TEL: 044-952-0102

血液悪性腫瘍のCAR-T療法 - 開発初期における展望:KOL (キーオピニオンリーダー) の考察

CAR-T Therapy in Haematological Malignancy: Early Stage Outlook: KOL Insight [2018]

発行 FirstWord 商品コード 520363
出版日 ページ情報 英文
納期: 即日から翌営業日
詳細については [見積・お問合せ] ボタンよりお問い合わせ下さい。
血液悪性腫瘍のCAR-T療法 - 開発初期における展望:KOL (キーオピニオンリーダー) の考察 CAR-T Therapy in Haematological Malignancy: Early Stage Outlook: KOL Insight [2018]
出版日: 2018年04月01日 ページ情報: 英文

当レポートでは、開発初期段階にある血液悪性腫瘍のCAR-T療法について取り上げ、米国の8名のキーオピニオンリーダー (KOL) による各種見解をまとめています。


  • パイプライン薬
    • tisagenlecleucel-T (CTL019:Novartis)
    • axicabtagene ciloleucel (KTE-019:Kite Pharma)
    • JCAR014 (Juno Therapeutics)
    • JCAR017 (Juno Therapeutics)
    • JCAR018 (Juno Therapeutics)
    • UCARTs (Cellectis/Pfizer/Cellectis)、など


  • 抗CD19 CAR-Tで非常に良好な反応が得られたが、長期的耐久性の重要度は?
    • 高いアンメットニーズを持つ再発/難治性患者において例のない有効性が示された
    • CAR-T療法の長期的耐久性・持続性に対するKOLの見解は?
  • CAR-T療法のメリットがもっとも大きい疾患とその理由は?
    • 抗CD19 CAR-T細胞:急性リンパ性白血病 (ALL)・慢性リンパ性白血病 (CLL)・非ホジキンリンパ腫 (NHL) で研究されている
    • 各疾患におけるCAR-Tの主な市場機会は?
    • KOLがもっとも興味深いと考える市場機会は?
  • 安全性の点で優先されるべきニーズは?
    • サイトカイン放出症候群 (CRS)・神経毒性
    • KOLのCAR-T療法の安全性に対する懸念は?
    • 副作用の管理に関する展望は?
  • 自家CAR-T細胞療法の主な理論的課題とは?
    • 克服の手段は?
    • 自家CAR-T細胞療法の理論的障害とKOLによる克服手段についての見解
  • CAR-T細胞療法の薬価・償還・市場アクセス
    • KOLの見解は?
    • CAR-T療法の主な市場障壁は?保険者は資金面で準備が出来ているか?
  • 次世代CAR-T細胞アプローチについてKOLの関心の高さは?
    • 同種「既製」CAR-T細胞とセーフティスイッチを組み込んだCAR-T細胞:すべてパイプライン初期の段階
    • KOLがもっとも有望と考えるアプローチとその理由は?
  • CAR-T細胞療法と代表的な移植術との比較
    • 造血幹細胞移植 (HSCT):血液腫瘍治療の基礎
    • KOLが見出すHSCTとの類似点は?
    • 専門家はCAR-T細胞療法を移植術に代わるものとみなしているか?
  • どの企業が商業的成功にもっとも近い場所に位置づけているか?その理由は?
    • Novartis・Kite Pharma・Juno Therapeutics間の競合の激化
    • CAR-T細胞療法を前進させるポジションに位置づけている企業は?
  • 企業各社はどのように自社の差別化ができるか?
    • 競争の激しい市場環境にCAR-T療法を組み込むために製造業者がすべきこととは?
  • CAR-T細胞療法の10-15年先の血液腫瘍の治療アルゴリズムへの影響は?
    • アンメットニーズを満たし、将来の治療環境においてニッチな立場を確保できるか?

How are CAR-T cell therapies revolutionising treatment of haematological cancer?

Last year witnessed the FDA approval of the first two anti-CD19 CAR-T cell therapies in two types of blood cancer. Find out how these novel treatments are impacting treatment choices so far and how their introduction is progressing in the US. Eight of the world's leading key opinion leaders (KOLs) from the US and Europe provide their views on the opportunities and challenges that face these exciting treatments across multiple haematological cancer settings and discuss the potential logistical, pricing, reimbursement and market access (PR&MA) hurdles, and how they can be overcome.

Learn how KOLs see the future CAR-T therapy market evolving, and how they expect developers to successfully differentiate their CAR-T therapies in KOL Insight: CAR-T Therapies in Haematological Malignancy. Experts also provide their candid views on next-generation CAR-T approaches and their potential adoption in other blood cancers.

Take a tour of the report now

The table of contents >

The key business questions answered >

The key KOL quotes >

See the therapies covered >

Find out who the 3 EU & 5 US KOLs are >

Review an extract from the report - 1 drug profile >

Top takeaways

  • Kymriah and Yescarta are now FDA-approved but how are they being prescribed in the post-approval setting? Experts reveal how the roll out of CAR-T therapies in the US is progressing so far and what factors impact their prescribing decisions.
  • Which anti-CD19 CAR-T therapy is likely to emerge on top in terms of efficacy and safety? Find out how KOLs compare and contrast the six-month efficacy and safety data reported for Kymriah, Yescarta and JCAR017 in the DLBCL setting.
  • Will CAR-T therapies reach the European market in 2018? European KOLs weigh up the prospects of CAR-T therapy launching in Europe. What are the particular challenges facing CAR-T in the European market?
  • Pricing, reimbursement and market access of CAR-T cell therapy; what are KOLs' opinions? What market barriers are CAR-T therapies facing in the US and Europe and are payers prepared to fund them?
  • Which other disease areas hold the most promise for CAR-T therapy? What are the key opportunities for CAR-T in other disease areas (multiple myeloma, lymphoma and chronic lymphocytic leukaemia [CLL]) and which ones are KOLs most excited about?
  • Which next-generation CAR-T cell approaches fuel KOL excitement? Allogeneic 'off-the-shelf' approaches, dual-targeting CAR-T and combination strategies are in the early-stage pipeline. Which approach do KOLs think offers the most promise and why?
  • Can CAR-T cell therapies compete with gold-standard transplantation? Do experts envisage CAR-T cell therapies eventually sidelining transplantation and what do they expect from key ongoing studies such as Kite/Gilead's ZUMA-7?
  • What are the main clinical and commercial differentiators for CAR-T therapy going forward? Find out if KOLs rank efficacy, safety, ease of use or cost as most important. What do CAR-T manufacturers need to do in order to compete effectively in this landscape?
  • How will CAR-T cell therapies impact future treatment algorithms of haematological cancers in 10 to 15 years? Can CAR-T therapies secure a niche in front-line, consolidation or first-relapse treatment settings?


"There's absolutely no reason that CAR-T should not be at least second-line for DLBCL; patients who relapse with large cell lymphoma are incurable and this is probably the easier and safer therapy." US Key Opinion Leader

"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 Key Opinion Leader

"[CAR-T] 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." EU Key Opinion Leader

Sample of therapies covered

Marketed Therapies

  • Kymriah (tisagenlecleucel-T/CTL019; Novartis)
  • Yescarta (axicabtagene ciloleucel/KTE-019; Kite Pharma/Gilead)

Key Pipeline Therapies

  • JCAR017 (lisocabtagene maraleucel; Juno Therapeutics/Celgene)
  • JCAR014 (Juno Therapeutics/Celgene)
  • JCAR018 (Juno Therapeutics/Celgene)
  • UCARTs (Cellectis/Servier/Pfizer/Allogene)
  • bb2121 (bluebird bio/Celgene)
  • bb21217 (bluebird bio/Celgene)
  • CTL119 (Novartis)
  • AUTO2 (Autolus)
  • AUTO3 (Autolus)
  • CYAD-01 (Celyad)
  • Sleeping Beauty CAR-T cell programme (Ziopharm)
  • Plus other next-generation CAR-T therapy approaches

KOLs interviewed

KOLs from North America

  • Dr. Terry J. Fry MD, Director of Cancer Immunotherapy and co-Director of Human Immunology and Immunotherapy Initiative at the University of Colorado School of Medicine, Denver, Colorado, US
  • Dr. Partow Kebriaei MD, Professor, Department of Stem Cell Transplantation, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas
  • Dr. Frederick Locke MD, Medical Oncologist and Translational Researcher in the Department of Blood and Marrow Transplant and Cellular Immunotherapy at the Moffitt Cancer Center, Tampa, Florida, US
  • Dr. Craig S. Sauter MD, Hematologic Oncologist at the Memorial Sloan Kettering Cancer Center (MSKCC), New York, US
  • One Anonymous US KOL (MD, PhD) leading expert in CAR-T cell research and transplantation

KOLs from Europe

  • Dr. Reuben Benjamin MBBS MRCP FRCPath PhD, Consultant Haematologist and CAR-T cell specialist at King's Hospital London, London, UK
  • Professor Dr. Ulrich Jager MD PhD, Professor of Hematology, Head of the Clinical Department of Hematology and Hemostasis, Medical University of Vienna, Austria
  • Professor Gilles Salles MD PhD, Chair of the Lymphoma Hub, Head of the Haematology Department of the Lyon Sud University, Lyon, France

Money Back Guarantee

At FirstWord, we stand behind our reports. If you're not completely satisfied, we'll refund your money. Guaranteed.

Ongoing Benefits

The world of pharma is ever changing and executives must always be up-to-date with new developments that could affect their own products, position and research. That is why FirstWord's guarantee to keep Therapy Trends clients up to date with Update Bulletins offers a real commercial advantage.

Update Bulletins include expert insight and analysis based on FirstWord analyst re-engagement with the KOLs after major events such as product approvals, key data releases and major conferences to deliver the most valuable insights with each update.

  • Your Therapy Trends Report purchase entitles you to receive three Update Bulletins, which are published approximately every three months for 12 months following the report's publication date.
  • You will receive a copy of each Update Bulletin once available, which are issued each quarter after the publication date.

Table of Contents

1. Executive Summary

2. Research Objectives

3. Research Focus

  • 3.1 What are CAR-T cell therapies?
  • 3.2 Key Indications
  • 3.3 Report Focus

4. Clinical Opportunities and Challenges for CAR-T Therapies

  • 4.1 Efficacy of Autologous anti-CD19 CAR-T therapies
  • 4.2 Safety and Toxicity Management of Autologous anti-CD19 CAR-T Therapies

5. Commercial Opportunities and Challenges for CAR-T Therapies

  • 5.1 Logistics of CAR-T cell therapies
  • 5.2 CAR-T Therapies and Transplantation
  • 5.3 Pricing, Reimbursement and Market Access of CAR-T cell therapies

6. Next-Generation CAR-T Programmes

  • 6.1 Disease Insights: Key Insights for CAR-T Therapies in Other Haematological Malignancies
  • 6.2 CAR-T therapies in multiple myeloma
  • 6.3 CAR-T therapies in other lymphomas, CLL and AML

7. Long-Term Outlook for CAR-T Cell Therapies

  • 7.1 Key insights summary

8. Conclusion

9. Appendix