— Reflecting experience with current framework, benchmarking with other HTA groups, and public input, proposals include new emphasis on real-world evidence, revised approach to voting on contextual considerations, and a formal process for report updates at 12 months after initial release —

— Proposals to be discussed during a webinar on September 4; Public comment now open through October 18 —

BOSTON, August 21, 2019 – The Institute for Clinical and Economic Review (ICER) today proposed a set of changes to its methods for conducting value assessments beginning in 2020. These proposals are based on ICER’s experience in methods development for health technology assessment (HTA) reports in the US, benchmarking with HTA agencies around the world, and input from stakeholders across the US health system, including suggestions from 97 organizations and individuals who submitted letters during a recent Open Input period.

“Since our inception, ICER has strived to combine state-of-the-art academic methods of technology assessment with procedures that create structures for active engagement of all stakeholders and that ultimately achieve the highest level of transparency possible,” said Steven D. Pearson, MD, MSc, President of ICER. “We also need to stay willing to change. We need to listen constantly to those engaged in our reviews and to explore ways to change our methods to keep evolving and improving. We thank the many organizations and individuals who have helped us hone our methods over the years, and also those who provided their formal suggestions during our recent call for input. Our proposed changes are presented with discussion of why we think they would be an improvement. In some areas, we are proposing no changes and have given further arguments addressing why we feel other options would have greater disadvantages than advantages. We look forward to further public comment to help guide us toward our final decisions on our methods update later this year.”

Key proposed changes, updates, and restatements of current methods include:

  1. Augmenting Efforts to Use Real-World Evidence: Responding to the most common suggestion we received from patient advocacy organizations, we reaffirm our ongoing commitment to use existing real-world evidence (RWE) and commit to exploring new collaborative relationships with organizations to generate RWE that can complement published data sources.
  2. 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 believe a new Likert-scale voting structure will send a more tangible and transparent signal of the relative importance of the broader factors that should guide decision-makers in choosing whether a treatment should be priced toward the low end or the high end of ICER’s value-based price benchmark range.
  3. Reaffirming Commitment to Multiple Cost-effectiveness Outcome Measures: ICER reaffirms our commitment to include a broad perspective on cost-effectiveness in all assessments, measuring both a treatment’s Quality-Adjusted Life Years (QALY) gained, as well as the complementary Equal Value of Life Years Gained (evLYG). By understanding a treatment’s cost per evLYG, as well as its traditional cost per QALY, policymakers can be reassured that they are considering information that poses no risk of discrimination against any patient group.
  4. Standardizing Cost-Effectiveness Thresholds: All of ICER’s reports will provide a set of pricing benchmark results using standardized cost-effectiveness thresholds from $50,000-$200,000 per QALY and per evLYG, including those for treatments of ultra-rare disorders. Our value-based price benchmarks will continue to use the threshold ranges of $100,000-$150,000 per QALY and per evLYG.
  5. 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.
  6. Addressing Alternative Modeling Assumptions: ICER will add a “Controversies and Uncertainties” section to the cost-effectiveness chapter of our reports to broaden the discussion of alternative model structures and assumptions suggested by manufacturers and other stakeholders.
  7. Applying More Precise Evidence Ratings: ICER will expand our evidence rating categories to more effectively distinguish between situations that may share a high certainty of at least a comparative net health benefit but have the potential for widely different best-case scenarios.
  8. Providing a Crosswalk Between ICER Evidence Ratings and Those of the German HTA System: In recognition of the growing international use of our assessments, ICER will now identify in each report how our own evidence rating would translate into the rating system Germany’s HTA agency uses to describe a treatment’s added clinical benefit.

More details about these and additional proposals can be found in the full document.

Valuing Potential Cures and Other Short-Term Transformative Therapies

In a parallel initiative, ICER is also developing new methods to be applied in the assessment of potential cures and other treatments that qualify as “single or short-term transformative therapies,” or SSTs. These complementary methods will be finalized by November 15, 2019, and will be folded into our Final 2020 Value Assessment Framework.

Patient Webinar and Public Comment

ICER invites all patient organizations and the general public to attend a webinar on September 4, 2019 to learn more about ICER’s mission, the rationale behind the proposed framework updates and how the feedback we received during the open input period informed our proposals.

The proposals announced today will be subject to a public comment period through 5 p.m. ET on October 18, 2019. Comments should be provided in Microsoft Word documents (no PDFs), no more than 10 pages, using 12-point Times News Roman, and submitted via email to publiccomments@icer.org.

After reviewing all public comments, ICER will reflect further and make any final changes before releasing our Final 2020 Value Assessment Framework on December 18, 2019. That final document will present a comprehensive description of all elements of the value assessment framework, and will be released with several companion updated documents, including special methods adaptations for treatments of ultra-rare diseases, the ICER Evidence Rating Matrix, the ICER reference case for economic evaluations, and ICER guides to patient and manufacturer engagement.

The full timeline for updating our Value Assessment Framework is available here.

About ICER

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.