ICER Releases Draft Evidence Report on Therapies for Cystic Fibrosis

— Public comment period now open until March 18, 2020; Requests to make oral comment during public meeting also being accepted  —

BOSTON, February 20, 2020 – The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of treatments for cystic fibrosis. ICER’s report focuses on elexacaftor/tezacaftor/ivacaftor (TrikaftaTM, Vertex). ICER also examined new data that has become available since its May 2018 review of three other cystic fibrosis treatments already approved by the FDA: tezacaftor/ivacaftor (Symdeko®, Vertex), lumacaftor/ivacaftor (Orkambi®, Vertex), and ivacaftor (Kalydeco®, Vertex).This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document should not be interpreted to be ICER’s final conclusions.

Submit a Public Comment

The Draft Evidence Report and Draft Voting Questions are now open to public comment until 5 PM ET on March 18, 2020. All stakeholders are invited to submit formal comments by email to publiccomments@icer-review.org. Guidelines for submitting public comments, including formatting specifications, are available on ICER’s website. ICER’s Manufacturer Engagement Guide and Patient Participation Guide provide additional detail on what types of information may be most informative to the report.
ICER will review all comments and incorporate any necessary changes in the Evidence Report and Revised Voting Questions that will be posted on or about April 9, 2020. All comments and ICER’s response to comments will be posted publicly along with the Evidence Report.
 
Register for the Public Meeting
 
The Evidence Report will be the subject of a public meeting of the CTAF on April 30, 2020 in Los Angeles, CA. During the meeting, the independent council will vote on key questions raised in the report. Registration for the public meeting and live webcast is now open.
 
Register to Make an Oral Comment
 
During the public meeting, there will be a limited amount of time available for interested stakeholders to make an oral comment on the report. Requests to submit oral comments must be emailed to publiccomments@icer-review.org by 5 PM ET on March 18, 2020. Individuals who wish to deliver oral comments must separately register to attend the meeting. For more information about registering for oral comment, please visit our website.
About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.