ICER Seeking Feedback on CF Treatments from Stakeholders

ICER Seeking Feedback on CF Treatments from Stakeholders

The clinical effectiveness and cost-benefit of several cystic fibrosis (CF) therapies will be the focus of a final evidence report by the Institute for Clinical and Economic Review (ICER), which is currently seeking feedback from CF stakeholders for the report.

The report will mainly evaluate the effects of Vertex Pharmaceuticalselexacaftor (VX-445), tezacaftor, and ivacaftor (Kalydeco) triple combination therapy, whose new drug application was recently given priority review status by the U.S. Food and Drug Administration (FDA). The FDA must come to a decision by March 19, 2020, about whether to approve this combination therapy for CF patients, 12 or older, and carrying certain genetic mutations.

In addition, ICER will include new data that has become available since its last evidence report in May 2018 on three other approved CF therapies, all marketed and developed by Vertex: Symdeko (tezacaftor/ivacaftor), Orkambi (lumacaftor/ivacaftor), and Kalydeco (ivacaftor).

Stakeholder organizations have the opportunity to comment and share relevant information during the open input period until Sept. 25, 2019. Their feedback will be incorporated into the final evidence report.

ICER, an independent nonprofit research institute, will also contact several CF groups and clinical professionals to gather the opinions of patients and medical experts about treatments.

To facilitate and expedite the information-gathering process, ICER has created a series of guides that detail what it considers important data, including the Manufacturer Engagement Guide, Patient Participation Guide, and Patient Guide to Open Input.

Submissions do not have to be limited in length but must be received by Sept. 25, 2019 (5 p.m. ET) for possible inclusion in the evidence report. They can be sent by email to [email protected]

After the open input period, ICER will release a draft scoping document on Sept. 30, 2019, for a public evaluation period of three weeks.

The document will be formally reviewed during a public meeting of the California Technology Assessment Forum that will take place April 30, 2020. The final evidence report is expected to be available May 21, 2020.

Joana holds a MSc in Biology and a MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. She is currently finishing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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Joana holds a MSc in Biology and a MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. She is currently finishing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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