Future Directions for Cost-effectiveness Analyses in Health and Medicine

Area of Science:

• Health economics
• Decision science
• Public health policy

Background:

• The Second Panel on Cost-effectiveness in Health and Medicine convened in 2016 to update foundational work from two decades prior.
• The panel reflected on the advancements in cost-effectiveness analysis (CEA) and provided guidance for future practitioners and policymakers.
• This update addresses the evolution of CEA methodologies and their practical application.

Purpose of the Study:

• To identify and present key topics for future research and policy in cost-effectiveness analysis.
• To highlight outstanding research needs in seven critical areas identified by the panel.
• To guide the next generation of practitioners and consumers in the field of CEA.

Main Methods:

• The Second Panel deliberated on numerous topics to advance CEA methods and improve decision-making.
• Seven primary areas for fruitful future research were identified based on repeated discussions.
• An online appendix provides brief expoundments on eight additional important topics.

Main Results:

• Key research areas include CEA perspectives, modeling transparency, valuing health outcomes (including temporary states and caregiver effects), and comprehensive costing.
• Further research is needed in evidence synthesis (learning across studies, combining diverse data), cost-effectiveness thresholds (opportunity costs, willingness to pay), and standardized reporting/communication of CEAs.
• Specific needs include developing theory for evidence synthesis, creating a cost catalogue, and establishing quality scoring systems for CEA reports.

Conclusions:

• Cost-effectiveness analysis is a dynamic and expanding field with significant research potential.
• Continued research is essential to optimize the integration of CEA into health policy and practice.
• Addressing the identified research gaps will enhance the utility and impact of CEA in healthcare decision-making.