PURPOSE: This workshop brings together experts on clinical outcome assessment and economic evaluation for a multiperspective dialogue on assessing the clinical meaningfulness and economic value of treatment benefits for chronic progressive diseases.
DESCRIPTION: Therapies for chronic progressive diseases can potentially delay or prevent the cumulative societal burden associated with these conditions. Regulatory approval for these therapies requires evidence on the meaningfulness of treatment benefits observed in clinical trials, whereas access and reimbursement decisions require evidence of long-term value driven by cumulative treatment benefits. Efforts to assemble this evidence for treatments for chronic progressive diseases have motivated advances in the estimation and application of clinical meaningfulness thresholds, statistical methods for analyzing trial data, and approaches for extrapolating trial endpoints to long-term outcomes.
Dr. Herring will orient the audience to the unique challenges presented by treatments for chronic progressive diseases (12 min). Dr. Coon will highlight distinctions between within-person changes and between-group differences and advocate for identifying context-specific meaningfulness thresholds by combining quantitative methods with qualitative stakeholder input (12 min). Dr. Lenderking will contrast the endpoint requirements for regulatory applications with those of clinical settings and health technology assessment (12 min). Dr. Whittington will discuss approaches for extrapolating treatment benefits over longer time horizons while emphasizing unique considerations for value assessment of disease-modifying therapies, such as valuing outcomes during life extension and real option value (12 min). We will conclude with a roundtable discussion (12 min). Audience polling will be used to gauge understanding and assess viewpoints. This workshop will be relevant for those involved in regulatory, access, and reimbursement decisions for treatments for chronic progressive diseases.
