Avalere identified the top trends in health economics and outcomes research (HEOR) that are significantly shaping the landscape in 2024 and beyond. In this final Insight, Avalere experts dive into Trend #10: Quantifying Indirect Burden and Measuring Novel Elements of Value.

Traditional Estimates of Burden and Value Elements

In HEOR, disease burden is often measured through quantitative estimates of direct healthcare-related costs and utilization. Traditional value assessments often utilize narrow cost-effectiveness analyses that center around the payer perspective instead of the patient perspective and mainly consider clinical benefit and direct costs. While these metrics have an important role in the valuation of health technologies, there is growing recognition that these methods may not be comprehensive enough, especially in their ability to capture indirect costs and account for the patient voice.

The patient voice is particularly important for rare diseases, which is a classification for conditions with prevalence below 200,000 in the United States. Unpredictable, high disease severity coupled with complex, burdensome treatments further obscures the total burden of rare disease, which affects not only patients, but their caregivers, family members, and society as a whole. These impacts extend beyond the healthcare system (e.g., absenteeism, lost productivity) and they are estimated to comprise more than half of the overall burden of rare diseases.

Evolving Methods to Measure Indirect Burden and Identify Novel Value Elements

Policymakers and health technology assessment (HTA) bodies are shifting towards a patient-centric model, making it imperative to better understand the indirect burden of disease to patients, caregivers, and society, and to augment existing value assessment methods to better account for that total burden. There are multiple organizations and initiatives focused on integrating the patient voice into value assessment.

One first step is to analyze disease-specific impacts alongside direct cost data, as detailed in a recent whitepaper. This approach involves conducting a mixed methods study with patients and caregivers to define disease-specific impact elements, quantify those elements alongside labor and productivity impacts through a survey, and supplement with a claims analysis on the traditional excess direct medical costs data. This approach results in a comprehensive understanding of the annual disease burden, which provides valuable insights and can serve as a foundation for stakeholder actions.

Implications and Importance

Understanding true disease burden has anticipated benefits for healthcare industry stakeholders, including but not limited to:

• Improved recognition of the impacts and perspectives of patients and caregivers
• Deeper understanding for payers of the healthcare utilization dynamics in their patients
• Tailored impact elements for use in value assessments by HTA bodies
• Better ability for providers to implement person-centered care
• Enhanced base of evidence to inform policymaker decisions
• Greater insights into patient-centered impacts that can be addressed by the treatments manufacturers develop

Despite these benefits, there are outstanding challenges with capturing the true total burden and using a person-centered approach to define value. This trend will continue to evolve as research and value assessment methodologies advance.

Look for future releases to stay on top of this evolving landscape and view the related video on indirect burden and novel elements of value. Take a look back at Avalere’s other , including a webinar on the first four trends.

To discuss how Avalere can support your HEOR initiatives or to receive regular Avalere updates, connect with us.