World's most expensive drug to be given to children for free through lottery

Patient advocacy groups are concerned choosing 'lucky' kids isn't the right approach

Clark Mindock
New York
Friday 20 December 2019 18:26 GMT

The pharmaceutical company Novartis is planning on setting up a lottery system to give away the world’s most expensive drug for free, but patient advocates are concerned that picking “lucky” people isn’t the best way to do things.

The drug, Zolgensma, is a $2.1m treatment for spinal muscular atrophy, a rare genetic disorder that leads to children losing control of their muscles.

The lottery system — which has been called a managed access program — would establish a bi-weekly draw for babies under the age of two who live in countries where Zolgensma is not approved.

Those who are not chosen will simply stay in the program until they are no longer medically eligible.

The British advocacy group TreatSMA has expressed concern with the program, which it describes as akin to picking “lucky” kids to receive the life-saving drug over others.

“We are yet to be convinced that a health lottery is an appropriate way of meeting the unmet medical needs in this severe disease,” the group said in a statement.

AveXis, the subsidiary that developed the drug, says that the program was created in response to growing demand for the drug outside of the US, where it was approved by the Food and Drug Administration in May.

The company is still trying to register the drug in around three dozen companies.

The dosage lottery is set to start on 2 January, and AveXis has said the program was created with the advice of a “bioethics advisory committee”, and manages manufacturing constraints.

Just two drugs for spinal muscular atrophy are approved in the US. The other, called Spinraza, requires recurring doses. The first costs $750,000 for the first year, and $375,000 each following year.

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