Tisagenlecleucel (1) – Kymriah®

• Clinical trials
  • The JULIET trial is a single-arm, multicentre Phase II trial designed to assess the safety and efficacy of tisagenlecleucel in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

Adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) following two or more lines of systemic therapy

• Consequently, the G-BA classifies the extent of the additional benefit of tisagenlecleucel alone, from a legal perspective, as unquantifiable on the basis of the criteria set out in Section 5(7) of the AM-NutzenV, taking into account the severity of the disease and the therapeutic objective in treating the disease, is non-quantifiable.
• An additional benefit exists in accordance with Section 35a(1), sentence 11, first half-sentence, of SGB V, but is non-quantifiable because the scientific evidence does not permit this.
• Overall assessment
  • For the assessment of the extent of the additional benefit of tisagenlecleucel for the treatment of relapsed or refractory diffuse large B-cell lymphoma, the pivotal single-arm Phase II JULIET trial provides results on mortality, morbidity, quality of life and side effects.
  • The data submitted for the JULIET study are incomplete. The current data sets lack fundamental information on the conduct and progress of the study, meaning that these data sets cannot be used for the benefit assessment.
  • The data from the primary data cut-off of 8 March 2017, which is usable for the benefit assessment, are subject to considerable uncertainty due to the fact that patient recruitment has not been completed and the median follow-up period is very short.
  • Furthermore, no overall survival analyses based on the ITT population are available for this data cut-off.
  • Inherent components of treatment with tisagenlecleucel include leukapheresis, the waiting time until the product becomes available and the administration of bridging chemotherapy frequently associated with this, as well as lymphocyte-depleting chemotherapy. The impact of these components on the treatment of patients with tisagenlecleucel in a clinical care setting can only be adequately assessed by considering the ITT population.
  • Overall, due to the lack of data, no reliable substantive conclusions can be drawn regarding the extent of the additional benefit.
  • Based on the indirect historical controls presented, and given the limited data available from the JULIET study as well as further uncertainties regarding comparability with the studies used for the indirect comparison, sufficiently valid conclusions regarding the extent of the additional benefit of tisagenlecleucel cannot currently be drawn.

Courtesy translation only, please refer to the German original.