ICER Seeks Public Comment on Proposed Methods Adaptations for Assessments of Potential Cures and Other Transformative Therapies

— Proposed adaptations help manage increased uncertainty, capture new value dimensions for appraisal committee voting, and adopt a “shared savings” approach to apportion potential cost offsets between innovators and the US health system —

— Public comment open through September 6 —

BOSTON, August, 6, 2019 – The Institute for Clinical and Economic Review (ICER) has released a draft set of proposed adaptations to its value framework to be applied in the assessment of potential cures and other treatments that qualify as “single or short-term transformative therapies,” or SSTs. A broader discussion of these proposals, as well as a summary of the advantages and disadvantages of alternative methods, is available in the companion technical brief. While these proposals do not fundamentally alter ICER’s approach to value assessment, they are intended to equip decision makers with a more reliable and transparent evaluation of SSTs’ uncertainty, value, and value-based pricing. The proposed adaptations will complement and build upon ICER’s ongoing update to our 2020 value assessment framework.

“Cell and gene therapies are starting to provide truly transformative advances for patients and their families, particularly those with conditions for which there has not been any effective treatment before,” said Dr. Steven D. Pearson, ICER’s President. “At the same time, however, many of these therapies are introduced with much higher levels of uncertainty about their long-term safety and effectiveness than standard treatments, and patients and insurers are being asked to pay extremely high prices upfront for the promise of long-term benefit. These tensions raise important questions for how best to adapt health technology assessment methods to ensure that all aspects of the value of these treatments are fully evaluated and the uncertainty put into perspective for decision-makers. We are in the midst of a year-long project to examine our assessment methods by getting input from stakeholders and methods experts, and we look forward to broader public comment on these draft proposals.”

These proposed methods adaptations are intended only for therapies that are delivered through a single intervention or a short-term course of treatment and that demonstrate a significant potential for substantial and sustained health benefits extending throughout patients’ lifetimes. SSTs include two subcategories: potential cures that can eradicate a disease or condition, and transformative therapies that can produce sustained major health gains or halt the progression of significant illnesses. As background to these proposed methods changes, ICER is also posting a companion technical brief examining the potential advantages and disadvantages of various alternative methods for the assessment of SSTs. The conceptual and empirical work in the technical brief was developed in collaboration with staff at the National Institute for Health and Care Excellence (NICE) and the Canadian Agency for Drugs and Technologies in Health (CADTH), but do not reflect the official position of either collaborating organization. The specific draft methods adaptations in this document are proposed by ICER alone, and they are not intended to reflect opinions of NICE or CADTH.

The key proposals in this document include:

  1. Standardizing the inclusion of multiple methods to evaluate and address uncertainty resulting from evidentiary limitations, including cure proportion modeling techniques; threshold analyses to determine time horizons needed for assumptions about short-term efficacy to remain true in order for SSTs to reach standard cost-effectiveness thresholds; and mechanisms to use probabilistic sensitivity analysis to provide value-based pricing recommendations linked more directly to the degree of uncertainty in the data.
  2. Adding two additional elements to ICER’s list of “potential other benefits or disadvantages.” The first will include consideration of whether therapies offer special advantages by offering patients a different timing or balance of risks and benefits, such as a greater risk of death in the short-term for a greater chance of a long-term cure. The second element will consider the potential for treatments to alter or even preclude the chance of effectiveness of future treatments.
  3. Producing an alternative “shared savings” cost-effectiveness scenario in which the economic surplus of SSTs is shared in different proportions between the innovator and the health system. For example, one scenario will demonstrate the impact on recommended value-based prices if 100% of cost offsets from successful treatment in the economic model accrue to the innovator during the first 12 years, after which 100% of cost offsets accrue to the health system. This approach is modeled to reflect the likelihood that many SSTs will not face the equivalent of generic competition and will therefore allow upfront prices to allocate a much greater share of the economic surplus to innovators compared to chronically delivered therapies.

The full outline of proposed changes will be open to public comment until September 6 at 5:00 p.m. ET. All comments should be emailed as an attached Word document to

On September 17 at 8:30 a.m. ET, ICER will livestream a meeting of invited stakeholders to gather additional input on whether and how to adapt its standard assessment methods for SSTs.

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.