Cystic Fibrosis Foundation Responds to ICER Review of CFTR Modulators

The Cystic Fibrosis Foundation responds to the Institute for Clinical and Economic Review's assessment of the comparative clinical effectiveness and value of cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

June 8, 2018 | 4 min read

In early May, the Institute for Clinical and Economic Review (ICER) published an evidence report analyzing the comparative clinical effectiveness and value of ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), and tezacaftor/ivacaftor (Symdeko™). The report found that although the therapies are clinically significant, the prices would have to be cut between 40 and 70 percent to be cost-effective.

As a nonprofit research institute, ICER produces reports that aim to contribute to the overall understanding of the cost and value of specialty therapies. These are important discussions as affordability is a top concern for people with cystic fibrosis as well as for public and private insurers who make coverage decisions.

The Cystic Fibrosis Foundation appreciates ICER's efforts to include clinical and community input throughout the process. Overall, we believe that ICER's final report falls short of conveying the long-term benefit of cystic fibrosis transmembrane conductance regulator (CFTR) modulators and recognizing the impact of CFTR modulators on overall care management. As shared in previous public comments to ICER, we believe the report does not capture important key points about modulators, including:

  • Early initiation and long-term use of these drugs will have profound implications in altering the course of the disease. An early start on modulators could dramatically change standard of care and prevent a host of complications, such as serious lung damage, declining pancreatic function, and challenges with nutrient absorption and weight.
  • CFTR modulators have clinical and quality of life benefits beyond lung function. Forced expiratory volume in one second (FEV1) is an important -- but not the only or best -- sign that a CFTR modulator is having a disease-modifying effect. Focusing on FEV1 undervalues and under-predicts the long-term, systemic benefits that people with CF derive from CFTR modulators.
  • The societal benefits associated with modulators will be seen in time. At best, the model captures a fraction of how modulators affect quality of life, education, and work productivity. 

“We welcome this important discussion as we frequently hear from people with CF who are concerned about their ability to afford these therapies as well as from public and private insurers with concerns about the budget impact of CF care,” said Mary Dwight, senior vice president of policy and advocacy at the CF Foundation.

Dwight added, “While cost-effectiveness analyses can be informative, they must be used carefully and as part of a holistic evaluation of the value a treatment provides. If applied to inform real-world coverage decisions, the inaccuracies and limitations of ICER's model must be recognized. We have serious reservations about the model used to generate this report and are concerned that it does not reflect the clinical significance of CFTR modulators and the realities that patients experience.”

The report was the subject of a public meeting hosted by ICER on May 17, 2018. During the meeting, an independent panel discussed and voted on the comparative clinical effectiveness, benefits or disadvantages, and other contextual considerations of the therapies. The implications of the vote were also discussed among a roundtable of clinical experts and people with CF.

Mary Dwight and Dr. Michael Boyle, senior vice president of therapeutics development, presented on behalf of the Foundation to help the panel understand cystic fibrosis and the complexity of treating the disease.

ICER released the final evidence report on June 7, 2018.


Reference to any specific product, process, or service does not necessarily constitute or imply its endorsement, recommendation, or favoring by the Cystic Fibrosis Foundation. The appearance of external hyperlinks does not constitute endorsement by the Cystic Fibrosis Foundation of the linked websites, or information, products, or services contained therein.

Information contained on this site does not cover all possible uses, actions, precautions, side effects, or interactions. This site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.

FDA-approved drug information is available at

Share this article
Our Advocacy Work
Recent news
The CF Foundation’s 16th Annual Teen Advocacy Day Empowers Teens to Make Their Voice Heard
News | 4 min read
Cystic Fibrosis Community Perspectives Influence Future of CF Care Model
News | 4 min read
CF Foundation Adapts Registry Reporting to New Race-Neutral Standards
News | 5 min read
Stay up to date with The Foundation

Sign up for our newsletter to get all of the latest news from The Foundation right in your inbox.