Childhood absence epilepsy (CAE) is characterized by frequent and brief absence seizures in children aged 4-12 and is diagnosed in approximately 10-15% of all childhood epilepsies. Only a handful of antiepileptic drugs (AEDs) are believed to be effective in CAE, and only two of these drugs (ethosuximide and valproate) are approved to treat absence seizures. Treatment becomes more complicated if the patient also suffers from co-occurring generalized tonic-clonic (GTC) seizures. Moreover, all AEDs currently used for CAE are associated with clinical shortcomings in efficacy, safety, and/or delivery. Thus, ample opportunity exists for new and improved alternatives, renewing developers’ interest in this space.

Questions Answered

  • What clinical end points and drug attributes are most influential in prescribing for CAE? How do currently available drugs like ethosuximide, valproate, and lamotrigine perform on these attributes?
  • What are the prevailing areas of unmet need and opportunity in the treatment of CAE?
  • Based on conjoint analysis and TPP simulation, what trade-offs between efficacy, safety, and price are neurologists willing to make for a hypothetical new AED for CAE?

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

Primary Research: Survey of 60 U.S. neurologists and 31 European neurologists.

Key Drugs Covered: Ethosuximide, valproate, lamotrigine, levetiracetam, topiramate, zonisamide.

Key metrics included:

  • Target Product Profile (TPP) simulator based on conjoint analysis methodology.
  • Stated vs. derived importance of product attributes on prescribing behavior.
  • Assessment of current drug performance against treatment drivers and goals.
  • Physician perceptions of unmet needs in the indication and related indications.
  • Analysis of remaining drug development opportunities.

Product Description

Unmet Need supports clinical development decisions by identifying key attributes and assessing areas of unmet need for a specific disease or subpopulation. Based on surveys with U.S. and European physicians, this report provides insight into key treatment drivers and goals, the performance of current therapies, and the remaining commercial opportunities. Two market scenarios are profiled in detail by DRG experts, and additional customized market scenarios can be evaluated with the corresponding TPP simulator.

Table of contents

  • Epilepsy - Unmet Need - Detailed, Expanded Analysis : Childhood Absence Epilepsy : US/EU
    • Introduction
      • Overview
      • Methodology
      • Rationale for Treatment Drivers and Goals Selection
        • Efficacy
        • Safety and Tolerability
        • Convenience of Administration
      • Rationale for Drug Selection
        • Products for Childhood Absence Epilepsy
    • 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 Childhood Absence Epilepsy
      • Importance of Efficacy Attributes to Prescribing Decisions in Childhood Absence Epilepsy: United States
      • Importance of Efficacy Attributes to Prescribing Decisions in Childhood Absence Epilepsy: Europe
      • Importance of Safety and Tolerability Attributes to Prescribing Decisions in Childhood Absence Epilepsy: United States
      • Importance of Safety and Tolerability Attributes to Prescribing Decisions in Childhood Absence Epilepsy: Europe
      • Importance of Convenience of Administration Attributes to Prescribing Decisions in Childhood Absence Epilepsy: United States
      • Importance of Convenience of Administration Attributes to Prescribing Decisions in Childhood Absence Epilepsy: 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 Childhood Absence Epilepsy: United States
      • Stated vs. Derived Importance of Key Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Attributes to Prescribing Decisions in Childhood Absence Epilepsy: Europe
    • Product Performance Against Treatment Drivers and Goals
      • Key Findings
      • Overall Performance of Key Therapies for Childhood Absence Epilepsy: United States
      • Overall Performance of Key Therapies for Childhood Absence Epilepsy: Europe
      • Mean Overall Performance of Key Therapies for Childhood Absence Epilepsy: United States and Europe
      • Relative Performance of Key Therapies for Childhood Absence Epilepsy Across Select Efficacy Attributes: United States
      • Relative Performance of Key Therapies for Childhood Absence Epilepsy Across Select Efficacy Attributes: Europe
      • Relative Performance of Key Therapies for Childhood Absence Epilepsy Across Select Safety and Tolerability Attributes: United States
      • Relative Performance of Key Therapies for Childhood Absence Epilepsy Across Select Safety and Tolerability Attributes: Europe
      • Relative Performance of Key Therapies for Childhood Absence Epilepsy Across Select Convenience of Administration Attributes: United States
      • Relative Performance of Key Therapies for Childhood Absence Epilepsy Across Select Convenience of Administration Attributes: Europe
    • Assessment of Unmet Need
      • Key Findings: Unmet Need in Childhood Absence Epilepsy
      • Surveyed Neurologists’ Satisfaction with the Performance of Key Therapies for Childhood Absence Epilepsy on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: United States
      • Surveyed Neurologists’ Satisfaction with the Performance of Key Therapies for Childhood Absence Epilepsy on Efficacy, Safety and Tolerability, Convenience of Administration, and Nonclinical Factors: Europe
      • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in Childhood Absence Epilepsy: United States
      • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Efficacy Attributes in Childhood Absence Epilepsy: Europe
      • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Childhood Absence Epilepsy: United States
      • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Safety and Tolerability Attributes in Childhood Absence Epilepsy: Europe
      • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Childhood Absence Epilepsy: United States
      • Surveyed Neurologists' Ascribed Level of Unmet Need Across Key Convenience of Administration Attributes in Childhood Absence Epilepsy: Europe
      • Key Findings: Unmet Need in Childhood Absence Epilepsy and Related Indications
      • Surveyed Neurologists' Ascribed Level of Unmet Need in Childhood Absence Epilepsy and Related Indications: United States
      • Surveyed Neurologists' Ascribed Level of Unmet Need in Childhood Absence Epilepsy and Related Indications: Europe
    • Opportunity Analysis
      • Areas of Opportunity in the Childhood Absence Epilepsy Market and Emerging Therapy Insights
        • Opportunity: A Novel Agent Offering More Complete Freedom from Treatment-Refractory Absences in CAE Patients
        • Opportunity: A Novel AED That Offers a Greater Reduction in the Frequency of Co-Occurring GTC Seizures in CAE Patients
        • Opportunity: New Therapies That Can Address Clinical Shortcomings of Drugs Currently Used to Treat CAE
    • Target Product Profiles
      • Assessing Drug Development Opportunities
      • Target Product Profile Methodology
        • Attributes and Attribute Levels
      • Attribute Importance and Part-Worth Utilities
        • Childhood Absence Epilepsy Target Product Profile: Attribute Importance
        • Percentage of Patients Free of Absence Seizures
        • Median Percent Reduction from Baseline in GTC Seizure Frequency
        • Incidence of Drowsiness
        • Incidence of Rash
        • Incidence of GI-Related Side Effects
        • Formulations Available
        • Price per Day
      • Conjoint Analysis-Based Simulations of Market Scenarios
        • Childhood Absence Epilepsy Market Simulations: Share of Preference of Target Product Profiles Included in Scenario 1
        • Childhood Absence Epilepsy Market Simulations: Likelihood to Prescribe Target Product Profiles Included in Scenario 1
        • Childhood Absence Epilepsy Market Simulations: Target Product Profiles Included in Scenario 1
        • Childhood Absence Epilepsy Market Simulations: Share of Preference of Target Product Profiles Included in Scenario 2
        • Childhood Absence Epilepsy Market Simulations: Likelihood to Prescribe Target Product Profiles Included in Scenario 2
        • Childhood Absence Epilepsy Market Simulations: Target Product Profiles Included in Scenario 2
    • Appendix
      • Key Abbreviations
      • Bibliography

Author(s): Niyati Khetarpal, Ph.D

"Niyati Khetarpal is a business insights analyst on the Central Nervous System, Pain, and Ophthalmology team at Decision Resources Group. 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. Prior to joining the company, Niyati obtained her doctorate from the International Centre for Genetic Engineering and Biotechnology for her research on Dengue virus vaccines. She received the Fulbright-Nehru doctoral scholarship in 2014 for her research on Dengue and West Nile virus envelope proteins.”


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