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遺伝子治療の商業化 - 機会と障壁

Datamonitor Healthcare Strategy: Gene Therapy Commercialization - Opportunities and Barriers

発行 Datamonitor Healthcare 商品コード 858342
出版日 ページ情報 英文 144 Pages
納期: 即日から翌営業日
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本日の銀行送金レート: 1USD=111.24円で換算しております。
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遺伝子治療の商業化 - 機会と障壁 Datamonitor Healthcare Strategy: Gene Therapy Commercialization - Opportunities and Barriers
出版日: 2018年10月25日 ページ情報: 英文 144 Pages
概要

当レポートでは、遺伝子治療の商業化について調査し、承認済み遺伝子治療の情勢、米国およびEUにおける医療費償還の動向、規制、および課題などについてまとめています。

概要

承認済み遺伝子治療の情勢

  • Datamonitor によるヘルスケア考察・提言
  • 遺伝子治療:50年にわたって存在する分野、しかし進歩が遅れている
  • これまでに世界全体で11の遺伝子治療が承認済み
  • 参考資料

遺伝子治療の価格・医療費償還

  • Datamonitor によるヘルスケア考察・提言
  • 主な承認済み遺伝子治療の治療費
  • ある種の問題は遺伝子治療の償還を課題に
  • 血友病の遺伝子治療は最も費用を相殺
  • 遺伝子治療の支払いオプション、ほか

米国の医療費償還

  • Datamonitor によるヘルスケア考察・提言
  • 遺伝子治療のためのバリューフレームワークはまだ存在しないが、ICERの役割は拡大している
  • 民間保険者の遺伝子治療向け保険保証範囲
  • CAR-T治療のMedicare保証範囲
  • 米国における主要な遺伝子治療の価格は場合によって高額、その他においては低い
  • 参考資料

EUの医療費償還

  • Datamonitor によるヘルスケア考察・提言
  • 価格およびアクセス制限による遺伝子治療の進歩は遅い
  • 欧州における次の大きな試験はCAR-T治療
  • HTA評価において、欧州の保険者は単一群試験および/または小規模サンプルによるデータを用いた製品にペナルティを科す可能性は低い
  • 参考資料

製造ガイダンスおよびベクター供給の検討事項

  • Datamonitor によるヘルスケア考察・提言
  • 規制ガイダンス
  • ベクターの製造・供給における課題・障壁
  • 参考資料

EX VIVO (生体外)/細胞および遺伝子治療の製造における課題

  • Datamonitor によるヘルスケア考察・提言
  • 製品としてのプロセスにより、細胞・遺伝子療法は大きなパラダイムシフトを示す
  • ベッドサイドCAR-Tプロセスの検討事項
  • 原料出発原料/における工程変数は特に重要
  • CAR-T療法における製造物流
  • 参考資料

付録

目次
Product Code: DMKC0198970

Overview

Commercialization of modern-day gene therapies is now a reality. Next-generation modalities such as chimeric antigen receptor T-cell (CAR- T) therapies are fully in launch mode in the US, with final approvals having also occurred in the EU. The first in vivo gene therapy for an inherited disease is also now available in the US. With a slew of gene therapies in the pipeline, across ex vivo and in vivo applications, and several indications including oncology and rare disorders, more treatments are advancing into later-stage development and onto potential regulatory approval over the next several years. Gene therapy developers can take advantage of a multitude manufacturing guidelines on gene therapy in the US and EU.

TABLE OF CONTENTS

OVERVIEW

APPROVED GENE THERAPY LANDSCAPE

  • Datamonitor Healthcare insights and recommendations
  • Gene therapy: a field in existence for 50 years, but setbacks have slowed progress
  • Across the globe, 11 gene therapies are approved to date
  • Bibliography

PRICING AND REIMBURSEMENT OF GENE THERAPIES

  • Datamonitor Healthcare insights and recommendations
  • Therapy costs of select approved gene therapies
  • Certain challenges make reimbursement of gene therapies an issue
  • Hemophilia gene therapies may benefit the most in showing offset of costs
  • Payment options for gene therapies
  • Early engagement with US and EU payers would be beneficial
  • The use of registries and real-world evidence in long-term follow-up will be important for payers
  • Patient advocacy will be important in supporting access for gene therapies
  • Bibliography

US REIMBURSEMENT

  • Datamonitor Healthcare insights and recommendations
  • Value frameworks for gene therapy do not yet exist, but ICER's role is growing
  • Commercial payer coverage policies for gene therapies
  • Medicare coverage of CAR-T therapies
  • Pricing for initial gene therapies in the US is perceived as high in some cases, lower in others
  • Bibliography

EU REIMBURSEMENT

  • Datamonitor Healthcare insights and recommendations
  • Progress of gene therapies has been slow because of price and access restrictions
  • The next big test in Europe will be CAR-T therapies
  • In HTA assessments, European payers are unlikely to penalize products with data from single-arm trials and/or small sample sizes
  • Bibliography

MANUFACTURING GUIDANCE AND VECTOR SUPPLY CONSIDERATIONS

  • Datamonitor Healthcare insights and recommendations
  • Regulatory guidance
  • Challenges and barriers in vector manufacturing and supply
  • Bibliography

ISSUES IN MANUFACTURING EX VIVO/CELL AND GENE THERAPIES

  • Datamonitor Healthcare insights and recommendations
  • With the process as the product, cell and gene therapies represent a major paradigm shift
  • Bedside CAR-T processing considerations
  • Process variability in raw/starting materials is of particular importance
  • Manufacturing logistics in CAR-T therapy
  • Bibliography

APPENDIX

  • Scope
  • Methodology

LIST OF FIGURES

  • Figure 1: Approved gene therapies by country, September 2018
  • Figure 2: Gene therapy pipeline by therapy area
  • Figure 3: Key challenges to reimbursement of gene therapies
  • Figure 4: Ethical concerns related to hypothetical gene therapy for brain-related conditions
  • Figure 5: The Alliance for Regenerative Medicine's suggested value framework for gene therapies
  • Figure 6: Top-level additional inputs for payers to consider when assessing regenerative medicines
  • Figure 7: Kymriah and Yescarta are forecast to pass $2bn in peak sales, 2018-26
  • Figure 8: Gene therapy pipeline by vector category and viral vector type
  • Figure 9: Process and unit operations in autologous CAR-T therapy manufacturing
  • Figure 10: Heat map of Kymriah and Yescarta CAR-T therapy certified centers in the US, August 2018
  • Figure 11: Open versus closed systems: benefits and challenges

LIST OF TABLES

  • Table 1: Approved gene therapies worldwide, September 2018
  • Table 2: Therapy costs of select approved gene therapies
  • Table 3: Late-stage hemophilia gene therapies in the pipeline
  • Table 4: Financing and payment options for gene therapies
  • Table 5: Summary of the main recommendations on utilization of registry data in supporting regulatory benefit-risk evaluations for CAR-T therapies
  • Table 6: Major US payer restrictions or selection criteria on gene therapies
  • Table 7: Medicare outpatient rates for CAR-T therapies
  • Table 8: ICER discounted lifetime costs and outcomes for Kymriah and Yescarta versus comparators
  • Table 9: ICER threshold analysis for Kymriah and Yescarta
  • Table 10: Estimated total cost of CAR-T treatment at the low and high end
  • Table 11: ICER incremental cost-effectiveness ratios for Luxturna
  • Table 12: Market access decisions for approved gene therapies in the five major EU markets
  • Table 13: Select gene therapies in the pipeline with pivotal single-arm trial designs
  • Table 14: Summary of EMA guidance on gene therapy manufacturing
  • Table 15: Summary of FDA guidance on gene therapy manufacturing
  • Table 16: Select group of CMOs and CDMOs with gene therapy vector supply capabilities
  • Table 17: In-house and external cell and vector manufacturing efforts by select gene therapy developers
  • Table 18: Vein-to-vein time comparison for CAR-T therapies
  • Table 19: Select companies that specialize in cell and gene therapy biopreservation
  • Table 20: US treatment centers for Kymriah and Yescarta, August 2018
  • Table 21: Select companies specializing in software tracking tools for cell and gene therapy
  • Table 22: Potential innovations in improving scale-out of cell and gene therapies
  • Table 23: Select companies that offer closed cell-processing systems or technologies
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