Cystic Fibrosis Drug Out of Reach for Many
When Seshagiri Buddana learned of a new cystic fibrosis drug that was transforming lives in the United States and Europe, he was filled with hope that it could help his son, Hemanth, who had spent much of his childhood in a hospital. But he could not get the drug because he lives in India.
The drug’s maker, Vertex Pharmaceuticals, a biotech company in Boston, is not making it available in virtually anywhere in the developing world. It is not trying to sell it, and it is blocking potential generic competitors by seeking patents in many places.
Hemanth died in December, a day before his ninth birthday and 18 months after he would have been eligible to get the drug, called Trikafta, had he lived in the United States.
In much of Asia, Africa and Latin America, families say they are blocked by the company in efforts to get the drug.
Trikafta, taken as three tablets a day, is the most powerful of Vertex’s four cystic fibrosis medications. With a list price of over $322,000 annually in the United States, it is expected to cost millions of dollars over the course of a lifetime. An analysis found that a year’s supply could be made at an estimated cost of just $5,700. Vertex has reported over $17 billion in sales for Trikafta since it was approved in 2019.
A group of patients and their families in four countries recently initiated legal steps to try to force their governments to override intellectual property protections and allow a low-cost generic version of Trikafta to be sold. Under the process, known as compulsory licensing, generic makers would pay Vertex a royalty. Three of the actions are in India, Ukraine and South Africa — where Vertex has been obstructing efforts to make the drug available. The fourth is in Brazil, where Vertex is trying to win coverage for the drug; the concern there is that it will be too expensive.
Vertex said it was pushing to increase access globally. Heather Nichols, a spokeswoman, said that Vertex has provided some form of access to at least one of its cystic fibrosis drugs in Brazil, Poland, Bulgaria, Estonia, Greece, Latvia, Slovakia, Slovenia, Romania and Oman.
Cystic fibrosis damages the lungs. Patients often die in early adulthood, but Trikafta is dramatically extending life expectancy. The genetic defect that causes the disease is most common in people of Northern European ancestry, as are the mutations needed for Trikafta to work. The number of patients in developing countries who are diagnosed and eligible for the drug is believed to be in the thousands.
“They say it’s a miracle drug, but it’s not a miracle if it is not available to everyone who need it,” said Shwetha Sree, who like Mr. Buddana lives in Hyderabad. Her 5-year-old son, Vihaan, has cystic fibrosis and the mutation that would make him eligible for Trikafta when he turns 6, if he were to live in the U.S.
Raphaelle Pereira, 22, had been waiting years for a lung transplant in Curitiba, Brazil. By 2021, her weight had dropped to 36 kilograms, and she no longer had the strength to walk to the bathroom. Her family scraped together $54,000 to buy a two-month supply of Trikafta in the U.S.
“I took it for a couple of days,” she said, “and then I just got up and said, ‘I think I’ll have a shower.’ My whole family was in shock. A few days before I couldn’t even lift my arm.” With the data on how the medication had changed her condition, Ms. Pereira used a legal process to get Brazil’s public health system to buy a steady supply for her.
Cheri Nel, a 38-year-old investment banker in South Africa who is eligible for Trikafta, said she had suggested several ways that Vertex could increase access and still safeguard its profit and intellectual property. She said she got nowhere and is now leading the action in South Africa.
“There’s a balance: You want to keep companies incentivized to investigate and do research and development,” Ms. Nel said. “But it does them no financial harm to let us import a generic because they’re not even trying to sell it.”