ICER Report Dampens Prospects for Cystic Fibrosis Treatment

March 23, 2018

If health plans heed a new report from the Institute for Clinical and Economic Review, patients with few options to treat their cystic fibrosis may soon have fewer still.

In its recent assessment, ICER finds innovative cystic fibrosis drugs called CFTR modulator therapies too expensive. The therapies are the first medication to address the underlying cause of cystic fibrosis, while past treatments addressed only the disease’s symptoms. CFTR modulator therapies don’t work for every patient, but they are life changing for those who can use them.

ICER acknowledges the drugs’ benefit.  CFTR modulators “substantially improve patient health outcomes,” the report notes.

But the value of that benefit is another matter. ICER’s analysis still argues that “drug prices would need to be reduced by about half” for the therapies to meet ICER’s benchmark for cost effectiveness.

That could be bad news for patients.  ICER’s cost assessment may intensify access restrictions, as insurance companies look to the organization’s report to inform their coverage. Drugs deemed too costly can receive less favorable coverage or get saddled with more onerous utilization management restrictions.

Some cystic fibrosis patients and their families are already overwhelmed by such restrictions.  Cost and coverage barriers can keep drugs from being broadly accessible, according to the Cystic Fibrosis Engagement Network. Even ICER confirms this reality, acknowledging prior authorization requirements such as a minimum age and genetic testing, among other prerequisites.  Cystic fibrosis patients must satisfy these before health plans will consider paying for their medication.

Now ICER’s valuation could increase these patients’ pain.  Without effective treatment, these patients are left to carry on with pills, inhalers and time-consuming treatments – tolerating persistent coughing and fighting for each breath.  But it seems these quality-of-life considerations just don’t carry the same weight for health economists as they do for patients.

The public comment period for ICER’s draft evidence report on modulator therapies for cystic fibrosis closes April 12.

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