Tisagenlecleucel is a chimeric antigen receptor (CAR) T-cell therapy for the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma (r/r-DLBCL) and children and young adults (25 years or younger) with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r-ALL).

Tisagenlecleucel was approved by the FDA for the treatment of adult r/r-DLBCL in May of 2018, and for the treatment of pediatric r/r-ALL in August of 2017. Prior to implementation in Canada, various stakeholders will need to evaluate the projected number of patients eligible for tisagenlecleucel, models of care, and the feasibility of an integrated work plan for identifying pediatric and adult populations with appropriate indications for tisagenlecleucel therapy. Questions about clinical effectiveness, cost-effectiveness, and implementation considerations (such as locations for treatment, system capacity, and clinical delivery protocols) will also need to be addressed. Contingency plans must be established if a scheduled tisagenlecleucel infusion cannot proceed because of manufacturing error, if the patient becomes too ill to receive the infusion, or dies during the waiting period after leukapheresis. Differences in the practice and use of CAR T-cell therapy between jurisdictions may result from variations in the availability or capacity to establish logistical and workforce requirements to deliver CAR T-cell therapies in the various provinces and territories.

Contents

• Background and Rationale
  • Clinical Need and Target Population
  • A Brief Overview of CAR T-cell Therapy and Tisagenlecleucel
  • Rationale for the Review
• Policy Question
• Objectives
• Research Questions
• Methods
  • Literature Search Strategy
  • Study Design
  • Study Eligibility
  • Literature Screening and Selection
  • Data Extraction
• Quality Assessment of Studies
• Data Analysis
• References
• Appendix 1. Therapy Procedure Overview
• Appendix 2. Review Framework
• Appendix 3. Clinical Studies Screening Checklists
• Appendix 4. Clinical Search Strategy

Suggested citation:

Tisagenlecleucel for B-Cell Acute Lymphoblastic Leukemia and Diffuse Large B-Cell Lymphoma — Project Protocol, Clinical Section. Ottawa: CADTH; 2018 Aug. (CADTH optimal use report; vol.8, no.3a).

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