Rare Diseases and Orphan Drugs | Access & Reimbursement | Special Topics - Gene Therapies for Rare Diseases (US/EU)

Publish date: December 2017

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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

  • Special Topics - Gene Therapies for Rare Diseases (US/EU)
    • Overview and Actionable Recommendations
      • Actionable Recommendations to Optimize Market Access for Gene Therapies
    • Lessons Learned: Commercialization Challenges for Gene Therapies
      • Overview
      • Gene Therapy Development Challenges
      • Gene Therapy Adoption Challenges
        • Evidence Evaluation Challenges
        • Safety Evaluation for Gene Therapies
        • Cost Effectiveness Challenges
        • Potential Payment Models for Gene Therapies
    • Payer Perspectives on Gene Therapies
      • Reimbursement Considerations: United States
      • Reimbursement Considerations: United Kingdom
      • Reimbursement Considerations: Germany
      • Reimbursement Considerations: France, Italy, and Spain
    • Gene Therapy Case Studies
      • UniQure/Chiesi's Glybera
        • Overview and Key Lessons
        • In-Depth Review: Glybera's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Glybera
      • Spark Therapeutics' Luxturna
        • Overview and Key Lessons
        • In-Depth Review: Luxturna's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Luxturna
      • Biogen/Ionis's Spinraza
        • Overview and Key Lessons
        • In-Depth Review: Spinraza's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Spinraza
      • Sarepta Therapeutics' Exondys 51
        • Overview and Key Lessons
        • In-Depth Review: Exondys 51's Product Profile
        • In Depth Review: Key Stakeholders' Acceptance of Exondys 51
      • GlaxoSmithKline's Strimvelis
        • Overview and Key Lessons
        • In-Depth Review: Strimvelis's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of Strimvelis
      • Bluebird Bio's LentiGlobin
        • Overview and Key Lessons
        • In-Depth Review: LentiGlobin's Product Profile
        • In-Depth Review: Key Stakeholders' Acceptance of LentiGlobin
    • Gene Therapy Technologies
      • Overview
      • Gene Replacement
      • Antisense Oligonucleotides
      • Gene Editing
    • Methodology
      • Primary Market Research
    • Appendix
      • 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 in medical genetics from Brescia University in Italy and a BS/MS degree in medical biotechnology from University of Milano in Italy.