Gebze, Turkey – Yusuf Polat is only five years old, but has enough medical conditions to last him a lifetime.
He has scoliosis and a hole in his stomach, his bones and muscles have deteriorated to the point where he can no longer walk, swallow or breathe, and he has to be strapped to seven pieces of equipment while he sleeps.
Yusuf’s parents have experienced this nightmare before. His elder sister, Melek, died when she was in the same condition for a year and a half.
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 an advanced drug that can halt the progression of the disease in eligible babies and, in some cases, impair motor functions in one dose can improve .
But the genetic therapy, onasemnogene abeparvovec, marketed as Zolgensma, is also known as the world’s most expensive drug, at $2.1 million — a price that, with runaway inflation in Turkey, is becoming less accessible every day.
“Those who discovered this treatment do not prioritize helping people, or curing children, or wiping disease from the face of the earth; they prioritize making money,” says Zeynep Ceylan, one of Yusuf’s “volunteering sisters” who met the family a few months ago during their fundraiser.
The company that owns the drug, Basel-based Novartis, has come under fire around the world for its pricing. Novartis said it initially planned to price it 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 a lifetime of medical visits.
“With a single therapy, Novartis Gene Therapies advances the current treatment paradigm for spinal muscular atrophy (SMA), which is built around chronic therapy that can cost the healthcare system tens of millions of dollars over a patient’s lifetime,” said a Novartis spokesperson. told Al Jazeera.
It also points out that it has fewer potential buyers, as SMA is a rare condition, affecting about one in 10,000 babies worldwide.
In Turkey, it is estimated to be about one in 6,000. As a genetic disease, it is more common in 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 production centers, where Kayhan worked in construction. He quit his job to keep up with the fundraiser and doctor visits, which caused the family to lose their health insurance.
Despite everything, Yusuf keeps a happy face for his guests and for the camera: His smile is pasted on billboards and cropped online to images that share his story and his bank account number, to make it easier for people to donate for Yusuf’s treatment.
“We have to ask people for their money,” Mensure said. “Some donate small amounts, some just drop by. It is a very difficult situation.”
When Yusuf’s parents launched the campaign last September, the equivalent of the negotiated price was about 16 million Turkish Lira. Since then, the lira has fallen sharply against the dollar, pushing its price to more than 34 million lira ($1.83 million). As the lira continues to fall, their target continues to rise and their 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 ended in a week.”
Some parents get celebrities or politicians, such as the mayor of Istanbul Ekrem Imamoğlu – to support their motives, and a group of them are working to meet with the president. With no such connections, Yusuf’s drive focused on playing concerts, holding booths at transit hubs, protesting in front of Novartis headquarters, and organizing actions 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 authorized to administer the drug, and therefore pay extra for multiple months of stay.
For example, in the United States, the total price tag can be as much as $3 million for those coming from abroad. Residents of the US and several dozen countries that have agreements with Novartis can have their insurance cover the treatment.
No government approval
The Turkish Ministry of Health negotiated with Novartis in 2020, but the talks fell through.
Health Minister Fahrettin Koca told the budget committee of Turkey’s parliament 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 subject forces us to act with the utmost care,” he said. “We can’t play with the hope of these fragile lives.”
The Turkish Ministry of Health 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 lawsuit last year to have Turkey import the gene therapy. While the agency that approves drugs in Turkey has added Zolgensma to its list, the Tezcan family says the ministry continues to block its importation.
Instead of waiting, the couple went to Germany, where Yiğit could be treated for free as Serpil is a German citizen. Now three years old, Yiğit can delay straightens his neck and learns to walk. His parents continue to fight for access to the drug in Turkey; they have applied to the European Court of Human Rights this year to have it covered by Turkish social security.
While Turkey already covers another SMA type 1 treatment, Spinraza, its effect only lasts for months before more doses are needed. Turkey now also covers genetic testing for couples about to get married, and if they carry the gene, it will cover their in vitro fertilization.
Yusuf’s parents have now been tested, as has their 11-year-old daughter. They said Yusuf was only able to live so long because of Spinraza, which is why they waited before deciding to try the gene therapy. Yet Yusuf is already three years older than 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 under the 13.5kg limit.
Until they achieve their goal, Mensure rations the milk she gives Yusuf by syringe to prevent him from gaining half a kilo.
If he had gone over the limit, the county would not have extended his fundraiser for another year.
Other families have a harder time launching campaigns on their own: Since the Department of Health does not recognize Zolgensma’s effectiveness, they can only launch it through a lawsuit.
“It was considered unfavorable to organize campaigns in our country to conduct gene therapy, for which there is still insufficient 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 showed Zolgensma’s effectiveness. While Novartis said it could not comment on Turkey’s regulatory processes, it did say it was seeking “sustainable access solutions for transformative gene therapies like Zolgensma” “regardless of the country”.
The lawyer of the Tezcan family, Mehmet Rıfat Bacanlı, has opened 15 cases against the Ministry of Health for stopping campaigns.
“The money raised by these families is not sustainable, because the number of babies diagnosed is increasing every day,” Bacanlı said. Last year he said he knew about 12 fundraisers; this year, likely due to more genetic testing and awareness, he said it must be over 100.
More than a year into their campaign, the Polats are finally nearing the end. But even if they raised the full amount, the rest is uncertain. The Dubai hospital is 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 it. We have to. We have no other choice.”