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血友病A遺伝子治療の世界市場:主要なオピニオンリーダー(KOL)の考察(2020年)

Hemophilia A Gene Therapy: Key Opinion Leaders (KOLs) Insight - An Executive Report 2020

出版日: | 発行: Mellalta Meets LLP | ページ情報: 英文 54 Pages | 納期: 即納可能 即納可能とは

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血友病A遺伝子治療の世界市場:主要なオピニオンリーダー(KOL)の考察(2020年)
出版日: 2020年08月18日
発行: Mellalta Meets LLP
ページ情報: 英文 54 Pages
納期: 即納可能 即納可能とは
担当者のコメント
先天性の血液凝固障害で、量的質的な異常症に該当する血友病A疾患の遺伝子治療に着目した調査レポートがMellalta Meets社よりリリースされました。米国、欧州における14のKOLへの詳細インタビューを実施した結果を基にした調査レポートになり、KOL考察の詳細をまとめた市場調査レポートになります。まずはサンプルページで概要を把握いただける資料を送付させていただきますので、お問い合わせください。
  • 全表示
  • 概要
  • 目次
概要

当レポートは、世界の血友病A遺伝子治療市場について調査し、現在の遺伝子治療の課題、安全性と有効性の懸念、市場浸透、遺伝子治療の競合、血友病A遺伝子治療の再開、HEMLIBRAおよび因子補充療法から遺伝子治療へ切り替える患者に対する理解に向けた競争上の優位性、および主要な米国および欧州のKOLとの14の詳細なインタビューを通じた、その他の重要な質問などを提供します。

目次

第1章 臨床的展望

  • エグゼクティブサマリー
  • 血友病遺伝子治療に関するKOLの展望
  • KOLの目から見たGTの安全性と有効性の懸念
  • 遺伝子治療の再投薬 - KOLの考察および競合情勢
  • 子供のための遺伝子治療

第2章 商業的展望

  • エグゼクティブサマリー
  • 推定患者:Roctavianの市場浸透および規模予測(2028年まで)
  • 血友病A遺伝子治療の競合情勢状況とKOLの意見
  • KOLの見解に基づくバイオマリンの遺伝子治療市場の取り込み
  • 患者は米国と欧州でヘムリブラと因子補充から遺伝子治療に切り替える

第3章 支払者の視点

  • エグゼクティブサマリー
  • 米国および欧州における遺伝子治療価格に関するKOLおよび患者の順応性
  • 遺伝子治療の償還
  • GT処方の意思決定プロセスにおけるハードエンドポイント
  • GTの処方における影響因子
目次
Product Code: MM202017

Hemophilia A Gene Therapy- Key opinion leaders (KOLs) Insight- An Executive Report 2020 Deck provides a competitive advantage towards understanding the current gene therapy challenges, safety and efficacy concerns, hemophilia A gene therapy market uptake, Gene therapy competition, Hem A gene therapy re-dosing, Patients switching to gene therapy from hemlibra and factor replacement therapies and other critical key intelligent questions through 14 in-depth interviews with leading US and European KOLs.

Market adoption of gene therapy in haemophilia A is still a cause for concern due to the expected high prices, durability issues, redosing, safety concerns and key unknown factors that are still under discussion. Establishing a new modality of treatment in any disease space has many proven challenges, but what if it offered a real cure?

Gene therapy in haemophilia can influence treatment practices, reduce treatment burden, and chronic drug delivery in patients whilst providing cure. But how bright is the future for hemophilia gene therapy for hematologists and gene therapy experts and what are their critical questions that would help pharmaceutical and biotech companies develop better market access strategies?

What to expect

A detailed report exploring, through Haematologist and gene therapy experts' insights, how gene therapy in Hemophilia is driving new thinking and solutions:

  • An examination of key issues that are shaping approaches to the adoption of gene therapy in hemophilia
  • 30 targeted questions put to gene therapy experts
  • Their responses which provided 14 interviews supported by 50 directly quoted comments

Hemophilia A gene therapy key findings

  • BioMarin with its early moves with hemophilia a gene therapy, Roctavian (Valrox or valoctocogene roxaparvovec) for Hemophilia A has an edge over its rivals. Roctavian is likely to gain FDA approval by this August 2020.
  • Roctavian Potential Sales is estimated to be $Billion by 2028
  • Increasing competition with major Gene players such as Sangamo / Pfizer (SB-525 or giroctocogene fitelparvovec), Spark / Roche (Spark-8011), Bayer (BAY-2599023 / DTX 201), Takeda (TAK-754 (formely BAX 888/SHP654)) and with various unknowns associated with gene therapy Roctavian, it is unlikely to predict that it will stay on top with patient choice after 10 years of time.
  • In spite of an enormous enthusiasm for gene therapy, the number of patients undertaking gene therapy will be relatively small in the beginning

This Hemophilia A Gene Therapy KOLs insight deck for Hemophilia A provides the high level analysis surrounding the KOLs viewpoint from the US and Europe region

  • Clinical perspective
  • Commercial Perspective
  • Payer perspective

The United States and European KOLs have critically assessed and concluded the prospects of gene therapy for haemophilia A in the present and future scenario. This Insightful Deck provides a competitive advantage towards understanding the current gene therapy challenges, safety and efficacy concerns, hemophilia A gene therapy market uptake, Hem A Gene therapy competition, Hem A gene therapy re-dosing, Patients switching to gene therapy from hemlibra and factor replacement therapies and other critical key intelligent questions through in-depth interviews with leading US and European KOLs. Having critical business questions answered, the Report helps Gene Players to strategise and position their product well in this competitive environment.

Hemophilia A Gene Therapy -Key Questions Answered:

Clinical Perspective

  • How do you rate current treatment options for HEM A? What are the main challenges and how effective are the current modalities?
  • How do we compare them on the results of various gene therapy clinical trials?
  • What is the expected duration of the effects of gene therapy? Will they be permanent or transient?
  • When a patient is given gene therapy, can they be treated again if factor VIII or IX levels drop? Can we also consider treating patients with factor VIII or factor IX inhibitors?
  • Can we treat patients with inhibitors?
  • What is the risk of developing an inhibitor after gene therapy?

Commercial Perspective

  • How much segment of the haemophilia market will be occupied by gene therapy?
  • As stated by Biomarin at a press conference, Valrox/ Roctavian (GT) is about to wipe out the whole haemophilia A market. Is that true?
  • How difficult do you think it is for patients to move from factor replacement to gene therapy?
  • Will Hemlibra patients also consider switching?
  • What are the KOLs opinion on the potential of Gene Therapy of three major upcoming GT players in Hemophilia?
  • As per the Biomarin's GT durability, it will not effective after 8 years, so can these patient treated again?
  • What are KOL's thoughts on re-dosing?

Payer Perspective

  • Are doctors excited about gene therapy or it's still a matter of concern for them to prescribe to the patients?
  • GTs are expensive and cost in millions. However, FDA and regulatory bodies are promoting the development of more GT based therapies, which will decrease the price while increasing the competition. What are KOLs view on affordability of GT ?
  • Reimbursement and Negotiations with Payers
  • Who will be the decision maker while prescribing the GT?
  • What are the hard endpoints of decision making while prescribing the GT.
  • What factors can influence prescriber's decision and make them overcome these hard endpoints and feel confident about prescribing gene therapy.
  • Will the KOLs advise patients to take GT? Are KOLs in favor of GT?
  • United States vs European Scenario

Participating KOLs -14 KOLs Interviewed

Gene therapy Researcher

  • Haematologist, Children's Hospital of Philadelphia, US

Medical Director, Special Haemostasis Laboratory & Associate Director

  • Hemophilia Treatment Centre, Washington, United States

Faculty, Health Sciences

  • University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa

Honorary Consultant Haematologist, Centre Director

  • Royal London Hospital Hemophilia Centre, Arts Health NHS Trust, UK

KOLs

  • Irish Haemophilia Society, Ireland

Table of Contents

1. Clinical Perspective

  • High Level Executive Summary
  • KOLs Outlook on Hemophilia Gene Therapy
  • Safety & Efficacy Concerns of GT from the eyes of KOLs
  • Gene Therapy Redosing- KOLs Insights & Competitive Landscape
  • Gene Therapies for Children

2. Commercial Perspective

  • High Level Executive Summary
  • Estimated Patients Uptake & Market size of Roctavian Forecasted to 2028
  • Hemophilia A Gene Therapy Competitive Landscape & KOLs opinion
  • Biomarin's Gene Therapy Market Uptake based on KOLs view
  • Patients Switch from Hemlibra and Factor Replacements to Gene Therapy in the United States & Europe

3. Payers Perspective

  • High Level Executive Summary
  • KOLs and Patients comfortability on Gene Therapy Price in the United States and Europe
  • Reimbursement of Gene Therapies
  • Hard end points in decision making process of GT prescription
  • Influencing factors in the prescription of GT
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