Trials Data Raises Hopes in Cystic Fibrosis Community

Promising clinical trials data has excited patients with cystic fibrosis and their families.  But will access barriers dash their hopes for more and better treatment options?

Clinical Trials Findings

Patients have long suffered a dearth of effective treatments for cystic fibrosis, a genetic condition that causes mucus to cover the lungs and makes it difficult to breathe.  New phase 3 clinical trials datasuggests that a combination treatment, tezacaftor and ivacaftor, significantly improves lung function for patient with one or two copies of a specific genetic mutation.

Compared to placebo, cystic fibrosis patients taking the combination therapy saw a clinically meaningful improvement in lung function and a 35 reduction in pulmonary complications.  Patients taking the combination treatment saw greater improvement than did the patients taking ivacaftor alone.

Potential Access Barriers

The combination therapy must undergo the Food and Drug Administration’s approval process before becoming available to patients.  The experience of past innovative cystic fibrosis treatments, however, suggests a troubling precedent for patients’ ability to actually access the medications once they come to market.

[Read: Orphan Drug Access Still Eludes Some Cystic Fibrosis Patients]

In 2016, a study revealed that not even half of eligible patients could access another combination therapy for cystic fibrosis, lumacaftor/ivacaftor.  And several Medicaid systems have established extensive prior authorization processes for ivacaftor monotherapy.   Arkansas, for example, required patients to demonstrate 12 months of failure on traditional cystic fibrosis medications before accessing the treatment.  Legal action ensued, leading Arkansas to revise its requirements.  But similar tactics seem likely to re-emerge given the high price tag that these innovative therapies often carry and the proportion of cystic fibrosis patients who depend upon Medicaid for their health care.

Such access barriers are especially troubling given that newer, innovative treatments address the cause of cystic fibrosis, while some older treatments target only the symptoms.

About Cystic Fibrosis

Cystic fibrosis is the most common fatal genetic disease in North America, affecting about 30,000 Americans.  Though advanced treatments have expanded patients’ lifespan and improved their quality of life, the median age of death remains 29.  No cure exists.

To learn more about cystic fibrosis and its patients’ access to treatment, visit the Cystic Fibrosis Engagement Network website.

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