Gebze, Turkey – Yusuf Polat is only five years old, but has accumulated enough medical conditions for a lifetime.
He has scoliosis and a hole in his stomach, his bones and muscles have deteriorated so much that he cannot walk or swallow or breathe correctly, and he has to be tied to seven devices when he sleeps.
Yusuf’s parents have been through this same nightmare before. His older sister, Melek, died when she was a year and a half of the same condition.
But this time, his parents have a plan.
Yusuf’s condition, spinal muscular atrophy (SMA) type 1, is a rare genetic disease, but one that now has a cutting-edge drug that may stop the progression of the disease in eligible babies, and in some cases improve motor functions in one dose.
Yet the genetic therapy, onasemnogene abeparvovec, marketed as Zolgensma, is also known as the world’s most expensive drug, at $2.1m – a price that, with runaway inflation in Turkey, becomes less accessible every day.
“Those who discovered this treatment do not prioritise helping people, or curing children, or wiping the disease off the face of the earth; they prioritise making money,” said Zeynep Ceylan, one of Yusuf’s “volunteer sisters” who met the family a few months ago through their fundraising drive.
The company that owns the drug, Basel-based Novartis, has come under fire around the world for its pricing. Novartis said it had initially planned to price it between $4m and $5m, 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 a lifetime of medical visits.
“With a one-time therapy, Novartis Gene Therapies is advancing the current treatment paradigm in spinal muscular atrophy (SMA), which is built around chronic therapy that can cost healthcare systems tens of millions of euros over a patient’s lifetime,” a Novartis spokesperson told Al Jazeera.
It also points out that it has fewer potential buyers since SMA is a rare condition, hitting roughly one in 10,000 babies globally.
In Turkey, the rate is estimated to be about one in 6,000. As a genetic disease, it is more prevalent in the children of couples who are related; 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 centres, where Kayhan worked in construction. He quit his job to be able to keep up with the fundraising campaign and the visits to doctors, causing the family to lose their health insurance.
Yusuf, despite it all, keeps a cheerful face for his guests and for the camera: his smile is plastered on billboards and cropped onto images online that share his story and his bank account number, to make it easier for people to donate towards Yusuf’s treatment.
“We have to ask people for their money,” said Mensure. “Some donate small amounts, some 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 roughly 16 million Turkish lire. Since then, the lira has plummeted against the dollar, raising the price to more than 34 million lire ($1.83m). As the lira keeps falling, their goal keeps climbing and wallets keep getting tighter.
“We can’t reach those who are better off,” said Ceylan. “Had Yusuf been the son of a parliamentarian, the campaign would have ended in a week.”
Some parents get celebrities or politicians – like Istanbul Mayor Ekrem Imamoğlu – to support their drives, and a group of them is working on meeting with the president. Without such connections, Yusuf’s drive has focused on throwing concerts, holding stands at transit hubs, protesting in front of Novartis headquarters and organising drives on his Instagram account, which now has 22,000 followers.
The worldwide distribution of COVID-19 vaccines made it clear that poorer countries, with less bargaining power, have a harder time negotiating prices and access. Patients in these countries must travel to countries authorised to administer the drug, and therefore pay extra for several months of lodging.
In the United States, for instance, the total price tag can go up to $3m for those coming from abroad. Residents of the US and a few dozen countries that have agreements with Novartis can have their insurance cover the treatment.
No government approval
Turkey’s health ministry 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 effectiveness of the drug and has not received the information it requested from Novartis.
“The sensitivity of the subject obliges us to act with the utmost care,” he said. “We cannot play with the hopes of these fragile lives.”
The Turkish 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 make Turkey import the gene therapy. While the agency that approves drugs in Turkey added Zolgensma to its list, the Tezcan family says that the ministry continues to block its importation.
Instead of waiting, the couple went to Germany, where Yiğit could be treated for free since Serpil is a German citizen. Now three years old, Yiğit can hold his neck straight and is learning to walk. His parents continue to fight for access to the drug in Turkey; they applied this year to the European Court of Human Rights to have it covered by Turkish social security.
While Turkey already covers another SMA type 1 treatment, Spinraza, its effect lasts only for months before more doses are needed. Turkey also now covers genetic testing for couples about to wed, and if they carry the gene, it covers their in vitro fertilisation.
Yusuf’s parents have now been tested, as has their 11-year-old daughter. They said Yusuf has only been able to live this long thanks to Spinraza, which is why they waited before deciding to try the gene therapy. Yet Yusuf is already three years past the recommended age for treatment. He was rejected by hospitals in the US and Germany, but a hospital in Dubai has said it will treat him as long as he stays below the 13.5kg (30 pounds) limit.
Until they reach their goal, Mensure rations the milk that she feeds Yusuf through a syringe to keep him from gaining half a kilo.
Had he gone over the limit, the provincial government would not have extended his fundraising campaign another year.
Other families are having a harder time just launching campaigns: as the health ministry does not recognise the effectiveness of Zolgensma, they can only launch them via court case.
“It was found unfavourable to organise campaigns in our country to carry out gene therapy, for which there is not yet enough evidence in terms of efficacy and safety,” said Koca in a statement last year.
When asked about the Turkish minister’s claims, Novartis pointed to several studies which showed the effectiveness of Zolgensma. While Novartis said it could not comment on Turkey’s regulatory processes, it did say that “no matter the country”, it aimed to find “sustainable access solutions for transformative gene therapies like Zolgensma”.
Tezcan family’s lawyer Mehmet Rıfat Bacanlı has opened 15 cases against the health ministry for shutting down campaigns.
“The money that these families collect is not sustainable, because the number of diagnosed babies rises every day,” said Bacanlı. Last year, he said he knew of about 12 fundraising drives; this year, likely due to more genetic testing and awareness, he said there must be more than 100.
More than a year into their campaign, the Polats are finally close to reaching the end. Yet even once they raise the full amount, the rest is uncertain. The hospital in Dubai has yet to share details on the procedure; for now, they say, they have only talked about money.
“We don’t know what the drug will do,” said Kayhan. “But whatever it is, we want to have it. We have to. We don’t have any other choice.”