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

  • Epilepsy - Unmet Need - Detailed, Expanded Analysis: Epilepsy-Dravet Syndrome
    • Key Updates
      • October 2017
      • August 2017
    • Treatment Drivers and Goals
      • Overview
      • 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
        • Importance of Efficacy Attributes to Prescribing Decisions in Dravet Syndrome: United States
        • Importance of Efficacy Attributes to Prescribing Decisions in Dravet Syndrome: Europe
        • Importance of Safety and Tolerability Attributes to Prescribing Decisions in Dravet Syndrome: United States
        • Importance of Safety and Tolerability Attributes to Prescribing Decisions in Dravet Syndrome: Europe
        • Importance of Convenience of Administration Attributes to Prescribing Decisions in Dravet Syndrome: United States
        • Importance of Convenience of Administration Attributes to Prescribing Decisions in Dravet Syndrome: Europe
        • Importance of Nonclinical Factors to Prescribing Decisions in Dravet Syndrome: United States
        • Importance of Nonclinical Factors to Prescribing Decisions in Dravet Syndrome: Europe
      • Stated vs. Derived Importance of Treatment Drivers and Goals
        • Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Prescribing Decisions in Dravet Syndrome: United States
        • Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Prescribing Decisions in Dravet Syndrome: Europe
    • Product Performance Against Treatment Drivers and Goals
      • Overview
      • Rationale for Drug Selection
        • Products for Dravet Syndrome
      • Current Brand Performance on Key Treatment Drivers and Goals
        • Overall Performance of Key Therapies for Dravet Syndrome: United States
        • Overall Performance of Key Therapies for Dravet Syndrome: Europe
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Efficacy Attributes: United States
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Efficacy Attributes: Europe
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Safety and Tolerability Attributes: United States
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Safety and Tolerability Attributes: Europe
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Convenience of Administration Attributes: United States
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Convenience of Administration Attributes: Europe
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Nonclinical Attributes: United States
        • Relative Performance of Key Therapies for Dravet Syndrome Across Select Nonclinical Attributes: Europe
    • Assessment of Unmet Need
      • Overview
      • Overall Satisfaction with Current Treatment
        • Surveyed Neurologists’ Satisfaction with the Performance of Key Therapies for DS on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: United States
        • Surveyed Neurologists’ Satisfaction with the Performance of Key Therapies for DS on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: Europe
      • Physician Rating of Unmet Need in Dravet Syndrome
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in Dravet Syndrome: United States
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in Dravet Syndrome: Europe
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Dravet Syndrome: United States
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Dravet Syndrome: Europe
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Dravet Syndrome: United States
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Dravet Syndrome: Europe
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Nonclinical Factors in Dravet Syndrome: United States
        • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Nonclinical Factors in Dravet Syndrome: Europe
      • Unmet Need in Dravet Syndrome and Related Indications
        • Surveyed Neurologists' Ascribed Level of Unmet Need in Dravet Syndrome and Related Indications: United States
        • Surveyed Neurologists' Ascribed Level of Unmet Need in Dravet Syndrome and Related Indications: Europe
    • 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
        • Attributes and Attribute Levels
      • Attribute Importance and Part-Worth Utilities
        • Dravet Syndrome Target Product Profile: Attribute Importance
        • Reduction in Frequency of Convulsive Seizures vs. Placebo
        • Improvement in 50% Responder Rate for Convulsive Seizures vs. Placebo
        • Reduction in Frequency of Nonconvulsive Seizures vs. Placebo
        • Mechanism of Action/Drug Class
        • Incidence of Somnolence vs. Placebo
        • Delivery Profile
        • Price per Day
      • Conjoint Analysis-Based Simulations of Market Scenarios
        • Dravet Syndrome Market Simulations: Share of Preference of Target Product Profiles Included in Scenario 1
        • Dravet Syndrome Market Simulations: Likelihood to Prescribe Target Product Profiles Included in Scenario 1
        • Dravet Syndrome Market Simulations: Target Product Profiles Included in Scenario 1
        • Dravet Syndrome Market Simulations: Share of Preference of Target Product Profiles Included in Scenario 2
        • Dravet Syndrome Market Simulations: Likelihood to Prescribe Target Product Profiles Included in Scenario 2
        • Dravet Syndrome Market Simulations: Target Product Profiles Included in Scenario 2
    • Appendix
      • Key Abbreviations
      • Experts Interviewed
      • Dravet Syndrome Bibliography

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

Jonathan W. Searles is a Senior Director in the CNS/Ophthalmology unit at DRG, overseeing the neurology research stream. Prior to joining DRG, Mr. Searles received a B.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, Ph.D., 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 Ph.D. 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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