— Updates to methods and public meeting procedures reflect experience with previous framework, benchmarking with other HTA groups, and input from external organizations and individuals across the US health system —
BOSTON, September 25, 2023 – 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 upcoming webinar on September 28th, President Steven Pearson, MD, MSc, and President-Elect Sarah Emond, MPP, will summarize the philosophy behind ICER’s approach to value assessment, as well as several key updates that will be implemented in the near term.
The assessment framework updates are based on ICER’s ongoing efforts to evolve our methods to 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 health technology assessment (HTA) organizations around the world and consideration of input from numerous organizations and individuals across the US health system. During this process, ICER received feedback from over 30 organizations.
“ICER’s value assessment framework serves an important purpose: to create a clear picture of what we are trying to accomplish and how we go about it,” said Dr. Pearson. “We use the framework as a guidebook to ensure that all participants in our reviews, and all stakeholders using or impacted by our work, understand what our goals are, the technical methods we use, and the ‘rules of the road’ for engaging with the process. We want the framework to empower stakeholders to fully participate in every stage of our work, because only with that engagement will our work reflect the broad perspectives needed to ensure that value assessment moves our health care system toward a future of fair pricing, fair access, and sustainable innovation.”
Key updates to ICER’s methods and procedures include:
- ICER will evaluate the demographic diversity of clinical trials (and implement other methods adaptations related to health equity). Pivotal trials of interventions under review will be rated on the relative diversity of race/ethnicity, sex, and age (older adults). New steps will also be introduced to evaluate the relative effectiveness of interventions in relevant subpopulations, including those that may be defined by race, ethnicity, sex, and age. These new approaches arose from investigation of a broad set of HTA methods related to equity, as described in our white paper on Advancing Health Technology Assessment Methods that Support Health Equity.
- ICER will invite the manufacturers of drugs under review to provide a written description of how patients were involved in the design of the clinical trial program. The FDA’s Patient-Focused Drug Development (PFDD) program has been an important step in acknowledging the critical importance of capturing outcomes that matter most to patients in the clinical development program for new drugs. Many of the PFDD efforts have yet to be realized in evidence currently being produced for new drugs, as changes to the development of clinical trial programs takes time. However, some manufacturers have made important strides in incorporating the patient perspective into their drug development programs. Manufacturers will be encouraged to provide ICER with a description of the methods used to collect patient experience data, and to detail how they identified the outcomes most important to patients in their clinical trials. We will include any written descriptions received from manufacturers in the Evidence Report.
- ICER will adopt new methods to ensure that cost-effectiveness analyses done according to a modified societal perspective have “non-zero” inputs for impacts on productivity for the patient and caregivers, even when direct data are lacking. The potential impact of treatment on the ability of patients to return to their full work and family activities is an important part of the broader value of treatments when assessed through a societal perspective. All too often clinical trials fail to gather this kind of information, leading to conservative “zero” assumptions that treatment does not improve productivity. ICER will now create estimates for “non-zero” productivity effects through an indirect approach using published relationships between patient utility scores and US-based patient time use data.
- ICER will develop methods to perform cost-effectiveness scenarios related to potential price decreases for drugs later in their market lifespan due to generic competition or Medicare drug price negotiation. Due to policy changes related to the Inflation Reduction Act, it is reasonable to assume that the net price increase for many Medicare drugs will not rise faster than the rate of inflation after launch. In addition, many of these drugs will face generic competition at some point or are likely to fall under the provisions for Medicare price negotiation nine years after approval for small molecule products and 13 years after approval for biological products. There are conceptual as well as practical challenges to factoring in a price decrease later in the market lifespan of a drug, so prior to changing our approach to include price dynamics within a scenario analysis, we will engage with our Health Economics Council, Methods Advisory Group, and other researchers and stakeholders, including international Health Technology Assessment bodies, to test the feasibility and impact of how best to include pricing dynamics within cost-effectiveness analyses.
- ICER will modify public meeting voting questions to capture additional benefits beyond health and special ethical priorities related to the treatment and condition. ICER will modify the voting questions used to capture elements and considerations that may not be directly measured in clinical trials or cost-effectiveness modeling but are relevant for judgments of the overall value of treatment. These voting questions address considerations of unmet need/severity, broader impact on caregivers, and different dimensions of the potential impact of treatment on health equity.
- ICER will lead a process to evaluate the potential application of the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) framework. Methods continue to evolve in academic health economics and across HTA bodies on ways to reflect individual and/or societal value judgments related to the severity of the condition and risk aversion. Standards for best practices in this area are in early stages of evolution and currently vary widely, making it is premature to adopt new methods at this time. Nonetheless, ICER believes that the GRACE framework merits further consideration, and so we will undertake a focused exploration of how to pilot test this framework in conjunction with our Health Economics Council and other external experts.
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.