Unconventional drug ‘lottery’ will hand out 50 doses of Zolgensma, world’s most expensive gene therapy – Genetic Literacy Project

The lottery that began [recently] was not about money, or about choosing a school, or about obtaining a visa. It was about a childs life. In this case, the children selected would receive a drug that otherwise was not available.

The treatment, a gene therapy calledZolgensma, is designed for children like Wynter who have a neuromuscular disease called spinal muscular atrophy, or SMA.

The lotterywas devised by the drugs manufacturer, Novartis, to give families in those places a chance to get it through a novel form of compassionate use a way to get medications that have not been approved while they wait. Fifty doses are slotted to be given away for free in the first half of the year, with up to 100 total.

Ethicists and advocates have debated the merits and the design of the unusual arrangement. Parents said that it was uncomfortable to cast their childs fate into what felt like a sweepstakes a kind of bizarre Willy Wonka contest in which, as Maura Blair, a Canadian mother of a child with SMA put it, were talking about lives.

Zolgensma costs $2.1 million in the United States the worlds most expensive drug.

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Unconventional drug 'lottery' will hand out 50 doses of Zolgensma, world's most expensive gene therapy - Genetic Literacy Project