Simplifying Cystic Fibrosis Therapy Leads To Cost Savings And Reduced Treatment Burden

Innovations in Pulmonology, Critical Care & Sleep Medicine | Summer 2024

People with cystic fibrosis (CF) and their caregivers commit large amounts of time, energy and money to accomplish their complex treatment regimens. Fortunately, new medications are not only improving health outcomes, but offering many patients with CF and their caregivers an opportunity to reduce the hardships associated with daily self-care.

Alex Gifford, MD, FCCP

According to an ancillary study of data collected from the SIMPLIFY clinical trial (Impact of Discontinuing Chronic Therapies in People with Cystic Fibrosis on Highly Effective CFTR Modulator Therapy), reducing use of one or both of the most commonly used CF drugs is not inferior to withdrawing them for many patients with CF treated with the newest CFTR modulator, says Alex Gifford, MD, FCCP, Director of the Adult Cystic Fibrosis Program at University Hospitals Cleveland Medical Center.

“SIMPLIFY enrolled a generally healthy population of adults and adolescents with CF taking the triple combination CFTR modulator containing elexacaftor, tezacaftor and ivacaftor, also known as ETI, or by its brand name, Trikafta®,” Dr. Gifford says. “One part of the study compared outcomes between groups randomly assigned to continue or discontinue inhaled dornase alfa (Pulmozyme®) for six weeks; the other part of the study compared the same outcomes between groups randomly assigned to continue or discontinue inhaled hypertonic saline for six weeks. SIMPLIFY found that discontinuing either drug was not inferior to continuing either drug with respect to lung function or respiratory symptoms while patients also took ETI.”

Potential Cost Reductions

Dr. Gifford and a team of investigators from the University of Washington analyzed data from the SIMPLIFY trial and Merative MarketScan Research Databases, which contain information about retail medication costs and prescribing patterns in the United States.  They identified people with CF in the MarketScan Databases from 2020 to 2021, the years during which SIMPLIFY was conducted. They also estimated the annual numbers of prescriptions for dornase alfa, hypertonic saline and ETI and their respective annual costs. They calculated differences in the median costs of outpatient prescription drugs among those who continued versus discontinued dornase alfa or hypertonic saline in SIMPLIFY.

“This ancillary study asked the question: How much money could be saved if people with CF on ETI no longer use dornase alfa or hypertonic saline every day and instead used them only during pulmonary exacerbations,” Dr. Gifford says.

It turns out, in relatively healthy CF patients taking ETI, the potential cost savings of reducing dornase alfa use is substantial: An estimated $1.21 billion of savings annually. Furthermore — because dornase alfa and hypertonic saline manage CF symptoms, while ETI addresses the underlying genetic problem in CF patients — Dr. Gifford says we would expect the incidence of exacerbations to be reduced due to ETI, further lessening the need for these symptom-focused therapies.

Dr. Gifford emphasizes that SIMPLIFY participants tended to be healthier than some people with CF, so findings may not apply to those who are generally sicker, and the cost savings may not be distributed equally throughout the population.

Shared Decision Making

At UH, these findings reinforce the importance of shared decision making between patients and clinicians about treatment continuation and discontinuation.

“With shared decision making, therapy decisions are co-produced, and we discuss all clinical priorities,” Dr. Gifford says. “I’m already advising some of my patients who are doing well to sequentially stop using these drugs, but to immediately report any change in symptoms.”

Leesa Prunty, PharmD, BCPS, BCPPS, Clinical Pharmacy Specialist for Cystic Fibrosis at University Hospitals Cleveland Medical Center, notes that even before the SIMPLIFY trial, patients were already simplifying their own treatment regimens. In a survey of University Hospitals CF patients, more than half indicated they had reduced how frequently they used at least one of their medications.

“This was already happening,” Prunty says, “so it was reassuring to see confirming data come out in a more scientific methodology. It validates our understanding that we should listen to patients when they tell us they feel better. If we can pull back on things, that’s great.”

Time Savings

“Treating CF is time consuming,” Prunty adds, noting by example that nebulized hypertonic saline may take up to 20 minutes twice daily. “So, reducing medications when appropriate offer time and quality of life benefits as well.”

“Whenever patients can avoid time on the nebulizer, it alleviates some of the burden of treatment,” Dr. Gifford says. “These medications are expensive, so it’s important to save money and reduce treatment burden if they are not conferring meaningful health benefits to people with CF who respond well to ETI.”

Dr. Gifford notes that SIMPLIFY was innovative because it focused on confirming non-inferiority of withdrawing, rather than adding, a medication to standard care. Participating in these clinical trials contributes to the collective knowledge about CF treatment and offers University Hospitals patients the most advanced therapeutic options available.

For more information about this study, call Dr. Gifford at 216-286-7415.

Contributing Experts:
Alex Gifford, MD, FCCP
Director, Adult Cystic Fibrosis Program
University Hospitals Cleveland Medical Center
Associate Professor, Cystic Fibrosis Research Center
Case Western Reserve University School of Medicine

Leesa Prunty, PharmD, BCPS, BCPPS
Clinical Pharmacy Specialist - Cystic Fibrosis
University Hospitals Cleveland Medical Center