Dravet syndrome (DS) is a rare, severe, pediatric-onset form of epilepsy characterized by developmental delay or regression and multiple seizure types. Only Diacomit (Biocodex’s stiripentol) is specifically approved for the adjunctive treatment of refractory DS (in Europe, Canada, and Japan), but the drug is difficult to access for U.S. prescribers and is not effective in all patients. Recognizing the clear unmet need for additional therapies specifically for DS, drug developers (e.g., GW Pharmaceuticals, Zogenix) are advancing new products through the pipeline that could offer important alternatives for this underserved orphan population. Understanding the drivers of clinical decision-making in DS and prescriber perceptions of today’s mostly off-label, generic options will help identify levers for new product positioning and differentiation.

Questions Answered:

  • What are the treatment drivers and goals for Dravet syndrome?
  • What attributes are key influences, which have limited impact, and which are hidden opportunities?
  • How do current therapies perform on key treatment drivers and goals for Dravet syndrome?
  • What are the prevailing areas of unmet need and opportunity in Dravet syndrome?
  • What trade-offs across different clinical attributes and price are acceptable to U.S. and European neurologists for a hypothetical new Dravet syndrome drug?

Markets covered: United States, France, Germany, United Kingdom

Primary research: Survey of 60 U.S. and 30 European neurologists fielded in December 2016.

Key companies: Biocodex, GW Pharmaceuticals, Xenon Pharma, Zogenix

Key drugs: Diacomit (stiripentol), Onfi (clobazam), valproic acid, levetiracetam, topiramate, clonazepam, Fycompa (perampanel), Epidiolex, ZX-008

Table of contents

  • Detailed, Expanded Analysis: Epilepsy-Dravet Syndrome
    • Key Updates
      • October 2017
      • August 2017
    • Treatment Drivers and Goals
      • Overview
      • Treatment Drivers and Goals
        • Methodology
        • Physician Weighting of Clinical and Nonclinical Attributes
      • Rationale for Treatment Drivers and Goals Selection
        • Efficacy
        • Safety and Tolerability
        • Convenience of Administration
        • Nonclinical Factors
      • Physician Rating of Treatment Drivers and Goals in Dravet Syndrome
        • Efficacy
        • Safety and Tolerability
        • Convenience of Administration
        • Nonclinical Factors
      • Stated vs. Derived Importance of Treatment Drivers and Goals
    • Product Performance Against Treatment Drivers and Goals
      • Overview
      • Rationale for Drug Selection
      • Current Brand Performance on Key Treatment Drivers and Goals
        • Efficacy
        • Safety and Tolerability
        • Convenience of Administration
        • Nonclinical Factors
    • Assessment of Unmet Need
      • Overview
      • Overall Satisfaction with Current Treatment
      • Physician Rating of Unmet Need in Dravet Syndrome
        • Efficacy
        • Safety and Tolerability
        • Convenience of Administration
        • Nonclinical Factors
      • Unmet Need in Dravet Syndrome and Related Indications
    • Opportunity Analysis
      • Areas of Opportunity in the Dravet Syndrome Market and Emerging Therapy Insights
        • Opportunity: Approved Alternatives for the Treatment of Dravet Syndrome
        • Opportunity: A Novel Agent That Does Not Negatively Impact Patients' Cognitive Ability and Function
        • Opportunity: Disease-Modifying Therapy
    • Target Product Profiles
      • Assessing Drug Development Opportunities
      • Target Product Profile Methodology
      • Attribute Importance and Part-Worth Utilities
        • Dravet Syndrome Target Product Profile: Attribute-Level Part-Worth Utilities
      • Conjoint Analysis-Based Simulations of Market Scenarios
        • Scenario 1
        • Scenario 2
    • Appendix
      • Experts Interviewed
      • Dravet Syndrome Bibliography

Author(s): Jonathan Searles; Bethany Christmann, PhD

Jonathan ;Searles is a Senior Director in the CNS/Ophthalmology unit at DRG, overseeing the neurology research stream. Prior to joining DRG, ;Searles received a degree, summa cum laude, from Brandeis University; during this time, Mr. Searles conducted research projects at the Harvard University Peabody Museum and at Brandeis.

Bethany Christmann, has been with DRG since 2015, and is a Senior Business Insights Analyst with the Central Nervous System/Ophthalmology team. In this role, she covers the neurology space, specializing in Parkinson’s disease and epilepsy; she provides expert insight and authors primary market research and forecasting content focused on these and other neurology indications. Prior to joining DRG, Bethany earned her in neuroscience from Brandeis University, where she studied the cellular interactions involved in memory consolidation and their link to sleep behavior.


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