World’s most expensive drug to be given to children for free through lottery
7 min readThe 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.
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This July 2012 photo provided by U.S. Customs and Border Protection shows methamphetamine disguised as a chocolate candy bar in Los Angeles. Officials said a California man tried to smuggle more than 4 pounds of methamphetamine out of the country disguised as 45 individually wrapped chocolate bars at Los Angeles International Airport.
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2/11 Cocaine in coffee
This October 2015 photo provided by U.S. Customs and Border Protection shows a packet of cocaine hidden in a bag of ground coffee in Miami. Three bags of roasted, ground coffee arriving at Miami International Airport in a package from Guatemala in October were actually filled with more than 3 pounds of heroin, customs officials said.
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3/11 Cocaine in powdered milk
This November 2014 photo provided by U.S. Customs and Border Protection shows bags of powdered dairy product that contained cocaine in New York. A woman arriving at Kennedy International Airport in New York from Guyana was found with six bags of milk and custard powder that were filled with cocaine.
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4/11 Cocaine in custard powder
This November 2014 photo provided by U.S. Customs and Border Protection shows bags, marked as holding powdered dairy products, that hold cocaine in New York. A woman arriving at Kennedy International Airport in New York from Guyana was found with six bags of milk and custard powder that were filled with cocaine.
AP
5/11 Cocaine in various foods
This October 2015 photo provided by U.S. Customs and Border Protection shows an array of food products concealing cocaine in Newark, N.J. A U.S. citizen arriving from Peru at Newark Liberty International Airport in October had an assortment of food in his luggage that customs officials found also included 10 pounds of cocaine.
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6/11 Cocaine in vanilla wafers
This April 2015 photo provided by U.S. Customs and Border Protection shows vanilla wafers filled with cocaine in Houston.
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7/11 Cocaine in vanilla wafers
A Guatemalan citizen arrived at George Bush Intercontinental Airport from Guatemala City in April with packages of vanilla wafers. But when customs officials opened them up, they said they found they were filled with cocaine instead of cream filling.
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8/11 Liquid cocaine in rum bottles
This December 2014 photo provided by U.S. Customs and Border Protection shows rum bottles filled with liquid cocaine in New York. A man arriving from Guyana at Kennedy International Airport in New York was found to be carrying the bottles that customs officials said were filled with 18 pounds worth of liquid cocaine.
AP
9/11 Cocaine paste in chocolate syrup
This February 2012 photo provided by U.S. Customs and Border Protection shows plastic packets of chocolate syrup and salad dressing concealing cocaine paste in Los Angeles. A mother and daughter traveling from Spain were carrying bags of condiments that customs officials at Los Angeles International Airport decided felt unusually thick. They opened it up to find a plastic bag with cocaine paste placed inside, and then found another syrup packet in their checked-in luggage that contained more cocaine paste.
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10/11 Opium in cinnamon packets
This June 2012 photo provided by U.S. Customs and Border Protection shows packets of opium covered in cinnamon hidden inside a rice cooker in Los Angeles. Officials found the rice cooker stuffed with 3 pounds’ worth of black opium, which had been coated in cinnamon and wrapped in plastic, being transported by a man arriving at Los Angeles International Airport from Iran.
AP
11/11 Cocaine in frozen meat
This undated photo provided by U.S. Customs and Border Protection in October 2015 shows a block of cocaine concealed in a package of frozen meat in New York. A man arrived at Kennedy International Airport from Trinidad with three large packages of frozen meat in his suitcase.
AP
1/11 Methamphetamine in chocolate bar
This July 2012 photo provided by U.S. Customs and Border Protection shows methamphetamine disguised as a chocolate candy bar in Los Angeles. Officials said a California man tried to smuggle more than 4 pounds of methamphetamine out of the country disguised as 45 individually wrapped chocolate bars at Los Angeles International Airport.
AP
2/11 Cocaine in coffee
This October 2015 photo provided by U.S. Customs and Border Protection shows a packet of cocaine hidden in a bag of ground coffee in Miami. Three bags of roasted, ground coffee arriving at Miami International Airport in a package from Guatemala in October were actually filled with more than 3 pounds of heroin, customs officials said.
AP
3/11 Cocaine in powdered milk
This November 2014 photo provided by U.S. Customs and Border Protection shows bags of powdered dairy product that contained cocaine in New York. A woman arriving at Kennedy International Airport in New York from Guyana was found with six bags of milk and custard powder that were filled with cocaine.
AP
4/11 Cocaine in custard powder
This November 2014 photo provided by U.S. Customs and Border Protection shows bags, marked as holding powdered dairy products, that hold cocaine in New York. A woman arriving at Kennedy International Airport in New York from Guyana was found with six bags of milk and custard powder that were filled with cocaine.
AP
5/11 Cocaine in various foods
This October 2015 photo provided by U.S. Customs and Border Protection shows an array of food products concealing cocaine in Newark, N.J. A U.S. citizen arriving from Peru at Newark Liberty International Airport in October had an assortment of food in his luggage that customs officials found also included 10 pounds of cocaine.
AP
6/11 Cocaine in vanilla wafers
This April 2015 photo provided by U.S. Customs and Border Protection shows vanilla wafers filled with cocaine in Houston.
AP
7/11 Cocaine in vanilla wafers
A Guatemalan citizen arrived at George Bush Intercontinental Airport from Guatemala City in April with packages of vanilla wafers. But when customs officials opened them up, they said they found they were filled with cocaine instead of cream filling.
AP
8/11 Liquid cocaine in rum bottles
This December 2014 photo provided by U.S. Customs and Border Protection shows rum bottles filled with liquid cocaine in New York. A man arriving from Guyana at Kennedy International Airport in New York was found to be carrying the bottles that customs officials said were filled with 18 pounds worth of liquid cocaine.
AP
9/11 Cocaine paste in chocolate syrup
This February 2012 photo provided by U.S. Customs and Border Protection shows plastic packets of chocolate syrup and salad dressing concealing cocaine paste in Los Angeles. A mother and daughter traveling from Spain were carrying bags of condiments that customs officials at Los Angeles International Airport decided felt unusually thick. They opened it up to find a plastic bag with cocaine paste placed inside, and then found another syrup packet in their checked-in luggage that contained more cocaine paste.
AP
10/11 Opium in cinnamon packets
This June 2012 photo provided by U.S. Customs and Border Protection shows packets of opium covered in cinnamon hidden inside a rice cooker in Los Angeles. Officials found the rice cooker stuffed with 3 pounds’ worth of black opium, which had been coated in cinnamon and wrapped in plastic, being transported by a man arriving at Los Angeles International Airport from Iran.
AP
11/11 Cocaine in frozen meat
This undated photo provided by U.S. Customs and Border Protection in October 2015 shows a block of cocaine concealed in a package of frozen meat in New York. A man arrived at Kennedy International Airport from Trinidad with three large packages of frozen meat in his suitcase.
AP
“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.
