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Economists at the Institute for Clinical and Economic Review advise a whopping 77 percent price discount on new CFTR modulators for cystic fibrosis, according to a final evidence report.  The drugs can “substantially improve patient health outcomes,” the group concedes, but their prices exceed ICER’s cost-effectiveness threshold. One manufacturer has called ICER’s analysis “a sham.”

So where does this leave patients?

The odds are already stacked against people with cystic fibrosis, many of them children and young adults.  The disease is marked by chronic coughing and breathing difficulties, making everyday life a challenge.  Hospitalizations are frequent.  And the predicted survival age is only 41 years old.

Treatment options have historically been limited.  CFTR modulator therapies offered a ray of hope, because the drugs treat not just the symptoms of cystic fibrosis but, for a subset of patients, the disease’s underlying cause.  Access to the drugs could be life-changing.  And while that reality may be difficult for ICER to quantify, it’s critical to cystic fibrosis patients and their families.

In an article for the St. Louis Post-Dispatch, former NFL quarterback and cystic fibrosis parent Boomer Esiason explained that patients like his son “undergo therapy and take dozens of medicines daily.”  They spend about two hours a day managing their symptoms, Esiason noted, adding that hospitalizations “for days or weeks at a time” are commonplace, disrupting patients’ “education, work, social life and financial security.”

Though treatment is critical for these patients, access barriers are nothing new.  Patients often face prior authorization requirements such as a minimum age and genetic testing, among other prerequisites.  Now, ICER’s report has the potential to impact health plan coverage for the CFTR modulators, which doesn’t bode well for patients.

The analysis could also dampen future investment in further treatments for the disease.  Though it’s the most common fatal genetic disease in North America, cystic fibrosis affects only about 30,000 people in the United States.  Treatments for rare disease have historically been scarce because of the financial risk they pose for drug manufacturers and their investors.  Policymakers have sought to address the challenge through the 1983 Orphan Drug Act and other incentives.

But to benefit patients and manufacturers alike, the drugs must be accessible.  And, patients fear, ICER’s report may make that much less likely.

To learn more about ICER’s approach to cost-effectiveness analyses, read IfPA’s “The ICER Myth.”

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