— Reflecting experience with previous framework, benchmarking with other HTA groups, and input from more than 100 organizations and individuals across the US health system, these methods updates build on ICER’s commitment to principles of respect, transparency, inclusivity, and objectivity —
— Updates place stronger emphasis on use of real-world evidence, deliberation that highlights the broader context of value to patients and the health system, and the launch of a formal Patient Engagement Program —
BOSTON, January 31, 2020 – The Institute for Clinical and Economic Review (ICER) today published an updated Value Assessment Framework document outlining the principles, methodology, and deliberation procedures that will guide future ICER assessments. In an accompanying video presentation, Steven D. Pearson, MD, MSc, President of ICER, summarized the philosophy behind ICER’s approach to value assessment, as well as several key updates that will be implemented immediately.
These updates are based on ICER’s ongoing efforts to learn from patient groups and others about how to build a unique approach to value assessment that can reflect core principles of respect for all participants, transparency of methods and decision-making rationales, inclusivity of all perspectives, and objectivity grounded in rigorous evaluation of all relevant evidence. The process of updating ICER’s value assessment framework included extensive benchmarking with HTA organizations around the world and a seven-month public dialogue with more than 100 organizations and individuals within the US health system.
“ICER’s value assessment framework has one main goal: to create a clear picture of what we are trying to accomplish and how we go about it,” said Dr. Pearson. “We do this for several reasons. First, as always, we want to bring value assessment out of the back rooms of insurers and manufacturers, and make it a public exercise, open to scrutiny and input from all. We also use the framework as an internal and external ‘rules of the road’ to ensure that a core set of principles guide all facets of our engagement with patients and other stakeholders, of our evaluation of evidence, and of our procedures for public deliberative meetings. But most importantly, we want the framework to help empower patients and others to fully participate in every stage of our work, because only with that engagement will we really meet the ultimate goal of moving our health care system toward a future of fair pricing, fair access, and sustainable innovation. Patients deserve no less.”
Key updates to ICER’s methods and procedures include:
- Augmenting Efforts to Use Real-World Evidence: Responding to the most common suggestion we received from patient advocacy organizations, ICER reaffirms its ongoing commitment to use existing real-world evidence (RWE). ICER also commits to exploring new collaborative relationships with organizations to generate RWE that can complement published data sources. In addition, for drugs that were approved under accelerated approval pathways, ICER will pilot a formal process to update our original assessment after the treatment has been on the market for at least 24 months; this pilot will explore how best to develop and assess new RWE as a cornerstone of the update process.
- Expanding and Revising Voting Structure to Capture Important Potential Other Benefits and Contextual Considerations: ICER will revise and expand the categories of potential other benefits and contextual considerations upon which our independent evidence appraisal committees vote. Further, we will use a new Likert-scale voting structure to send a more tangible and transparent signal of the relative importance of the broader factors that should guide decision-makers in their overall judgments on value.
- Adding a Formal Section on Heterogeneity and Subgroups to Highlight Subpopulations in Which a Treatment May Work Best: In cases where there are knowable effect modifiers or knowable substantial differences in baseline risk, ICER will highlight these differences in our discussion of the evidence in a new subsection of each report.
- Standardizing Cost-Effectiveness Thresholds: All of ICER’s reports, including those for treatments of ultra-rare disorders, will provide a set of pricing benchmark results using standardized cost-effectiveness thresholds from $50,000-$200,000 per Quality-Adjusted Life Year (QALY) and per Equal Value of Life Year Gained (evLYG). Our health-benefit price benchmarks (formerly labeled “value-based price benchmarks”) will continue to use the threshold ranges of $100,000-$150,000 per QALY and per evLYG.
- Promoting a Modified Societal Perspective to “Co-Base Case” in Certain Cost-Effectiveness Analyses: To try to strike a balance between the ethical and other risks of a societal analysis and the potential interest of decision-makers in the results of analyses done with modified societal perspective, ICER will now present a modified societal perspective as a co-base case when the societal costs of care for any disease are large relative to the direct health care costs, and the impact of treatment on these costs is substantial.
- Creating a New Process for Re-evaluating Evidence: One year after the release of each Final Evidence Report, ICER will formally reassess whether new evidence has emerged that should be included in an update to the report, and we will issue a public statement describing next steps.
- Implementing a More Formal Patient Engagement Program: ICER is launching a formal Patient Engagement Program to augment our efforts to empower patient organizations to contribute substantively to every phase of value assessment. The ICER Patient Engagement Program will increase its span from efforts prior to topic selection all the way to efforts following the end of the assessment to advocate for better patient-centered evidence in the future.Key aspects of this new Patient Engagement Program include:
- Preparing Patient Organizations for Future ICER Assessments: In advance of beginning a new value assessment, ICER will conduct outreach to disease-specific patient groups to provide an overview of ICER procedures and facilitate connections with other patient leaders who have participated in an ICER review. For major therapeutic classes, ICER will schedule an annual discussion of the emerging pipeline of new treatments, get patient input on key priorities, and explore opportunities to gather new data on outcomes of care that are important to patients and families. For other disease areas, ICER will provide early notification to relevant patient groups when ICER has high certainty of an upcoming assessment pertaining to their focus.
- Understanding Disease Burden and Translating Patient Input into the Economic Model: ICER will continue to work with patient groups at the outset of each review to determine whether and how patients can contribute empirically to the economic model.
- Working with Patient Organizations to Identify, Use, and Generate RWE: ICER will identify gaps in key outcomes measures and work with patient groups to identify sources of RWE and potentially develop patient surveys to inform the economic model and provide additional information regarding potential other benefits or disadvantages and contextual considerations surrounding the disease and its treatment.
- Partnering with Patient Organizations After the Completion of an ICER Assessment to Encourage the Broader Adoption of Key Policy Recommendations by the FDA: At the conclusion of each review, ICER will invite participating patient groups for a formal debrief on the experience. In addition, ICER will offer to co-author and promote a letter to the FDA and other stakeholders with proposals for improving the generation of specific patient-relevant data as part of the drug development process in that disease area.
The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.
ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.