–Document open to public comment until  November 20, 2017–
Boston, Mass., October 31, 2017– The Institute for Clinical and Economic Review (ICER) has posted a Draft Scoping Document outlining its planned review of the comparative clinical effectiveness and value of tezacaftor (Vertex Pharmaceuticals) used in combination with ivacaftor (Kalydeco®, Vertex Pharmaceuticals). The 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, as well as ivacaftor monotherapy, approved in 2012.

The scoping document will be open to public comment for three weeks until November 20, 2017 at 5pm ET. It will inform development of a report that will be subject to public deliberation during a May 2018 meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), one of ICER’s three independent evidence appraisal committees.

The Draft Scoping Document was developed with input from key stakeholders, including clinical experts, patients and their families, and patient advocacy groups, who provided important insights into the context of treating CF. In particular, patients highlighted the ability to work and exercise, management of respiratory exacerbations and infections and minimization of consequent hospital stays, and management of gastrointestinal and other symptoms of CF as key priorities in treatment.


ICER will continue to engage with patients, clinicians, drug manufacturers, and other stakeholders to assess appropriate outcomes and comparators for the review, and to enrich the evidence available from clinical trials.

All interested stakeholders are encouraged to submit comments and suggested refinements to the scope to ensure all perspectives are adequately considered. Comments can be submitted by email to publiccomments@icer-review.org and must be received by November 20th at 5pm ET. All comments submitted must meet ICER’s formatting specifications.


ICER’s Patient Participation Guide and Manufacturer Engagement Guide provide additional guidance for submitting public comments, including suggestions for what types of information may be most useful.

Following the public comment period, a revised scoping document will be posted on or about November 30, 2017.

During the May 2018 public meeting, the Midwest CEPAC will vote on key questions raised in the report, and a policy roundtable of patient representatives, clinical experts, drug manufacturers, and payers will discuss recommendations for how best to apply the evidence to policy and practice. The Midwest CEPAC, composed of practicing physicians, methodological experts, and leaders in patient advocacy, provides objective, independent guidance on the application of medical evidence to clinical practice and payer policy decisions.