In the absence of a formal health technology assessment (HTA), US payers have informally turned to third-party HTA organizations to inform coverage decisions, formulary placement, and price negotiations. The Institute for Clinical and Economic Review (ICER) has evolved in recent years to position itself as a frontrunner in the US third-party value-assessment landscape. ICER is a nonprofit organization that seeks to use evidence-based methods to improve access to and affordability of health technologies. It assesses the value of health technologies and services in accordance with the methods defined in its Value Assessment Framework (VAF). ICER updates its VAF every three years, and the current framework—established in 2020—expires at the end of 2023.

Previous updates made significant changes to the institute’s methods and outputs, including the introduction of a modified societal perspective as a co-base case when societal cost or impact is large, conducting cost-effectiveness assessments using equal value of life-year gained (evLYG) measures in parallel with the quality-adjusted life year (QALY) cost-effectiveness analyses. They also launched an adapted assessment approach for high-impact single and short-term transformative therapies. On June 5, ICER released proposed changes to its VAF and invited public comment on the proposal.

ICER’s Proposed Updates

Compared to the breadth of updates included in the 2020 VAF update, ICER has proposed fewer major methods or process changes for the 2024 update. Key proposed changes include:

• Clinical trial diversity: ICER proposes to include a rating of clinical trial diversity and a description of demographic diversity in trial data in its evidence reports.
• Subpopulations: ICER proposes to include an a priori list of subpopulations of interest and a scientific rationale for evaluating these subpopulations in the scoping document and research protocol. The institute also proposes using a formal credibility assessment tool to evaluate the credibility of subgroup findings in the heterogeneity and subgroups section of its evidence reports.
• A dynamic pricing scenario for products targeting Medicare-eligible populations: To account for the impact of the Inflation Reduction Act (IRA) on long-term cost-effectiveness, ICER proposes to conduct a dynamic pricing scenario for small molecule and biological products that primarily target Medicare-eligible populations and, in ICER’s estimation, could be subject to the IRA’s price negotiations.
• An indirect approach to estimate productivity: To ensure that cost-effectiveness analyses using the modified societal perspective have “non-zero” inputs for impacts on productivity, ICER proposes to use an indirect approach to estimate potential impacts on patient and caregiver productivity when direct data are unavailable.
• Quantifying additional dimensions of value: ICER intends to keep several novel elements of value (e.g., the value of hope, reduction in uncertainty) qualitative. To address severity as a potential modifier of the value of health gains, ICER will regularly calculate QALY and evLYG shortfalls. ICER proposes to continue using the Health Improvement Distribution Index to measure health equity.

In addition to these proposed methods changes, ICER also proposed changes to its process for addressing the treatment of unmet needs and health equity:

• Topic selection: ICER proposes to incorporate health disparities in the topic selection process for future reviews by including health equity concerns in horizon scanning exercises and presenting relevant health disparities data during topic selection meetings.
• Patient engagement program: ICER proposes to simplify program processes, increase the diversity of patient perspectives, add new formal and informal engagement opportunities, and provide an honorarium to participants.

Several proposed changes to the VAF address stakeholder feedback on limitations of the current framework. For example, adopting an indirect approach to estimating productivity impacts would increase ICER’s ability to assess the value a treatment may provide in its modified societal perspective cost-effectiveness analyses when direct data is not available.

Notably, the proposed updates signal that ICER does not intend to make other changes that some stakeholders were anticipating, such as extending its application of the modified societal perspective, developing evidence standards for inclusion of real-world evidence in ICER assessments, or adapting its methodology for orphan drugs and accelerated approval therapies with short-term trial data.

Key Considerations and Implications

Despite several legislative and administrative efforts to introduce a formal role for HTA, the US does not have a formal HTA body or advisory group with the authority to review clinical or cost effectiveness of health products to formulate reimbursement decisions. Although the introduction of the Medicare drug price negotiation program under the IRA introduces a role for government in assessing drug prices, use of QALYs (a prevailing summary measure in cost-effectiveness analyses) in Medicare programs was barred in the Affordable Care Act and a bill (HR 485) to ban QALYs “and similar measures” across federal health agencies more broadly was recently introduced. The absence of a formal HTA body has given ICER an expanded role in the value assessment landscape, with many public and private payers explicitly and informally referencing ICER’s evidence reports in their coverage and pricing decisions.

As ICER’s importance in the value-assessment space has grown in recent years, stakeholders have been paying close attention to see how ICER would respond to critiques that its health utility estimates are discriminatory, concerns that the organization has failed to adhere to its principles for application of a modified societal perspective base case analysis, and lack of differentiation by disease state or progression. Some stakeholders were looking to ICER to incorporate additional elements of value (e.g., those described in ISPOR’s Value Flower) quantitatively in its methodologies or to reduce or remove the use of QALY/evLYG shortfalls as a proxy for disease state severity, but ICER did not propose these changes.

Some of ICER’s proposed methods and process changes reflect continued focus in the healthcare sector on addressing health inequities, and changes to the comparative clinical effectiveness section align with increasing industry-wide focus on diversity in clinical trials. If finalized, the grading system ICER uses to measure diversity in clinical trial data will further incentivize manufacturers to recruit diverse patient populations, in addition to the Food and Drug Administration’s draft guidance to increase clinical trial diversity.

Lastly, ICER’s single, short public comment period (June 5–30) for this year’s proposed framework updates is a departure from previous update cycles. In the past, ICER has solicited public comment on particular topics of interest before releasing draft VAF updates for additional public comment.  This year, it is employing an expedited update cycle with only one round of public comments. The resulting final VAF will be used for ICER reviews in 2024–2027. Manufacturers and other healthcare stakeholders have a limited time window to review ICER’s proposed changes and comment on those with more significant implications to treatment valuation results.

For More Information

Avalere’s expertise in value assessment supports clients throughout the healthcare industry in understanding how evolving methodologies may affect evaluation of a product’s value. To learn more about how Avalere can help your business respond to the draft VAF update and prepare for potential changes, connect with us.