The revolution in genomic sciences has spurred significant drug development in rare diseases, approximately 80% of which have a genetic etiology. The field of gene-based therapies, while still in the early stages, with therapies currently in various phases of preclinical and clinical development, has recently seen its first approvals in Europe. Gene-based therapies have the potential to transform the treatment of rare diseases and even provide a cure for many of them. Coupled with high pricing potential, recent technological advances will likely unlock a tremendous market potential for novel rare disease treatments. Broadly, gene therapies can be classified into three mechanistic categories: viral-based gene delivery systems, oligonucleotide-based therapies, and DNA-editing systems. Each category poses unique therapeutic advantages and development challenges. This special report will provide an analysis of the unique challenges and advantages of these approaches as well as examine the pipeline for gene-based therapies and the regulatory and market access issues that will impact the approval and uptake of these potentially transformational treatments.

QUESTIONS ANSWERED 

What are the unique clinical development challenges for gene therapies? What regulatory pathways exist for gene therapies in the United States and Europe?

How are payers approaching pricing and reimbursement of gene therapies; which may be curative? How will high annualized costs affect reimbursement?

What lessons can be learned from successful and unsuccessful commercial launches in Europe?

 What characteristics make an indication a good opportunity for a gene therapy?  Within each subcategory of gene-based therapies, what are the key technological differentiators and how might they provide a competitive advantage? What are the unique manufacturing challenges that gene therapies present?

Table of contents

  • Rare Diseases And Orphan Drugs - Access & Reimbursement - Special Topics - Gene Therapies For Rare Diseases (US/EU)
    • Overview and Actionable Recommendations
      • Actionable Recommendations to Optimize Market Access for Gene Therapies
      • Factors Influencing Pricing for Gene Therapies
    • Lessons Learned: Commercialization Challenges for Gene Therapies
      • Overview
      • Gene Therapy Development Challenges
        • Level of Acceptance of Clinical Development Program Features For Current and Emerging Gene Therapies
      • Gene Therapy Adoption Challenges
        • Evidence Evaluation Challenges
        • Safety Evaluation for Gene Therapies
        • Level of Acceptance Efficacy and Cost Across Current and Emerging Gene Therapies
        • Cost Effectiveness Challenges
        • Potential Payment Models for Gene Therapies
        • Price and Estimated Eligible Patient Population for Current and Emerging Gene Therapies
    • Payer Perspectives on Gene Therapies
      • Reimbursement Considerations: United States
      • ICER Evaluation Framework for Ultra-Rare Diseases
      • Reimbursement Considerations: United Kingdom
      • NICE Gene Therapy Evaluation Framework
      • Reimbursement Considerations: Germany
      • Reimbursement Considerations: France, Italy, and Spain
      • Expert Insights: United States
      • Expert Insights: United Kingdom
      • Expert Insights: Germany
    • Gene Therapy Case Studies
      • UniQure/Chiesi's Glybera
        • Overview and Key Lessons
        • Glybera's Product Profile
        • Level of Acceptance of Glybera's Product Profile
        • Glybera Development Timeline
        • Key Stakeholder Insights
        • In-Depth Review: Glybera's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Glybera
      • Spark Therapeutics' Luxturna
        • Overview and Key Lessons
        • Luxturna's Product Profile
        • Level of Acceptance of Luxturna's Product Profile
        • Luxturna Development Timeline
        • Key Stakeholder Insights
        • In-Depth Review: Luxturna's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Luxturna
      • Biogen/Ionis's Spinraza
        • Overview and Key Lessons
        • Spinraza's Product Profile
        • Level of Acceptance of Spinraza's Product Profile
        • Spinraza Development Timeline
        • Key Stakeholder Insights
        • In-Depth Review: Spinraza's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Spinraza
      • Sarepta Therapeutics' Exondys 51
        • Overview and Key Lessons
        • Exondys 51's Product Profile
        • Level of Acceptance of Exondys 51's Product Profile
        • Exondys 51 Development Timeline
        • Key Stakeholder Insights
        • In-Depth Review: Exondys 51's Product Profile
        • In Depth Review: Key Stakeholders' Acceptance of Exondys 51
      • GlaxoSmithKline's Strimvelis
        • Overview and Key Lessons
        • Strimvelis's Product Profile
        • Level of Acceptance of Strimvelis's Product Profile
        • Strimvelis Development Timeline
        • Key Stakeholder Insights
        • In-Depth Review: Strimvelis's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Strimvelis
      • Bluebird Bio's LentiGlobin
        • Overview and Key Lessons
        • LentiGlobin's Product Profile
        • Level of Acceptance of LentiGlobin's Product Profile
        • LentiGlobin Development Timeline
        • Key Stakeholder Insights
        • In-Depth Review: LentiGlobin's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of LentiGlobin
    • Gene Therapy Technologies
      • Overview
      • Gene Replacement
      • Viral Vectors for Gene Replacement
      • Antisense Oligonucleotides
      • Antisense Oligonucleotides Technology Approaches
      • Gene Editing
      • Gene Editing Approaches
      • Gene Editing Platforms
    • Methodology
      • Primary Market Research
      • Clinicians Interviewed
      • Payers Interviewed
    • Appendix
      • List of Abbreviations
      • Bibliography
      • About The Author

Author(s): Claudia Dall'Osso

Claudia is a Senior Business Insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing on niche and rare indications.

Before joining DRG, she held a management and strategy consultant position at Precision Medicine Group, where she worked for clients in the biopharmaceutical, medical device and diagnostic industries. Claudia completed her Master’s in Management at Harvard University; she also holds a Ph.D. in medical genetics from Brescia University in Italy and a BS/MS degree in medical biotechnology from University of Milano in Italy.


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