The cost of a popular Vertex Pharmaceuticals therapy could be contributing to cystic fibrosis (CF) treatment disparities around the world, researchers argued in a recent paper.
Just 12% of some 162,000 people estimated to have CF worldwide are getting Vertex’s pricey triple-combination therapy Trikafta, The Journal of Cystic Fibrosis reports. CF fibrosis transmembrane conductance regulator modulators like Trikafta, of which Vertex sells four, represent an “unparalleled opportunity to increase quality and length of life for almost all CF patients,” the researchers wrote.
Problem is, “the medicines are so expensive they are essentially unavailable unless reimbursed by the government or health system authorities," the team added.
Vertex’s CFTR modulators, which include Orkambi (ivacaftor/lumacaftor), Symdeko (ivacaftor/tezacaftor) and Trikafta (ivacaftor/tezacaftor/elexacaftor), carry U.S. list prices between $270,000 and $310,000 per year before discounts, according to the journal. Patients with CF need treatment for life.
Since the drugs carry high list prices, they're often unavailable to patients even after a regulatory approval, the researchers wrote. Therefore, “urgent action is needed to improve rates of diagnosis and treatment for CF, to ensure a higher percentage of patients receive the most effective triple combination treatment,” they argued.
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At the end of 2020, Vertex had Trikafta reimbursement agreements in the U.S., U.K., Ireland, Denmark, Germany and Slovenia. Since then, Vertex has clinched Trikafta coverage deals in more than 16 additional countries, including Slovakia, Malta, Italy, France, Portugal, Croatia, Spain and the Netherlands, a company spokesperson said over email. The company's other drugs are available in even more countries, the spokesperson said.
Vertex estimates there are some 83,000 CF patients across the U.S., Europe, Canada and Australia, CEO Reshma Kewalramani said last month at the 2022 J.P. Morgan Healthcare Conference. Among that group, Vertex figures about 25,000 could benefit from a triple-combination therapy who aren't yet receiving treatment.
Given Trikafta's slate of reimbursement deals last year, plus a U.S. launch in kids ages 6 to 11, the nascent blockbuster has reached "thousands" of more patients, the CEO added.
With another triple combination therapy expected to advance to phase 3 testing this year, access to Trikafta could set a precedent for future therapies, the journal researchers point out. The drug is expected to remain under patent until 2037. Therefore, they argue that "without intervention," the medicine "will remain out of reach for patients outside of the world’s richest countries."
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The researchers acknowledge their estimates of undiagnosed CF patients in low- and middle-income countries are "highly uncertain" thanks to a "paucity” of high-quality data. The team used data from patient registries where available. Elsewhere, they conducted medical searches and consulted with dozens of CF experts to determine the size of the diagnosed patient population.
To measure Trikafta access, meanwhile, the journal used Vertex’s 2020 annual report, homing in exclusively on fourth-quarter revenues. Trikafta achieved sales of $5.7 billion in 2021, the company said.
Meanwhile, the drug in January snagged a European label extension to treat certain CF patients ages 6 to 11 years old, which should further expand the drug’s reach beyond the 12% figure quoted by the Journal of Cystic Fibrosis. The drug won a similar approval in the U.S. back in June.