In The Know: Analyst answers—What’s next on the horizon for CAR-T cell therapies?

What advances have been made in the CAR-T cell therapy field over the last 12 months? We sat down with Sorcha Cassidy, senior analyst in Oncology & Haematology to dive into recent research. Here she shares some key findings from in-depth interviews with key opinion leaders (KOLs) on the topic, providing a sneak peek into what changes have come to the CAR-T therapy landscape and what experts envision for the near future.

FirstWord: What was the main objective for this research?

Sorcha Cassidy: Many exciting developments have taken place in the haematological cancer space over the past year as we bore witness to the FDA approval of the first two CAR-T cell therapies. The goal of this report was to gain insight into how these therapies are currently perceived by the experts and how their uptake is progressing in the US so far. The research focussed on how world-renowned KOLs see these therapies stacking up in terms of their clinical efficacy, safety, logistics and cost, and more importantly, how they will impact the future treatment landscape.


FW: Based on the research, what is the general sentiment from KOLs on the evolution of the CAR-T therapy landscape?

SC: Overall, the KOLs are extremely optimistic about the use and potential for CAR-T therapies, not only in the relapsed/refractory leukaemia and lymphoma setting but also across many other blood cancers such as multiple myeloma. In the research conducted by FirstWord, the KOLs weigh up many of the opportunities and challenges facing key CAR-T products.  

Some of the key conclusions presented in the research are:

  1. Safety will become a crucial differentiating factor between key anti-CD19 CAR-T products going forward

  2. Cost will heavily factor into European approval and reimbursement decisions

  3. Expansion of CAR-T therapies into other diseases and treatment settings is anticipated

  4. CAR-T therapies have the potential to compete with gold-standard transplantation in second-line treatment settings

  5. Prescribing trends will change significantly in both acute lymphocytic leukaemia (ALL) and diffuse large B-cell lymphoma (DLBCL) with the incorporation of CAR-T therapies


FW: What were some of the most insightful interview quotes? What did they teach us?

SC: When questioned about the key clinical and commercial differentiators for the anti-CD19 CAR-T therapies going forward one expert stated that the ‘ease of use’ of the therapies from a logistical standpoint will be paramount;

“What is going to ultimately differentiate are things like the ease of use of the therapies, not only from the side of the patient but also from the side of the centre. The whole supply chain, logistics, billing and contracting and all these pieces that can be quite complex. The easier they are to use, the more likely that their products will be gravitated towards.” [US KOL]

On discussing the European Medicines Agency (EMA) approval and reimbursement of CAR-T therapies, one specialist is confident of their imminent approval and foresees no major obstacles.

“There is no doubt about the fact that EMA will approve [CAR-T]. Judging from the data plus the experience, I think there is no doubt that both products will be approved.” [EU KOL]

Lastly, when questioned as to the approximate market share potential CAR-T therapies may achieve in the approved indications, one KOL is hopeful that CAR-T therapies eventually could be expanded to 30 percent to 50 percent of patients.

“[CAR-T therapies] will be used anywhere between 10 and 30 percent of patients in general with these diseases. I would not be surprised if it's 50 percent or more.”  [US KOL]


FW: What is one of the most notable changes over the last 12 months in the CAR-T space?

SC: Undoubtedly, the most important change in this space over the past 12 months was the FDA approval of the first two anti-CD19 CAR-T therapies, Novartis’ Kymriah and Kite/Gilead Science’s  Yescarta. A key focus point of this research was to get a handle on how the KOLs are using the two approved products in a commercial setting (outside of clinical trials) and what their real-world experience has been so far. Many next-generation CAR-T approaches are also advancing in the pipeline, such as dual-targeting CAR-T strategies and ‘off-the-shelf’ allogeneic CAR-T therapies, so we wanted to also discover how the experts view these novel approaches.


FW: What is our research team going to explore for the next bulletin on this topic?

SC: Our next update bulletin will focus on three key topics:

  1. Data presented at the 2018 ASCO meeting for Novartis’ Kymriah, Kite/Gilead’s Yescarta and Juno/Celgene’s JCAR017

  2. Views from KOLs on the positive data presented for bluebird bio/Celgene’s anti-BCMA CAR-T therapy bb2121 in patients with heavily pretreated multiple myeloma

  3. New insights on the approval and perception of CAR-T therapies in Europe


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