Belgian toddler's family reaches crowdfund goal for world's most expensive medicine

Belgian toddler's family reaches crowdfund goal for world's most expensive medicine
The family of 9-month-old Pia, who suffers from spinal muscular atrophy, will be able to treat their toddler with the world's most expensive drug. Credit: TeamPia/Facebook

A Flemish toddler fighting a life-threatening genetic condition will be able to afford treatment with the world's most expensive medicine thanks to a massive outpour of support from across Belgium.

After just two days, the family of 9-month old Pia announced they were "overwhelmed" after they had reached their million-euro fundraising goal, thanks to a mass response from Belgians to their nation-wide SMS campaign.

"It is amazing that this happens in two days," Pia's mother told VRT News in reference to the campaign they launched to afford Zolgensma, the only medicine capable of curing their child.

"It is overwhelming that a country can come together for something like that," she added.

Pia suffers from spinal muscular atrophy, an aggressive genetic condition which progressively leads to muscular failure, and which sees most children diagnosed with it live no further than four years old.

With a price tag of €1.9 million for a single dose, the recently released gene treatment is the most expensive medicine in the world.

The Flemish family's fundraiser effort, which received widespread social and mass media attention, saw a mass show of solidarity across Belgium, with 957,549 text messages received in total, surpassing the original goal of 917,000.

In a Facebook post, the family thanked their supporters and said they had "no words," as they announced they were getting ready to travel to the United States, where the treatment can be administered.

"We are getting ready for a trip to Chicago, towards Pia's future!" the family wrote.

The family's efforts to treat their child drew the attention of government officials and even from people beyond Belgium's borders, with Pia's mother saying they had received supportive messages from the Netherlands.

Responding to the fundraising campaign, the cabinet of federal health minister, Maggie De Block, said they could not do anything before Zolgensma was approved for the European market.

But some observers say that governments must do more to keep prices in the health and pharmaceutical industry in check, even as industry giants boast claims of innovation to justify rocketing prices.

"You can hardly speak of a connection," Jan Rosier a member of KU Leuven's pharmaceutical faculty told De Standaard, adding that, for years, "the prices of new medicines have been rising faster than the costs of research and development behind them."

"We are overjoyed for our Pia," the toddler's mother told Het Nieuwsblad, adding: "but at the same time hope that no one ever has to do this again to save the life of their child."

Gabriela Galindo

The Brussels Times

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