Epilepsy – Unmet Need – Detailed, Expanded Analysis : Childhood Absence Epilepsy : US/EU
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
- 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 Neurologistsu2019 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 Neurologistsu2019 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
- Areas of Opportunity in the Childhood Absence Epilepsy Market and Emerging Therapy Insights
- Target Product Profiles
- Appendix
- 2019 TPP Simulator - Childhood Absence Epilepsy
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.