–Report tentatively expected to review axicabtagene ciloleucel (Kite Pharmaceuticals) and tisagenlecleucel-t (Novartis)–



Boston, Mass., July 19, 2017 – The Institute for Clinical and Economic Review (ICER) will develop a report assessing the comparative clinical effectiveness and value of chimeric antigen receptor T-cell (CAR-T) therapies for treatment of certain types of cancer. The report is tentatively expected to consider two therapies currently under review by the FDA: axicabtagene ciloleucel (Kite Pharmaceuticals) and tisagenlecleucel-t (Novartis).

If approved, axicabtagene ciloleucel is expected to be used in the treatment of relapsed or refractory aggressive non-Hodgkin’s lymphoma in adult patients who are not eligible for autologous stem cell transplant. An approval decision is expected in December of 2017.

Tisagenlecleucel-t is under review for treatment of relapsed or refractory B-cell acute lymphoblastic leukemia in pediatric and young adult patients, with an approval decision expected in September of 2017. Novartis is also expected to file later this year for a second indication for diffuse large B-cell lymphoma in adults.

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 August 4, 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 Guide and ICER’s Patient 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 publiccomments@icer-review.org and must be received by 5 PM ET on August 4, 2017 in order to be considered.

A Draft Scoping Document, which will provide more detail on ICER’s proposed scope for the review, will be available on or about August 9, 2017 and will be open to public comment for three weeks.

The report will be the subject of a March 2018 meeting of the California Technology Assessment Forum (CTAF). 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 CAR-T therapies are included below:
  • Open Input Period: July 19 – August 4, 2017
  • Draft Scoping Document Posted: August 9, 2017
    • Public comment period: August 9- August 29, 2017
  • Final Scoping Document Posted: September 7, 2017
  • Draft Evidence Report Posted: December 19, 2017
    • Public comment period: December 19, 2017 – January 24, 2018
  • Evidence Report Posted: February 15, 2018
  • Public Meeting: March 2, 2018
  • Final Report Posted: March 16, 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.