartis’s decision to give away a limited number of doses.

“We’re starting the discussion with it being over $2-million per dose,” she said. “Yes, but why? I’m calling that into question.”

Story continues below advertisement

However, when it comes to the giveaway, Prof. Ravitsky said that, “lotteries are usually considered one of the fairest ways to resolve a situation like this” – despite some people’s emotional reaction against the concept of a life-or-death draw.

If patients’ medical needs are all the same, she said, the unsavory alternatives to a lottery could include selecting winners based on their social or financial status, or other metrics of worthiness.

Laura and Mark Silva of Toronto say they will seek a spot in the lottery for their daughter, seven-month-old Rebecca. There is little chance Zolgensma will be approved and funded in Canada before their daughter turns 2, the age cutoff in both the U.S. FDA’s approval criteria and the Novartis lottery.

“It’s crazy to think that there’s a lottery to help essentially save our daughter’s life," Ms. Silva said. "But we’re going to try. We’re going to do whatever we can to help her.”

Our Morning Update and Evening Update newsletters are written by Globe editors, giving you a concise summary of the day’s most important headlines. Sign up today.

Related topics

Report an error Editorial code of conduct
Due to technical reasons, we have temporarily removed commenting from our articles. We hope to have this fixed soon. Thank you for your patience. If you are looking to give feedback on our new site, please send it along to feedback@globeandmail.com. If you want to write a letter to the editor, please forward to letters@globeandmail.com.

Welcome to The Globe and Mail’s comment community. This is a space where subscribers can engage with each other and Globe staff. Non-subscribers can read and sort comments but will not be able to engage with them in any way. Click here to subscribe.

If you would like to write a letter to the editor, please forward it to letters@globeandmail.com. Readers can also interact with The Globe on Facebook and Twitter .

Welcome to The Globe and Mail’s comment community. This is a space where subscribers can engage with each other and Globe staff. Non-subscribers can read and sort comments but will not be able to engage with them in any way. Click here to subscribe.

If you would like to write a letter to the editor, please forward it to letters@globeandmail.com. Readers can also interact with The Globe on Facebook and Twitter .

Welcome to The Globe and Mail’s comment community. This is a space where subscribers can engage with each other and Globe staff.

We aim to create a safe and valuable space for discussion and debate. That means:

  • Treat others as you wish to be treated
  • Criticize ideas, not people
  • Stay on topic
  • Avoid the use of toxic and offensive language
  • Flag bad behaviour

Comments that violate our community guidelines will be removed.

Read our community guidelines here

Discussion loading ...

Cannabis pro newsletter
To view this site properly, enable cookies in your browser. Read our privacy policy to learn more.
How to enable cookies