ICER Seeks Public Comment on Proposed Value Assessment Framework for Treatments That Represent a Potential Major Advance for Serious Ultra-Rare Conditions

— ICER’s fundamental approach to value assessment remains constant with proposed changes for special circumstances– 
Boston, Mass., July 25, 2017 – The Institute for Clinical and Economic Review (ICER) has released an outline of proposed adaptations to its value framework to be applied in the assessment of certain treatments for ultra-rare conditions.  ICER proposes that its fundamental approach to value assessment remain constant; however, several key changes are proposed to provide decision-makers with information reflecting the distinctive practical and ethical challenges associated with potential major advances for serious ultra-rare conditions.

To qualify for a distinctive assessment approach, ICER proposes that a treatment must be expected to affect a patient population of fewer than 10,000 individuals, with little chance of future expansion of indication or population, and it must offer a major gain in quality and/or length of life for patients with a serious condition.  The proposed updates will be open to public comment for 60 days until Monday, September 25, 2017.

The proposed adaptations are meant to complement and build upon the recent update to the ICER value assessment framework that will guide ICER’s methods of evidence analysis and stakeholder engagement during the coming two year report cycle 2017-2019.  As noted during that update process, ICER was aware of the importance of distinguishing methods for assessments of treatments for rare conditions.  To provide a basis for stimulating early discussion of these issues, ICER produced a white paper on the evidentiary and ethical challenges associated with evaluation of treatments for rare conditions.  ICER then hosted an all-stakeholder meeting on May 31, 2017 to gather further input on whether and how to adapt its standard assessment methods.

Among the key methods statements and proposed changes proposed in this document are:
  1. ICER will continue to apply the same standards of evidence for its ratings of comparative clinical effectiveness, but proposes a format in which it will provide specific context regarding the potential challenges of generating evidence for these treatments, including considerations of challenges to conducting randomized controlled trials, to validating surrogate outcome measures, and for obtaining long-term data on safety and on the durability of clinical benefit.
  2. ICER will continue its practice of developing a cost-effectiveness model for every new treatment; however, analyses for ultra-rare conditions will provide a broader range of cost-effectiveness threshold results, from $50,000 per quality-adjusted life year (QALY) to $500,000 per QALY. ICER will calculate a value-based price benchmark for these treatments using the standard range from $100,000 to $150,000 per QALY, but will add language in all report formats indicating that decision-makers in the US and in international settings often give special weighting to other benefits and to contextual considerations that lead to coverage and funding decisions at higher prices, and thus higher cost-effectiveness ratios, than applied to decisions about other treatments.
  3. For report sections on “other benefits and disadvantages” and “contextual considerations,” ICER will include a broader frame to seek evidence and perspective on the potential for these treatments to affect positively the family, school, and community.  Information will also be sought on the potential impact of new treatments on the infrastructure for screening and care of the affected individuals.
  4. ICER will conduct over the coming year a collaborative process through which it will seek to develop a template for providing information in its reports on the research, development, and other relevant costs related to new treatments for serious ultra-rare conditions.  Until this template is completed, ICER will work with individual manufacturers of treatments under review to determine what, if any, information related to the costs of development can be shared as part of the public deliberation regarding the value of these treatments and their appropriate pricing.

The proposed updates were developed following a day-long, multi-stakeholder policy summit held in Washington, DC. During the summit, representatives from the rare disease patient community, life sciences, clinical, and payer communities convened to discuss methods for assessing the value of new drugs for rare conditions, including how these methods can be applied to recommend fair prices that reflect the value of orphan drugs to patients and the health system, allowing for broader insurance coverage for innovative new treatments.

The full outline of proposed changes will be open to public comment for 60 days, until Monday, September 25th. All comments should be emailed as an attached Word document to publiccomments@icer-review.org.
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.