Gebze, Turkey – Yusuf Polat is only five years old, but he has accumulated enough medical conditions to last his entire life.
He has scoliosis and a hole in his stomach, his bones and muscles have deteriorated so badly that he can’t walk or swallow or breathe properly, and he has to be strapped to seven devices when he sleeps.
Yusuf’s parents have been through this same nightmare before. His older sister, Melek, died when he was a year and a half from the same condition.
But this time, her parents have a plan.
Yusuf’s condition, spinal muscular atrophy (SMA) type 1, is a rare genetic disease, but now he has a next-generation drug that can stop disease progression in eligible babies and, in some cases, improve motor functions with a single dose. .
However, the gene therapy, onasemnogene abeparvovec, marketed as Zolgensma, is also known as the most expensive drug in the world, at $2.1 million, a price that, with runaway inflation in Turkey, is becoming less affordable by the day. .
“Those who discovered this treatment do not prioritize helping people, nor curing children, nor erasing the disease from the face of the earth; they prioritize making money,” said Zeynep Ceylan, one of Yusuf’s “volunteer sisters” who met the family a few months ago through her fundraising campaign.
The company that owns the drug, Basel-based Novartis, has been criticized around the world for its price. Novartis said it had initially planned to price it at between $4 million and $5 million, though a drug price watchdog said a fair price would have been between $310,000 and $900,000.
Novartis has justified the price by arguing that it uses advanced technology and saves families lifetime medical visits.
“With a unique therapy, Novartis Gene Therapies is advancing the current treatment paradigm in spinal muscular atrophy (SMA), which is based on a chronic therapy that can cost healthcare systems tens of millions of euros over the lifetime of a patient. a patient,” a Novartis spokesperson said. he told Al Jazeera.
He also notes that he has fewer potential buyers since SMA is a rare condition, affecting roughly one in 10,000. you drink globally.
In Turkey, the rate is estimated to be one in 6,000. As a genetic disease, it is more prevalent in the children of related couples; these couples, like Yusuf’s parents, are also often the least able to afford treatment.
‘A difficult situation’
Kayhan and Mensure Polat live in Gebze, one of Turkey’s manufacturing hubs, where Kayhan worked in construction. He quit his job so he could keep up with the fundraiser and doctor visits, causing the family to lose their health insurance.
Yusuf, through it all, keeps a cheerful face for his guests and for the camera: his smile is plastered on billboards and cropped in images online that share his story and bank account number, to make it easier for people donate for Yusuf’s treatment. .
“We have to ask people for their money,” Mensure said. “Some donate small amounts, others just pass by. It’s a very difficult situation.”
When Yusuf’s parents launched the campaign last September, the equivalent of the negotiated price was approximately 16 million Turkish lira. Since then, the lira has tumbled against the dollar, pushing the price to more than 34 million liras ($1.83 million). As the lira keeps falling, its target keeps rising, and wallets get tighter and tighter.
“We can’t reach those who are better off,” Ceylan said. “If Yusuf had been the son of a parliamentarian, the campaign would have been over in a week.”
Some parents get celebrities or politicians, like the mayor of Istanbul Ekrem Imamoğlu – to support their campaigns, and a group of them are working to meet with the president. Without such connections, Yusuf’s campaign has focused on holding concerts, setting up booths at transit centers, protesting outside Novartis headquarters and organizing campaigns on his Instagram account, which now has 22,000 followers.
The global distribution of COVID-19 vaccines made it clear that poorer countries, with less bargaining power, have a harder time negotiating prices and access. Patients from these countries must travel to countries authorized to administer the drug and therefore pay extra for several months of accommodation.
In the United States, for example, the total price can rise to 3 million dollars for those who come from abroad. Residents of the US and a few dozen countries that have agreements with Novartis can have the treatment covered by their insurance.
No government approval
Turkey’s Ministry of Health negotiated with Novartis in 2020, but the talks fell through.
Health Minister Fahrettin Koca told the Turkish parliament’s budget committee last year that the ministry’s SMA Scientific Committee is still evaluating the drug’s effectiveness and has not received the information it requested from Novartis.
“The sensitivity of the issue forces us to act with great care,” he said. “We cannot play with the hopes of these fragile lives.”
Turkey’s Health Ministry did not respond to multiple requests for comment.
Fikri and Serpil Tezcan, whose son Yiğit was diagnosed with SMA type 1 when he was 60 days old, won a court case last year to get Turkey to import the gene therapy. While Turkey’s drug-approving agency added Zolgensma to its list, the Tezcan family says the ministry continues to block its importation.
Rather than wait, the couple went to Germany, where Yiğit could receive free treatment as Serpil is a German citizen. Now three years old, Yiğit can hold her neck straight and she is learning to walk. Her parents continue to fight for access to drugs in Turkey; they applied this year to the European Court of Human Rights to be covered by Turkish social security.
While Turkey already covers another treatment for SMA type 1, Spinraza, its effect lasts only months before more doses are needed. Turkey now also covers genetic testing for couples who are about to get married, and if they do carry the gene, it covers their in vitro fertilization.
Yusuf’s parents have now been tested, as has their 11-year-old daughter. They said that Yusuf has only been able to live so long because of Spinraza, which is why they waited before deciding to try gene therapy. However, Yusuf is already three years past the recommended age for treatment. He was turned away by hospitals in the US and Germany, but a hospital in Dubai said he will treat him as long as he stays under the 13.5kg (30lb) limit.
Until they reach their goal, Mensure rations the milk he gives Yusuf through a syringe to prevent him from gaining half a kilo.
If it had exceeded the limit, the provincial government would not have extended its fundraising campaign for another year.
Other families are finding it more difficult to simply launch campaigns: as the Ministry of Health does not recognize the efficacy of Zolgensma, they can only launch them through a judicial process.
“It was considered unfavorable to organize campaigns in our country to carry out gene therapy, for which there is still not enough evidence in terms of efficacy and safety,” Koca said in a statement last year.
When asked about the Turkish minister’s claims, Novartis pointed to several studies that demonstrated the efficacy of Zolgensma. While Novartis said he could not comment on Turkey’s regulatory processes, he did say that “regardless of the country,” his goal was to find “sustainable access solutions for transformative gene therapies like Zolgensma.”
The Tezcan family’s lawyer, Mehmet Rıfat Bacanlı, has opened 15 cases against the Ministry of Health for shutting down campaigns.
“The money these families collect is not sustainable, because the number of babies diagnosed is increasing every day,” Bacanlı said. Last year, he said that he knew of about 12 fundraising campaigns; this year, likely due to more genetic testing and awareness, she said there must be more than 100.
More than a year into their campaign, the Polats are finally nearing the end. However, even once they raise the full amount, the rest is uncertain. The Dubai hospital has yet to share details about the procedure; For now, they say, they have only talked about money.
“We don’t know what the drug will do,” Kayhan said. “But whatever it is, we want to have it. We have to. We have no other choice.”