Turkey under pressure over funds to treat rare disease

A new faultline of polarization emerged between the Turkish government and rights defenders over the treatment for some 1,100 children who suffer from a rare disease.

Several desperate families have been conducting online campaigns to secure funds for the costly treatment of their children with spinal muscular atrophy (SMA) — a neuromuscular disorder that causes the gradual wasting of muscles and newborn mortality.

The disease is treated either with a drug, called Sprinraza, that is administered every four months, or with a gene therapy, called Zolgensma, which is a one-off cure for babies under the age of 2 or below 21kg.

Didem Demir, 2, has been struggling with SMA for a year because of a delayed diagnosis in a public hospital in the northwestern city of Bursa.

Her family collected about €416,000 ($510,000) but they need €650,000 more.

After two weeks, she will not be eligible to apply for gene therapy, which is available in Hungary.

Zolgensma was approved by the US Food and Drug Administration and is offered in more than 40 countries around the world, including Germany, France and Italy, but it costs over $2 million — discouraging its use without state support.

Since 2019, doses of Sprinraza — costing about €90,000 — are reimbursed to some families in Turkey, but Zolgensma is not included in the reimbursement list despite a court ruling in favor of it.

“Babies who use the first drug carry the risk of death, while the gene therapy is successful with 95 percent of patients. It is the last chance for our children to be alive,” Didem’s aunt Cigdem Demir told.

Although Koca said the state already covers the expenses of SMA patients, not all children can benefit from Sprinraza-based treatment.

“They are subject to very detailed neurological and physical tests, and they can be easily removed from the reimbursement list if they are deemed ineligible in one of those tests,” Kardelen Yarli, an Istanbul-based lawyer, told.

Some of the eligibility tests, like not using respirators for a couple of hours, have been criticized by medical experts for not being required in European countries.

The gene therapy, despite being added to the Foreign Medicine List by Turkey, is not covered by the state-run Social Security Institution, rendering low- and medium-income families much more desperate as they cannot afford it.

After the New Year’s Eve lottery prize, which stood at 75 million liras ($10 million), was not claimed by anyone, thousands of social media users, whose messages were echoed by opposition politicians and dissident mayors, initiated a campaign to ask the government to re-allocate the money to SMA patients rather than the Turkish Wealth Fund.

However, this spontaneous social mobilization received a backlash from the government, with Health Minister Fahrettin Koca defaming the efforts as a “dirty campaign.”

He added in a statement: “Our government is strong enough to treat all patients without concern over resources. The main issue here is supporting pharmaceutical companies at the expense of moral parameters and making our state look weak.”

For Mahir Unal, deputy chair of the ruling Justice and Development Party, the campaign was a “dark propaganda based on lies” by the opposition.

With a recently adopted legislation, donation campaigns cannot be conducted online and they have to get approval from provincial authorities.

“These families are racing against time to save their children before the age of 2 to get success in the treatment. The health minister’s statement meant to get clear of his responsibilities,” Burhanettin Bulut, a lawmaker from the main opposition party CHP, told Arab News.

“The state should set up a specific budget for rare diseases to fulfil its obligations vis-a-vis these babies. The cries of help from these parents are a disgrace for the country,” he added.
SMA occurs approximately once in every 10,000 live births around the world, but the rate rises to once in every 6,000 babies in Turkey.

According to Yarli, families want to apply the one-off gene therapy before the age of 2 because its effectiveness has been proved globally.

“Despite the court ruling, the government insists on not approving this second drug into its reimbursement list,” she said.

She added: “This is against the constitution, against the European Convention of Human Rights on the right to health of children.

“Turkey is under a positive obligation to secure the right to effective respect for physical integrity of citizens.”

Yarli thinks that banning these donation campaigns was meant to undermine human rights efforts rather than performing the criteria for a social welfare state.