May 29, 2020

ICER’s Value Framework: A Work in Progress for Women’s Health

By Sarah Wells Kocsis, MBA, Vice President of Public Policy

New medical treatments may have drastically different value to different people because drugs don’t work the same in every individual. For patients who have not yet found a treatment that works for them, a new medicine may be much more valuable than for a patient who has already found an effective therapy.

Because assessing the value of health care interventions is so challenging, SWHR engages with organizations that conduct value assessments to ensure women’s unique needs as patients, caregivers, and health care decision-makers are taken into account.

Earlier this year, SWHR shared its value assessment principles for women and 11 detailed recommendations with the Institute for Clinical and Economic Review (ICER) as it worked to improve its value framework, the tool that outlines how ICER conducts its value assessments.

ICER takes a population-level approach to value assessment and estimates a health care intervention’s value from two perspectives: long-term “value for money” and short-term affordability. Other frameworks such as the National Comprehensive Cancer Network (NCCN) Evidence Blocks take a patient-level approach by providing patients and clinicians with clinical and economic information about therapies to inform treatment choices. Both approaches to value assessment consider factors such as burden of illness and indirect costs (e.g. productivity), but they incorporate and measure them in different ways. SWHR believes that it is vitally important that all frameworks appropriately capture the value of factors relevant to women, such as caregiver burden and ability to function/work.

Despite ICER embracing several of SWHR’s recommendations, its updated framework remains a work in progress to adequately evaluate the value of health care interventions for women.

Accounting for Patient Diversity

Capturing differences within patient groups is necessary to understand variation in response to treatments. Biological sex, gender, age, stage of illness, and comorbidities can be contributing factors to a patient’s response to treatment. Women, for example, are disproportionately affected by migraine disease and are more likely than men to experience longer and more intense migraine attacks and have more comorbid conditions.

To rigorously capture patient heterogeneity in value assessments, SWHR urged ICER to increase subgroup analyses and explore ways to build subpopulation value metrics into its model. For future assessments, ICER will add a formal report section on heterogeneity to highlight subpopulations in which a treatment may work. While a good first step, more can be done to measure a treatment’s value for a given patient population subgroup. All value frameworks should have the analytic capability to capture patient heterogeneity and report more than a single price for an average patient.

Elevating the Societal Perspective

In addition to measuring clinical outcomes, value frameworks should account for what matters most to patients, caregivers, and society. Factors that reflect a disease’s burden – like survival, ability to function or work, social well-being, and caregiver burden –should be included to provide a comprehensive snapshot of a treatment’s value. SWHR identified these specific factors that should be quantitatively measured in ICER’s model to achieve optimal health outcomes for women.

Additionally, ICER’s updated framework will now consider including the societal perspective in its model as a base case, but only under certain conditions — when societal care costs are “large relative to the direct health care costs” and when the impact of treatment on these costs is “substantial.” Augmenting ICER’s model in this way could represent a significant improvement, depending on how ICER defines and implements these thresholds.

Incorporating Real-World Data

SWHR called on ICER to explain its methods for incorporating real-world evidence (RWE) into future topic reviews, including discussion of when RWE may be discarded, as value assessments should use a broad range of evidence sources to demonstrate an improvement in outcomes. As part of the updated framework, ICER will pilot a process for updating original assessments that will include new RWE as a key component.

SWHR will be following along closely as this is a new role for ICER, whose traditional role has been to evaluate evidence, not generate it. SWHR calls on stakeholders, particularly manufacturers, to share their experiences and insights in generating and analyzing RWE to inform best practices for application in value assessment.

Keeping Reports Fresh

Measuring both short- and long-term outcomes is important to reflect the full value of a therapy or intervention, which is why SWHR called on ICER to regularly update its value assessment reports as new evidence emerges post-approval.

ICER’s updated framework will employ two approaches for considering new evidence:

SWHR encourages stakeholders to continue to engage with ICER as it fleshes out these processes for updating reports.

Evaluating Emerging Issues

ICER describes its 2020 framework as “an internal and external ‘rules of the road’” to guide its engagement with patients and other stakeholders, its evaluation of evidence, and its procedures for public meetings.

As ICER tackles rapidly evolving new issues, such as those related to the current pandemic, SWHR is closely monitoring how ICER applies its framework to guide reviews and encourages use of transparent processes and opportunities for patient and stakeholder participation. Transparency and stakeholder feedback are especially critical when evaluating value in response to urgent public health needs like treatments for COVID-19.

Forging Ahead

ICER is taking important steps to update its value framework, but there is room for improvement. SWHR will continue to advocate for changes to ensure value frameworks are appropriately designed and used to provide appropriate access to innovative new therapies and interventions for women. We call on clinicians, researchers, and patients to join us in constructively participating in all facets of ICER’s value assessment process to drive further meaningful improvements.  Doing so will help achieve our ultimate goal of optimizing health outcomes for women as patients, caregivers, and health care decision-makers for themselves and their families.