Report expected to review tezacaftor, lumacaftor, and ivacaftor (Vertex Pharmaceuticals); Open Input accepted until October 27th–

Boston, Mass., October 12, 2017 – The Institute for Clinical and Economic Review (ICER) will develop a report assessing the comparative clinical effectiveness and value of treatments for cystic fibrosis. The report is tentatively expected to include tezacaftor (Vertex Pharmaceuticals) used in combination with ivacaftor (Kalydeco®, Vertex Pharmaceuticals), a combination therapy currently under review by the Food and Drug Administration, with an approval decision expected in February of 2018. The report is also expected to review combination therapy lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals), approved in 2015.

The scope of ICER’s review is subject to change based on feedback received from stakeholders, including clinical experts and patients.

An “Open Input” period begins today, and comments on the topic will be accepted from all interested stakeholders until October 27, 2017 at 5 PM ET. The Open Input period is intended to allow stakeholders to share key information relevant to the development of the evidence report. During this time, ICER will also contact key patient groups and clinical experts to gain further insights on the patient perspective and clinical context of this new treatment option.

For more information about the Open Input period, including further explanation of the types of information that may be most helpful to development of the report, visit ICER’s website. ICER’s Manufacturer Engagement GuidePatient Participation Guide, and Patient Guide to Open Input provide additional information for manufacturers and patient groups.

There are no page limits to Open Input submissions, and input received will be incorporated throughout report development. All input can be emailed to and must be received by 5 PM ET on October 27, 2017 in order to be considered. Patients are also invited to submit input via our online form.

A Draft Scoping Document, which will provide more detail on ICER’s proposed scope for the review, will be available on or about October 31, 2017 and will be open to public comment for three weeks.

The report will be the subject of a May 2018 meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC). During the meeting, the independent panel will deliberate and vote on the comparative clinical effectiveness, benefits or disadvantages, and contextual considerations of the therapy. The panel will discuss the implications of the votes for policy and practice with a roundtable including clinical experts and patients to provide guidance to patients, clinicians, insurers, and policymakers in making informed, evidence-based decisions related to use of the treatments.

The key dates for ICER’s review of therapies for cystic fibrosis are included below:

• Open Input Period: October 12-October 27, 2017

• Draft Scoping Document Posted: October 31, 2017

◦ Public comment period: October 31 – November 20, 2017

• Final Scoping Document Posted: November 30, 2017

• Draft Evidence Report Posted: March 15, 2018

◦ Public comment period: March 15 – April 12, 2018

• Evidence Report Posted: May 3, 2018

• Public Meeting: May 17, 2018

• Final Report Posted: On or about May 31, 2018

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.