Epilepsy – Unmet Need – Detailed, Expanded Analysis : Dravet Syndrome: US/EU
Dravet syndrome (DS) is a rare, severe, pediatric-onset form of epilepsy characterized by developmental delay or regression and multiple types of difficult-to-treat seizures. Only a handful of antiepileptic drugs (AEDs), including Diacomit (stiripentol), Epidiolex / Epidyolex (cannabidiol), and Fintepla (fenfluramine), are approved for the adjunctive treatment of DS, but owing to various factors, off-label generic AEDs such as clobazam and valproate remain the first-line choice in DS treatment. Moreover, most patients do not achieve complete seizure control with currently available AEDs, even when prescribed concomitantly. Understanding the drivers of clinical decision-making in DS, prescribers’ perception of current treatment options, and areas of remaining unmet need will help drug developers identify levers to optimize the positioning and differentiation of new products.
QUESTIONS ANSWERED
- What are the treatment drivers and goals for DS?
- What attributes drive decision-making in DS, which have limited impact, and which are hidden opportunities?
- How do approved agents (i.e., Diacomit, Epidiolex / Epidyolex, and Fintepla) perform on these attributes? How do key off-label AEDs (i.e., clobazam, topiramate, and valproate) perform on these attributes?
- What are the prevailing areas of unmet need and opportunity in DS?
- Based on a conjoint analysis and TPP simulation, what trade-offs across different clinical attributes and price are acceptable to neurologists for a hypothetical new DS drug?
PRODUCT DESCRIPTION
Provides quantitative insight into U.S. and European physician perceptions of key treatment drivers and goals and the current level of unmet need for a specific disease. Commercial opportunities are analyzed, and the extent to which emerging therapies may capitalize on these opportunities is evaluated.
Markets covered: United States, United Kingdom, France, Germany
Primary research: Survey of 60 U.S. and 31 European neurologists fielded in January 2021
Key companies: Biocodex, GW Pharmaceuticals, Zogenix, Takeda, Zynerba Pharmaceuticals
Key drugs: Diacomit, Epidiolex / Epidyolex, Fintepla, clobazam, topiramate, valproate
Table of contents
- Epilepsy - Unmet Need - Detailed, Expanded Analysis : Dravet Syndrome: US/EU
- Unmet Need - Dravet Syndrome - TPP Simulator - March 2021
- Executive Summary
- Introduction
- Treatment Drivers and Goals
- Key Findings: Attribute Importance
- Relative Importance of Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Surveyed Neurologists' Prescribing Decisions 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
- Key Findings: Stated vs. Derived Importance
- 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
- Key Findings
- Overall Performance of Key Therapies for Dravet Syndrome: United States
- Overall Performance of Key Therapies for Dravet Syndrome: Europe
- Mean Overall Performance of Key Therapies for Dravet Syndrome: United States and 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
- Key Findings: Unmet Need in Dravet Syndrome
- Surveyed Neurologists' Satisfaction with the Performance of Key Therapies for Dravet Syndrome on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: United States
- Surveyed Neurologists' Satisfaction with the Performance of Key Therapies for Dravet Syndrome on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: Europe
- 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
- Key Findings: 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
- Target Product Profiles
- Appendix
Niyati Khetarpal, M.Sc., M.B.A., Ph.D., is a senior analyst on the CNS/Ophthalmology Disorders team at Clarivate. In this role, she conducts primary and secondary research to analyze pharmaceutical markets, with a focus on neurology indications, including epilepsy and MS. She also has specific expertise in patent/IP research. Dr. Khetarpal obtained her doctorate from the International Centre for Genetic Engineering and Biotechnology in New Delhi, where she conducted research on Dengue virus vaccines. In 2014, she received a Fulbright-Nehru doctoral scholarship for her research on Dengue and West Nile virus envelope proteins.