Novartis plans to give away about 100 doses of its $2.1-million gene therapy Zolgensma (onasemnogene abeparvovac-xioi) for free next year, although the move is being criticised by patient advocates who describe the "lottery-style" scheme as an inappropriate way to distribute a lifesaving treatment aimed at babies. The global managed-access programme will provide the spinal muscular atrophy (SMA) therapy, which is so far only approved in the US for children under two, to SMA patients outside the country.
A spokesperson for the Swiss drugmaker said it adopted the lottery approach on the advice of a group of bioethics experts, who worried that creating complicated criteria could unfairly discriminate against some patients. "In the end we're dealing with a difficult ethical dilemma," he said, and "unfortunately, there is no perfect solution."
Doctors can submit requests for Zolgensma under the new programme, with eligible patients entered into a draw every two weeks for free doses. Novartis' AveXis unit plans to distribute around 100 free doses a year as long as production capacity allows. The drugmaker said it cannot provide the gene therapy, which is given as a one-time infusion, to all eligible patients due supply restrictions. Zolgensma is currently manufactured at a single facility in the US, but Novartis hopes to open two more manufacturing sites within the next year.
Still, Olga Germanenko, a board member of SMA Europe, said the scheme makes Zolgensma "look like a prize." Meanwhile, Kacper Rucinski of the TreatSMA patient advocacy group called the programme "too crude," and that the company is "making patients compete. Which will be the lucky one? That's not helpful." Instead, he suggested that prioritising patients or countries with the highest needs would have been a better option.
Novartis' programme does not prioritise countries where Biogen and Ionis Pharmaceuticals' SMA treatment Spinraza (nusinersen) isn't routinely available, or for patients who do not respond to Spinraza, according to a report in The Wall Street Journal. Spinraza was cleared in the US in 2016, costing $750,000 for the first year and then $375,000 for each year after that. The lifelong treatment has also been approved in Europe.
Zolgensma, which beat sales forecasts in the third quarter coming in at $160 million, is currently under review by regulators in Europe and Japan, with decisions expected next year. Once a country or region approves the gene therapy, Novartis will stop offering the lottery programme there.
For related analysis, see ViewPoints: Novartis' Zolgensma hits a speed bump. See also, ViewPoints: Payers hopeful that Zolgensma may spark new thinking in gene therapy cost models.
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