Putting Stakeholder Engagement at the Center of Health Economic Modeling for Health Technology Assessment in the United States

Abstract

While evidence generated from health economic (HE) models is being used more commonly in health technology assessment (HTA) in the US, it is not consistently adopted by different stakeholder groups or across therapeutic areas. We hypothesize that actively engaging with multiple stakeholder groups throughout the model development process may result in models more widely considered by decision makers. To test this hypothesis, the Innovation and Value Initiative has launched a modeling effort to build an open-source HE model focusing on the disease state 'major depressive disorder'. A 20-member advisory group has been formed with representatives from patients, employers, clinicians, innovators, payers, and researchers to guide the model development process. While this effort is still in the early stages, the ongoing stakeholder engagement effort has yielded valuable insights that inform the model design. We have also identified several challenges to implementing this new approach. Our early findings suggest that the stakeholder engagement approach to HE model development has the potential to improve HTA in the US.

Factors Limiting Subgroup Analysis in Cost-Effectiveness Analysis and a Call for Transparency

“…Increased stakeholder engagement throughout the process of economic evaluation (from conceptualisation to final reporting) may also support the identification and justification of subgroup analysis. An example of stakeholder engagement in guiding economic model development and subgroups (including identifying subgroups and relevant data sources) has been reported by Xie et al [43]. We would also encourage researchers to consider the full range of methods available to acknowledge patient heterogeneity in cost-effectiveness analysis (not restricted to subgroup analysis), as well as any guidelines that are applicable to their setting of interest [1].…”

Preferences for Treatments for Major Depressive Disorder: Formative Qualitative Research Using the Patient Experience

“…A co-author and patient stakeholder from the PAVE Center, who raised a child with a mental health condition, assisted with the interpretation and contextualization of the patient-informed value elements influencing treatment decisions for MDD. A 20-member MDD-specific multi-stakeholder advisory group (SAG) [ 16 ] with representation from patient communities, payers, manufacturers, clinical experts, researchers, and employers assisted with the selection and definition of the key value elements and with participant recruitment.…”

The Growing Burden of Major Depressive Disorders (MDD): Implications for Researchers and Policy Makers

“…Xie et al develop an iterative multi-stakeholder model development approach, which includes an advisory group that incorporates heterogeneous perspectives [16]. Compared with traditional approaches, engagement with key stakeholders offers promise in building better inputs from underrepresented stakeholder groups such as patients or employers, validating key assumptions, and identifying methodological challenges early on.…”

“…As with many other diseases, SES has been shown to greatly affect access to care, insurance status, consistency in treatment follow-up, and other factors that affect the clinical outcomes of patients with MDD. These factors all contribute to the heterogeneity and, ultimately, the economic burden of disease and therefore can be critical inputs in economic models [16].…”