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Epilepsy | Unmet Need | Dravet Syndrome | US/EU | 2021

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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

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