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Hemophilia A Gene Therapy- Key opinion leaders (KOLs) Insight- An Executive Report 2020

$6,250.00$16,750.00

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.

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Description

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’s Roctavian (Valrox or valoctocogene roxaparvovec) and for Hemophilia A could be the first GT to hit the market.
  • 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.
  • The leading gene therapy Potential Sales
  • 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?
    • To be Continued…
  • 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?
    • To be Continued…
  • 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
    • To be Continued….

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
  • To be Continued……

Additional information

Price

Enterprise License, Single User License, Site User License

Table of Contents

1.Clinical Perspective…………………………………………………….7

  • 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……………………………………………22

  • High Level Executive Summary
  • Estimated Patients Uptake & Market size of Roctavian Forecasted [Year 1 to Year 9]
  • 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…………………………………………………….42

  • 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

What to expect!

A detailed report exploring, through Haematologist 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

Deliverable

  • Deliverables: Includes PDF report and PowerPoint slide deck…Hemophilia A Gene Therapy-KOLs Insights Executive Report, 2020 is delivered as:
    • A detailed PDF report including all insights, quotes and intelligence exhibits
    • A useful PowerPoint slide deck providing a summary of the report’s key findings to use in your presentations and for sharing with colleagues

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