Trikafta cost in CF seen to far exceed medical savings it provides

New analysis does confirm benefits of Vertex therapy for patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A hand holds up a coin among dollar signs and stacks of money.

In the months after starting on Trikafta, people with cystic fibrosis (CF) generally experience lower rates of hospitalizations and lung infections — but nonetheless, the total cost of healthcare for these patients tends to increase, a new economic analysis indicates.

The findings are in line with prior analyses suggesting that, while Trikafta offers benefits to CF patients, its current price is higher than the economic value it provides, the researchers said.

Specifically, the new analysis found CF patients starting for the first time on therapy saw their average pharmacy costs increase by more than $150,000 “prestudy to poststudy 180 days.” Meanwhile, however, the average costs of medical benefits for these patients were reduced by less than $20,000 across the same period. In sum, it cost more than $9.50 for these treatments for each $1 of overall savings.

“If we were to account for the current manufacturer patient assistance program maximal annual benefit of $20,000 ($10,000 benefit over 180 days), our results would show a pharmacy benefit spend of $9.02 to save $1 of medical benefit spend,” the researchers wrote.

The study, “Real-world outcomes and direct care cost before and after elexacaftor/tezacaftor/ivacaftor initiation in commercially insured members with cystic fibrosis,” was published in the Journal of Managed Care & Specialty Pharmacy. The work was funded by pharmacy benefit management company Prime Therapeutics.

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Patient cost for Trikafta: more than $300,000 annually

Trikafta (elexacaftor/tezacaftor/ivacaftor) is a triple-combination therapy containing three CFTR modulators. These molecules can boost the functionality of the mutated CFTR protein in people with CF caused by specific mutations.

CFTR modulators are a relatively new class of medications for cystic fibrosis — the first such therapy was approved in the U.S. in 2012. While shown to be effective, these treatments come with a high price tag: The cost of Trikafta is more than $300,000 annually.

The therapy, sold by Vertex Pharmaceuticals, is approved in the U.S. for patients ages 2 and older who have at least one copy of F508del, the most common CF-causing mutation, or a mutation that responds to Trikafta in laboratory studies.

Clinical trials and real-world data have demonstrated that Trikafta can help to improve lung function and ease other CF symptoms in eligible patients. However, economic analyses have suggested that, while the therapy is effective, its annual list price of $311,741 per year is much higher than the value it delivers.

Now, a team of scientists at Prime conducted an analysis aiming to evaluate how starting on Trikafta affected short-term healthcare spending for people with CF in the real world.

Using an insurance and pharmacy database, the team identified 494 people with CF in the U.S. who started on Trikafta between 2019 and 2021. A little more than half of these patients were male, most lived in the South or the Midwest, and more than two-thirds were on pancreatic enzyme replacement therapy.

From the available data, the researchers compared outcomes in the 180 days (about six months) before initiating Trikafta to the 180 days after starting on the therapy.

The findings indicated that the rate of pulmonary exacerbations — a sudden worsening of lung function, usually due to infection — was significantly lower, by 44.1%, in the months after starting on Trikafta. The rate of hospitalizations also was significantly lower, by 61.9%, and the rate of emergency department visits decreased by 42.8%.

“Our findings highlight the real-world clinical effectiveness of [Trikafta] demonstrating a substantial reduction in the total number of pulmonary exacerbation events, hospitalizations, and emergency department visits,” the researchers wrote.

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Cost to manufacture Trikafta: less than $6,000 annually

In the 180 days prior to starting on Trikafta, the average total cost of healthcare was estimated at $58,180. In the six months after starting on the therapy, the average cost more than doubled, to $198,815.

This increase in cost was mostly attributable to the cost of Trikafta itself, which totaled an average of $156,629 per patient. In fact, costs for non-pharmacy medical services decreased significantly, from an average of $28,764 to $12,484 after patients started on Trikafta.

Taking these numbers together, the researchers estimated that Trikafta adds more than $9 worth of pharmacy cost for every $1 in medical savings it affords by reducing the need for other healthcare resources. These findings “are consistent with previous analyses showing that CFTR modulator therapy direct costs exceed direct medical cost offset,” the researchers wrote.

It is essential for therapies to be priced based on value. … The rising drug costs ultimately drive-up [health insurance] premiums for all covered individuals, not just those with CF.

Importantly, the team also noted that the list price of Trikafta far exceeds the cost to manufacture the medication, estimated at less than $6,000 per patient per year.

“It is essential for therapies to be priced based on value,” the scientists wrote, noting that the high costs of therapies “are straining health care affordability.”

“The rising drug costs ultimately drive-up [health insurance] premiums for all covered individuals, not just those with CF,” they wrote.

The scientists noted that this analysis was limited only to patients with commercial insurance, and it only considered spending directly related to healthcare. Thus, the findings are limited to direct medical cost offsets.