No one disputes the therapeutic value of Vertex Pharmaceuticals’ triplet drug Trikafta for cystic fibrosis—not even the cost watchdogs at the Institute for Clinical and Economic Review (ICER).
But ICER has plenty of bones to pick with Vertex over Trikafta’s $311,000 list price.
So when the institute released its final report on the cost effectiveness of cystic fibrosis drugs today, in which it awarded Trikafta its highest “A” rating for the strength of the evidence that the drug improves patient outcomes, it also issued an “Access and Affordability” alert. “Unfortunately, the manufacturer has leveraged its monopoly to set a price—costing many millions of dollars over the lifetime of an average patient,” David Rind, M.D., ICER’s chief medical officer, said in a statement.
ICER studied four subsets of CF patients with different variations of the delF508 CFTR gene mutation that the drug targets. The agency concluded that for three of them, Trikafta provides a “substantial net health benefit,” and for the fourth, the benefit is at least the same, if not better, than that provided by Vertex’s older CF drug Symdeko.
Trikafta pairs a CFTR modulator with Symdeko, which is itself a combination that includes another Vertex CF drug, Kalydeco. That addition boosts the eligible CF patient population to 90% from the 50% the company’s older drugs were cleared by the FDA to treat. Analysts have estimated Trikafta will be pulling in $6.6 billion in sales by 2025.
ICER determined that all of Vertex’s CF treatments, including Trikafta, have a cost per quality-adjusted life year (QALY) gained of more than $1 million. That figure “is substantially higher than cost effectiveness thresholds in the U.S. and the rest of the world,” the report (PDF) said.
To bring the cost of Trikafta in line with the health benefits it provides, Vertex would need to lower the price to between $67,900 and $85,500 per year, ICER concluded. Maintaining the higher price will “cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance,” the agency contended.
ICER released a draft of its CF review in April, when it was likely overshadowed by the COVID-19 pandemic. Nevertheless, the agency started raising alarm bells over Trikafta’s price back then, publishing a letter to the CF community expressing concern that Vertex’s ownership of the market for drugs to treat the condition would ultimately harm all patients.
“Our broader view is that when we as a nation give a company a monopoly on a treatment and, instead of ‘wrestling’ with them over coverage, tacitly agree that coverage WILL be provided because we want all patients to benefit, we need some mechanism to suggest an upper limit to the price that a company feels it can charge,” ICER said.
Neither ICER’s criticisms nor the pandemic have dampened Vertex’s Trikafta launch, however. The drug hauled in $895 million in its first full quarter on the market, prompting the company to raise its 2020 sales projections.
Vertex CEO Reshma Kewalramani doesn’t seem concerned about pricing controversies. In August, the drug was approved in Europe, where it’s called Kaftrio. Kewalramani said during Vertex’s second-quarter earnings announcement that the company has reimbursement deals with the National Health Service in England, as well as payers in Ireland and Denmark.
“The rate of uptake for Trikafta and the speed at which we have obtained broad reimbursement speak to the appreciation of the therapeutic profile of this medicine, which we believe will be the foundation of CF therapy for many years to come,” Kewalramani said.