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Health ministers condemn Novartis lottery for Zolgensma, the world’s most expensive drug

BMJ 2020; 368 doi: https://doi.org/10.1136/bmj.m580 (Published 12 February 2020) Cite this as: BMJ 2020;368:m580
  1. Owen Dyer
  1. Montreal

Novartis has held the first draw to choose four babies who will receive its one-shot treatment for the genetic disease spinal muscular atrophy, Zolgensma (onasemnogene abeparvovec), amid criticism of its lottery programme from patient groups and EU health ministers.

Priced in the United States at $2.1m (£1.6m; €1.9m), the most expensive drug course of treatment ever, Zolgensma is not yet approved elsewhere. In December the company announced a plan to give away 50 treatments in other countries over the next six months, the recipients to be chosen randomly from among applicants every two weeks.

Recipients must be under 2, the upper age limit for which the drug is approved in the US. Most of the children in the Zolgensma draw were registered by their doctors. About one child in every 8000 live births is born with spinal muscular atrophy. The most severe type, called type 1 or Werdnig-Hoffmann disease, usually causes death during early childhood if untreated.

The lottery drew condemnation from patient group SMA Europe. “We are alarmed that the programme will make thousands of babies [with spinal muscular atrophy] compete with each other for a lifesaving treatment, splitting tightly knit communities,” the group said in a statement.

The health ministers of Belgium, the Netherlands, Luxembourg, Austria, and Ireland issued a joint statement expressing their “strong reservations” about the programme. “The high level of uncertainty and the non-transparent approach is unacceptable,” the ministers said.

“It proves no sincere commitment to patients and only increases the distress of the families concerned. They are given false hope. If one equals the fate of a patient to a lottery ballot, human dignity and moral values get out of sight. Lotteries are by their nature a form of gambling, and this is absolutely the wrong model to bring to healthcare.”

In response Novartis said that it had “collaborated with an independent bioethics advisory committee” in developing the plan. “We recognise that this programme has inherent challenges and know it will not be a solution for all patients in all countries, but we were compelled to take action to impact the lives that we could and to do so in an expedient manner,” the company said.

Of its critics, Novartis said: “We are reflecting on their input and will consider adaptations to the programme as we move forward.”

Last August, the US Food and Drug Administration publicly rebuked Novartis subsidiary AveXis for withholding evidence of data manipulation in early Zolgensma tests until after the drug was approved. In that case, the altered data served to justify a particular manufacturing process rather than exaggerate the drug’s safety or efficacy.

But in November, the FDA put a partial clinical hold on Zolgensma after learning that AveXis had withheld data from a primate study that suggested possible nerve damage. The hold paused a trial testing higher doses in patients aged 2 to 5. Novartis hopes to expand Zolgensma’s approval to include older patients.

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