— Registration now open for April 24 “Early Insights” webinar —
— Public comment period now open until May 9, 2023; Requests to make oral comment during public meeting also being accepted —
BOSTON, April 12, 2023 – The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of exagamglogene autotemcel (“exa-cel”, Vertex Pharmaceuticals and CRISPR Therapeutics) and lovotibeglogene autotemcel (“lovo-cel”, bluebird bio) for sickle cell disease. 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.
“Sickle cell disease can affect nearly every organ system in the body, and severe sickle cell disease affects nearly every aspect of a person’s life,” said ICER’s Chief Medical Officer, David Rind, MD. “From the earliest days of gene therapy, patients, families, and clinicians have imagined that someday it might be possible to address the underlying genetics of sickle cell to achieve a cure. These first two genetic therapies, using different technologies and altering different genetic targets may mean that day has nearly arrived.”
The model developed to generate cost-effectiveness findings in ICER’s draft report used cutting-edge evidence from academic researchers and was informed by what matters to patients. Through collaborative input from patients, clinicians, health economists, payers, and manufacturers, ICER’s draft model not only includes the projected health benefits and cost offsets from reducing the acute events as measured in the clinical studies but also includes the projected benefits from eliminating the fear of future acute events, reductions in chronic events and mortality, health equity considerations, and reductions in lost productivity and caregiver burden.
Based on announcements from Vertex and bluebird bio, we have greater clarity on the earliest likely PDUFA dates for both products. Currently there is only very limited data on exa-cel in the public domain with more expected to be released in coming weeks. As such, ICER has decided to postpone the publication of the Evidence Report to July 13, 2023 to allow time for the Evidence Report to include any newly available information. We will also move the ICER virtual public meeting to July 27, 2023. Click here for the updated timeline.
Register for ICER’s Early Insights Webinar
On April 24, 2023, as part of ICER’s Early Insights Webinar Series, ICER’s Chief Medical Officer, David Rind, MD will present the initial findings of this draft report. This webinar is exclusively available to all users of the ICER Analytics platform; registration for the webinar is now open.
Submit a Public Comment
The Draft Evidence Report and Draft Voting Questions are now open to public comment. All stakeholders are invited to submit formal comments by email to email@example.com, which must be received by 5 PM ET on May 9, 2023. Please submit public comments as a Word document in the following format:
- 12-point Times New Roman font
- Five-page maximum (not including references or appendices)
ICER’s Manufacturer Engagement Guide and Patient Portal 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 July 13, 2023. All comments and ICER’s response to comments will be posted publicly along with the Evidence Report.
Register for the Virtual Public Meeting
The Evidence Report will be the subject of a virtual public meeting of the CTAF on July 27, 2023. During the meeting, the independent council will vote on key questions raised in the report. Registration for the virtual public meeting is now open.
Register to Make an Oral Comment
During the virtual 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 firstname.lastname@example.org by 5 PM ET on May 9, 2023. 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.
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.