Stopping dornase alfa while on Trikafta may save $1B

SIMPLIFY clinical trial compared prescription costs of the cystic fibrosis treatments

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Discontinuing dornase alfa while on Trikafta (elexacaftor/tezacaftor/ivacaftor) could cut down on prescription costs by more than $1 billion each year in the U.S., according to estimates based on data from a randomized controlled trial.

The savings would not compromise health, at least not for the short term, as people with cystic fibrosis (CF) who stopped taking dornase alfa or hypertonic saline as part of the six-week trial, dubbed SIMPLIFY (NCT04378153), did not experience a decline in lung function.

“We found that stopping these medications could save money and may allow people with CF to spend less time on treatment, but changes to treatment are important for people with CF to discuss with their providers,” the researchers wrote.

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The data are described in “The cost of simplifying treatments for cystic fibrosis: Implications of the SIMPLIFY trial,” which was published in the Journal of Managed Care & Specialty Pharmacy.

CFTR modulators like Trikafta target the cause of CF by improving the function of the faulty CFTR protein that drives the disease. Before CFTR modulators emerged, managing CF relied largely on treatments such as inhaled therapy to keep symptoms under control.

While CFTR modulators can help ease symptoms of CF, some people continue taking inhaled therapy — for example, dornase alfa (sold as Pulmozyme) or hypertonic saline (a concentrated salt solution) — to clear the lungs from the thick, sticky mucus that marks the disease and helps with breathing.

The SIMPLIFY trial showed that people with CF, ages 12 and older, who had relatively well-preserved lung function were able to safely stop taking dornase alfa or hypertonic saline for six weeks while on Trikafta, without negatively affecting their lung function.

Analyzing prescription costs

Now, the researchers compared prescription costs for those on Trikafta who either continued or stopped taking either dornase alfa or hypertonic saline. They also projected cost differences if these two medications were used only intermittently for symptom relief during exacerbations instead of long periods of time.

The analysis included a total of 392 people with CF who were taking dornase alfa; 193 (49%) were assigned to continue and 199 (51%) to discontinue the medication. Of the 273 people who were using hypertonic saline, 140 (51%) were assigned to continue and 133 (49%) to discontinue the concentrated salt solution.

While the prescription costs of those who were assigned to discontinue hypertonic saline were a median $604 lower over the six-week trial, compared with those who continued it, the adjusted difference was not statistically significant.

Those who stopped taking dornase alfa, however, spent a median $5,860 less during the same time window compared with those who were assigned to continue taking dornase alfa. This would translate into savings of more than $50,000 in a year.

To estimate savings on a national level, the researchers considered that 36,000 people in the U.S. carry CF-causing mutations — that is, at least one copy of the F508del or other responsive mutation — that make them eligible for Trikafta if they are 2 or older.

About two-thirds filled prescriptions for both Trikafta and dornase alfa between 2020 and 2021. If they would discontinue dornase alfa except for intermittent use, which was assumed to cost about $5 million, the total savings would amount to $1.21 billion each year.

Data from longer-term studies needed

Because the SIMPLIFY trial was limited to six weeks, “data from longer-term studies, including the ongoing CF-STORM and HERO-2 (NCT04798014), will be necessary to understand the potential health effects resulting from discontinuing daily use of these medications,” the researchers wrote.

“People with CF and their caregivers spend a lot of time administering treatments,” the researchers wrote. “In addition to cost savings, an important potential benefit of discontinuing or drastically reducing use of these medications is that it would reduce the substantial treatment burden on people with CF.”