Preparing for launch in multiple countries with a comprehensive evidence strategy

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Health Economics and Outcomes Research (HEOR) Strategy Plans and Gap Analyses for a Treatment Targeting Multiple Rare Diseases

VALUE TO CLIENT

Our team’s synthesis of the available information across multiple indications and our expert guidance on the additional evidence required helped our client plan the evidence needed to help inform future work streams to improve market access of their new product. Additionally, our work allowed our client to quickly disseminate the findings of the burden of illness and gap analysis in each indication at ISPOR.

OPPORTUNITY

Our client had a product in multiple phase 2 and 3 clinical trials for the treatment of numerous rare disease indications (chronic inflammatory demyelinating polyneuropathy (CIDP), pemphigus vulgaris (PV) and pemphigus foliaceus (PF), and primary immune thrombocytopenia (ITP), bullous pemphigoid (BP), idiopathic inflammatory myositis (IIM), thyroid eye disease (TED), dermatomyositis, and scleroderma). As they prepared for launch in the United States, France, Germany, Italy, Spain, and the United Kingdom, they needed an experienced partner to help ensure they had robust evidence to support optimal market access.

OUR APPROACH

  • For each indication, we performed the following: 
    • conducted targeted literature reviews of the epidemiology, disease state, quality-of-life measures, and gaps in treatment evidence. 
    • reviewed designs for studies and the resulting data on competitive treatments (pipeline and supportive care).
    • created the product value proposition and potential value messages (efficacy, safety, humanistic/quality of life, economic, and dosing administration).
    • assessed evidence gaps and considered evidence needed to generate dossiers, economic models, and real-world evidence to support the product launch.
    • developed an HEOR strategy plan detailing the evidence and budget needed to help inform future work.
  • We designed the HEOR strategy plans to include new study recommendations and communication tools as well as timelines for those activities.