–Report expected to review axicabtagene ciloleucel and tisagenlecleucel-t, first CAR-T therapies submitted for FDA review– 

Boston, Mass., August 9, 2017- The Institute for Clinical and Economic Review(ICER) has posted a Draft Scoping Document on chimeric antigen receptor t-cell (CAR-T) therapies for treatment of B-cell malignancies. The document will be open to public comment for three weeks until August 29, 2017 at 5pm ET. It will inform a report assessing the comparative clinical effectiveness and value of CAR-T therapies, which will serve as the basis of discussion during a March 2018 public meeting of the California Technology Assessment Forum (CTAF).

ICER’s report is expected to consider the comparative clinical effectiveness and value of two therapies currently under review by the FDA: tisagenlecleucel-t (CTL-019, Novartis) and axicabtagene ciloleucel (KTE-019, Kite Pharma). FDA approval decisions are expected in September and December of 2017, respectively.  If approved, tisagenlecleucel-t is expected to be used in the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia in pediatric and young adult patients. Novartis is also expected to file later this year for a second indication for diffuse large B-cell lymphoma in adults. Axicabtagene ciloleucel is under review for the treatment of relapsed or refractory aggressive non-Hodgkin’s lymphoma in adult patients who are not eligible for autologous stem cell transplant.

The Draft Scoping Document was developed with input from key stakeholders, including patients and patient advocacy groups, who provided important insights into the context of treatment decisions. Of note, patients expressed hope that CAR-T therapy would offer improved survival and better quality of life compared to other treatments they had tried. While CAR-T therapies come with risks of their own, such as cytokine release syndrome and neurotoxicity, patients were optimistic about avoiding the toxicities of treatments like chemotherapy or stem cell transplants, which may be accompanied by prolonged hospitalizations and serious cardiovascular, hematologic, and infectious complications. 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. All comments must be received by August 29th at 5pm ET. Comments can be submitted by email to publiccomments@icer-review.org. More information about submitting a public comment, including formatting specifications, is available on ICER’s website. ICER’s Patient Participation Guide and Manufacturer Engagement Guide provide additional guidance for submitting public comments.

Following the public comment period, a revised scoping document will be posted on or about September 7, 2017.

During the March 2018 public meeting, the CTAF panel 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. CTAF is one of ICER’s three independent evidence appraisal committees composed of practicing physicians, methodological experts, and leaders in patient advocacy. The Panel provides objective, independent guidance on the application of medical evidence to clinical practice and payer policy decisions.

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.

Additional information regarding the report and meeting are available on ICER’s website.