Zolgensma Lottery: parents enter the baby drug lottery to save the daughter’s life

Ontario’s parents of a five-month-old girl with a rare and life-threatening disorder have entered a lottery to obtain the world’s most expensive drug in the hope of saving her life.

Ricardo and Jessica Batista risk that their daughter Eva, who has spinal muscular atrophy (SMA), is randomly selected by the Swiss pharmaceutical company that makes the multimillion-dollar drug.

The unique and unique gene therapy called Zolgensma costs $ 2.8 million and is reportedly the only possible cure in the market. It has not been approved by Health Canada. For the medication to be effective, Eva must receive it before the age of 2.

“She is a beautiful girl who always has a smile on her face,” Ricardo said on Your Morning on CTV Wednesday. “She doesn’t know something is wrong. Sometimes we look at her and she kills us inside that she is so happy and has no idea.”

The SPA affects simple actions such as breathing, swallowing and Eva’s ability to keep her head upright. This week, she is back in the hospital, Ricardo said. She was intubated after contracting pneumonia and two viruses.

The Swiss pharmaceutical company Novartis began accepting applicants for the Zolgensma lottery earlier this month and said it will select one at random every two weeks to receive therapy. It will give away 100 doses worldwide. If Eva does not win, she will continue with a lifelong alternative therapy called Spinraza, which is approved in Canada and costs more than $ 700,000 in the first year and more than $ 350,000 for the following years. Ricardo said Spinraza has not proved as effective as Zolgensma.

“There is a lot of risk in that. The effect on the people we have seen is not the same (as the lottery drug),” he said. “We have the possibility of winning this lottery, which is still a remote possibility, or we have to go somewhere and pay for it ourselves.”

The Batistas received an estimated cost of $ 4 million for Eva to administer Zolgensma in a US hospital. UU.

In an emotional interview on Your Morning on CTV, Ricardo wore a “For Eva Strong” sweater and said there was no response from the Canadian government. “It doesn’t look like any financing or help,” he said.

The lottery system, which has been compared to the Hunger Games and referred to by some means as the “baby lottery,” has been criticized by patient groups around the world.

“It’s really too crude,” said Kacper Rucinski, co-founder of the UK-based patient advocacy group, TreatSMA, in an interview with the Wall Street Journal. “They are making patients compete. What will be the lucky one? That is not useful. “

The Batistas have been organizing fundraising events and launched a GoFundMe campaign in October to raise $ 2.8 million, the cost of the life-saving medicine. So far, they have raised more than $ 743,000.

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