Gene drug is ‘only hope’ for Turkish child, but costs millions | Health

Gebze, Turkey – Yusuf Polat is only five years old, but has accumulated enough health problems for 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, swallow or breathe properly, and he has to be strapped to seven devices when he sleeps.

Yusuf’s parents have already experienced this same nightmare. Her older sister, Melek, died when she was a year and a half from the same condition.

But this time, her parents have a plan.

Yusuf’s disease, spinal muscular atrophy (SMA) type 1, is a rare genetic condition, but now has a breakthrough drug that can halt disease progression in eligible babies and, in some cases, improve motor functions in a single dose. .

Yet the gene therapy, onasemnogene abeparvovec, marketed as Zolgensma, is also known as the world’s most expensive drug, at $2.1 million – a price that, with rampant inflation in Turkey, is becoming every less accessible day.

“Those who discovered this cure don’t make it a priority to help people, or cure children, or wipe disease off 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 during their fundraising campaign.

The company that owns the drug, Basel-based Novartis, has been criticized around the world for its prices. Novartis said it originally planned to set it at $4 million to $5 million, although a drug price watchdog said a fair price would have been between $310,000 and $900,000.

Novartis justified the price by arguing that it uses state-of-the-art technology and saves families a lifetime of doctor visits.

“With a single therapy, Novartis Gene Therapies is advancing the current Spinal Muscular Atrophy (SMA) treatment paradigm, which is built around a chronic therapy that can cost healthcare systems tens of millions of dollars over the course of of a patient’s life,” a Novartis spokesperson said. told Al Jazeera.

He also points out that he has fewer potential buyers because SMA is a rare disease, affecting around one in 10,000 babies worldwide.

In Turkey, the rate is estimated at around one in 6,000. As a genetic condition, it is most common in children of related couples; these couples, like Yusuf’s parents, are also often the least able to afford treatment.

Yusuf Polat’s family used every means possible to raise money for his treatment, such as billboards displaying his bank account details. [Naomi Cohen/Al Jazeera]

‘A difficult situation’

Kayhan and Mensure Polat live in Gebze, one of Turkey’s manufacturing hubs, where Kayhan used to work in construction. He quit his job so he could keep up with fundraising and doctor visits, causing the family to lose their health insurance.

Yusuf, however, keeps a happy face for his guests and for the camera: his smile is plastered on billboards and cropped in online images that share his story and bank account number, to facilitate donations for the treatment of Yusuf. .

“We have to ask people for their money,” Mensure said. “Some give 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 around 16 million Turkish liras. Since then, the lira has fallen against the dollar, pushing the price to over 34 million lira ($1.83 million). As the pound continues to fall, their target continues to climb and the portfolios tighten.

“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 receive celebrities or politicians – like the mayor of Istanbul Ekrem Imamoglu – to support their urges, and a group of them are working to meet the president. Without such connections, Yusuf’s campaign has focused on hosting concerts, holding stalls at transit centers, protesting outside Novartis headquarters, and running campaigns on his Instagram account, which counts now 22,000 subscribers.

The global distribution of COVID-19 vaccines has clearly shown 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 reach 3 million dollars for those coming from abroad. Residents of the United States and a few dozen countries that have agreements with Novartis can have the treatment covered by their insurance.

No government approval

The Turkish 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 was still evaluating the drug’s effectiveness and had not received the information it requested from Novartis. .

“The sensitivity of the subject requires us to act with the utmost caution,” 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 lawsuit last year to force Turkey to import gene therapy. While the agency that licenses 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 since Serpil is a German citizen. Now three years old, Yiğit can hold his neck straight and learning to walk. His parents continue to fight for access to drugs in Turkey; this year they seized the European Court of Human Rights so that it is supported by the 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 also now covers genetic testing for couples about to marry, and if they are carriers of 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 was only able to live so long because of Spinraza, which is why they waited before deciding to try gene therapy. Yet Yusuf is already three years past the recommended age for treatment. He was rejected by hospitals in the United States and Germany, but a hospital in Dubai said it would treat him as long as he remained below the 13.5 kg (30 lb) limit.

Until they achieve their goal, Mensure rations the milk she gives Yusuf using a syringe to keep him from gaining half a kilo.

If it had exceeded the limit, the provincial government would not have extended its fundraising campaign for a year.

Other families find it more difficult to content themselves with launching campaigns: the Ministry of Health does not recognize the effectiveness of Zolgensma, they can only launch them through legal proceedings.

“It was considered unfavorable to organize campaigns in our country to practice gene therapy, for which there is not yet enough evidence in terms of efficacy and safety,” Koca said in a statement. last year.

Asked about the Turkish minister’s claims, Novartis pointed to several studies that showed the effectiveness of Zolgensma. Although Novartis said it could not comment on Turkey’s regulatory processes, it said “regardless of the country” it aims to find “sustainable access solutions for transformative gene therapies like Zolgensma.” .

The Tezcan family’s lawyer, Mehmet Rıfat Bacanlı, has filed 15 lawsuits against the Ministry of Health for interrupting campaigns.

“The money these families collect is not sustainable, as the number of babies diagnosed is increasing every day,” Bacanlı said. Last year, he said he was aware of about 12 fundraising campaigns; this year, probably due to more genetic testing and awareness, he said there must be over 100.

More than a year after the start of their campaign, the Poles are finally close to the end. Yet even once they lift the full amount, the rest is uncertain. Dubai Hospital has yet to share details of the procedure; so far, they say, they’ve 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 must. We have no other choice. »

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