Cystic Fibrosis

CTAF

Cystic fibrosis is an autosomal recessive condition caused by mutations in the CFTR gene. It is relatively rare, occurring in approximately 1 in 2,500 to 3,000 livebirths, but is the most common, lethal genetic disease in Caucasian populations. CF is a progressive disease that affects many organ systems, but most of its morbidity and mortality are associated with its impact on the respiratory system.

Interventions of Interest:

    • Tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals)
    • Lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals)
    • Ivacaftor (Kalydeco®, Vertex Pharmaceuticals)
    • Elexacaftor/tezacaftor/ivacaftor (Vertex Pharmaceuticals)

Date of review: April 2020

Click here to see ICER’s 2018 cystic fibrosis review

For questions, please contact Matt Seidner, Program Director, at mseidner@icer-review.org.

ASSOCIATED MEETING & MATERIALS

CTAF
April 30, 2020 10am-4pm ET

TBA

The California Technology Assessment Forum (CTAF) will convene to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis.

Key Dates

Associated Materials

09/06/2019 – 09/25/2019
Open Input Period

09/30/2019

09/30/2019 – 10/21/2019

09/30/2019

10/29/2019

12/05/2019
Research Protocol

01/06/2020
Model Analysis Plan

02/20/2020
Draft Evidence Report

02/20/2020
Draft Voting Questions

02/20/2020 – 03/18/2020
Public Comments

04/09/2020
Revised Voting Questions

04/09/2020
Response to Public Comments

04/09/2020
Evidence Report

04/30/2020
Meeting Agenda

04/30/2020
Evidence Presentation

05/21/2020
Final Evidence Report and Meeting Summary

05/21/2020
Report-at-a-Glance


ASSOCIATED MEETING & MATERIALS

Midwest CEPAC
May 17, 2018 9:30am-3:30pm CT

ICER’s first CF review
University of Missouri-St. Louis
Century BC Room, Millennium Student Center
1 University Boulevard
St. Louis, MO 63121

The Midwest CEPAC will convene to discuss treatments for cystic fibrosis.

Key Dates

Associated Materials

10/11/2017 – 10/27/2017

10/31/2017

10/31/2017

10/31/2017 – 11/20/2017

11/30/2017

02/07/2018

02/07/2018

03/15/2018

Open to public comment from March 15 – April 12, 2018. For more information on submitting a public comment, please visit our stakeholder engagement page.