A Lottery Like No Other Offers Up a Cutting-Edge Medicine–with Lives on the Line

February 7, 2020

(STAT News) – The lottery was devised by the drug’s 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. The first drawing occurred Monday. Ethicists and advocates have debated the merits and the design of the unusual arrangement. Parents said that it was uncomfortable to cast their child’s 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, “we’re talking about lives.” But if it was a chance to get the drug, it was worth trying.