— NICE in the UK and CADTH in Canada among international agencies participating; Public input on key methodological issues is being accepted through February 20, 2019 —

BOSTON, January 23, 2019 – The Institute for Clinical and Economic Review (ICER) today announced a new project to develop and test alternative methods for the evaluation of potentially curative treatments and for translating the results of cost-effectiveness analyses into recommendations for value-based price benchmarks. In this work, ICER will collaborate with methodology experts, stakeholders, and several leading international health technology assessment (HTA) groups, including both the United Kingdom’s National Institute for Health and Care Excellence (NICE) and the Canadian Agency for Drugs and Technologies in Health (CADTH). The goal of this initiative is to ensure that assessment methods are tailored appropriately to the distinctive nature of the evidence base for potential cures. The initiative will inform ICER’s 2019 update to its value assessment framework while seeking to build consensus across HTA groups in anticipation of a rising tide of gene therapies and other potential cures.

“Patients and those who care for them eagerly anticipate the coming wave of treatments that may cure a wide range of illnesses,” said Steven D. Pearson, MD, MSc, President of ICER. “The science is undeniably exciting. But when these treatments are first launched, which is when pricing and coverage decisions have to be made, the evidence on the long-term value of these treatments may be extremely limited. Given the stakes for patients, and the high prices that one-time curative treatments are likely to command, we are taking the lead now to assemble and work with key experts to ensure we are using the best possible methods for assessing the value of these treatments. If we don’t, we risk undervaluing potential cures, or over-valuing them, either of which would ultimately harm patients and the health care system as a whole. Exploring different approaches to assess the value of potential cures, and building some early consensus across groups that will be doing this work in the US and internationally, will be essential to arm the policymaker, payer, and manufacturer communities with a platform that can reward innovation while supporting a sustainable health insurance system.”

An open input period is now open during which ICER hopes to receive ideas and guidance regarding the key methodological questions highlighted below:

  • How should value-based prices for potential cures reflect substantial uncertainty regarding clinical safety and effectiveness due to limitations in study design, outcome measures, and the size and duration of clinical trials?
  • How should value-based prices for potential cures reflect uncertainty regarding inclusion of additional elements of value that may be important for potential cures, but which are not part of standard cost-effectiveness methods?
  • How should value-based prices for potential cures reflect extreme magnitudes of lifetime health gains and cost offsets that are far beyond those generated by traditional therapies?

Comments can be submitted by email to publiccomments@icer.org and to be integrated usefully into the research effort must be received by 5 p.m. ET on February 20, 2019. ICER will post a draft white paper for additional public comment sometime this summer, before finalizing the white paper and methodology recommendations before the end of the year.

This initiative is made possible with financial support from the Commonwealth Fund and the National Institute for Health Care Management.

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.