Cystic Fibrosis

Breakthrough Cystic Fibrosis Treatment on the Horizon

June 7, 2019

Elated. Nervous. Hopeful. That’s how thousands of cystic fibrosis patients felt upon hearing last week’s news: the Food and Drug Administration will soon review a new triple combination therapy that could drastically improve their quality of life.

My Co-Pay Accumulator Story

January 25, 2019

“Important Prescription Plan Change,” the letter announced.  “We’re writing to inform you of an important upcoming change to your specialty prescription benefits.”

The Orphan Drug Disconnect

January 9, 2019

Decades after Congress passed the Orphan Drug Act, only about 10 percent of patients with rare diseases actually receive treatment with orphan drugs, a new report finds.  

Final ICER Report Leaves Cystic Fibrosis Community Uneasy

June 8, 2018

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.”

Cystic Fibrosis Patients Assert Health Care Rights

May 24, 2018

Certain elements are critical to the health care that patients with cystic fibrosis need.  The Cystic Fibrosis Engagement Network has identified those elements in its new Health Care Bill of Rights for Individuals with Cystic Fibrosis, released this week.

Making Treatment Less Rare for Rare Disease Patients

February 26, 2018

Waiting is a fact of life for patients with rare diseases such as cystic fibrosis. Many of these men and women, whose disease causes wheezing and frequent lung infections, live waiting for new and effective medications.  They have to delay activities due to symptoms that make breathing a struggle.  They wait for treatment, fighting through health plan barriers such as prior authorization or awaiting the decision of opaque bureaucracies such as Medicaid pharmacy and therapeutics committees.