Institute for Clinical & Economic Review Finalizes Method Adaptations for Assessing Potential Cures

BOSTON, Massachusetts, Nov. 12 -- The Institute for Clinical and Economic Review issued the following announcement:

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- Collaborative project with NICE and CADTH leads to important adaptations for ICER's methods including creation of optimistic and conservative benefit scenarios; new dimensions of benefits and contextual considerations for voting; and two new scenarios that display the potential impact on pricing that would result from sharing the savings of downstream cost offsets between society and the manufacturer

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The Institute for Clinical and Economic Review (ICER) has finalized a set of adaptations to its value assessment framework to be applied when assessing potential cures and other treatments that qualify as high-impact "single or short-term therapies," or SSTs. These new methods have emerged from a year-long collaborative project with the National Institute for Health and Care Excellence (NICE) in the UK and the Canadian Agency for Drugs and Technologies in Health (CADTH), and they have evolved from preliminary proposals with ongoing input from multiple stakeholder groups. It is important to note that these methods adaptations will be implemented by ICER and do not represent preliminary judgments on methodology by either NICE or CADTH. The newly adapted methods will be launched by ICER with reviews commencing in 2020 as part of ICER's overall update to our value assessment framework.

"Patients and caregivers are celebrating the first cell and gene therapies to reach the market, and they eagerly anticipate many additional one-time therapies that are poised for FDA review over the next few years," said Dr. Steven D. Pearson, ICER's President. "We need to think hard about whether the methods of technology assessment and cost-effectiveness analysis are ready to capture the potential for broader benefits of these treatments. We also need to consider whether new methods can better describe for decision-makers the important uncertainties about long-term benefits of short-term treatments. Lastly, some potential cures will offer the promise of preventing massive health care costs over a patient's lifetime; we need to ask whether the traditional methods of summing up all those cost-offsets and making it a part of a calculation of a 'fair' price makes sense for these specialized treatments.

"Our methods adaptations address each of these points and represent an important step in assuring that our assessment methods are fully modernized to meet the needs of decision-makers now and in the future. We are grateful to the many organizations that have provided suggestions and guidance throughout this process, and we are hopeful that these adapted methods will help reward innovation while supporting broad access for patients and a sustainable health insurance system."

Key methods adaptations include:

* Optimistic and Conservative Benefit Scenarios: In addition to the standard cost-effectiveness base case and associated sensitivity analyses, ICER will now develop two specific scenario analyses to reflect an optimistic and a conservative assumption regarding the benefit of SSTs under review. These scenarios will test uncertainty regarding the duration of benefit, the magnitude or quality of the benefit, or the proportion of patients achieving clinical benefit over their lifetime. These scenario analyses will be presented in conjunction with the base case for consideration by the independent appraisal committees.

* Additional Elements of Value: For all ICER reviews, including non-SSTs, the independent appraisal committees will now vote on additional domains of "potential other benefits or disadvantages." These include a potential advantage for therapies that offer a new treatment choice with a different balance or timing of risks and benefits that may be valued by patients with different risk preferences; a potential advantage for therapies that, if successful, offer the potential to increase access to future treatment that may be approved over patients' lifetime; and a potential disadvantage for therapies that, if not successful, could reduce or even preclude the potential effectiveness of future treatments through auto-immunity or other mechanisms.

* Hypothetical "Shared Savings" Scenarios: High-impact SSTs have the potential to lead to very large cost offsets by preventing the need for expensive, chronic treatments. In recognition of the economic surplus that is achieved through substantial cost offsets associated with certain new treatments, ICER will now publish two additional scenario analyses within every assessment of SSTs, and assessments for non-SSTs when relevant. In the first new scenario, 50% of the lifetime health system cost offsets from a new treatment will be assigned back to the health system. In the second scenario, cost offsets will be assigned entirely to the new treatment but capped at $150,000 per year. These two informational scenarios will not be presented as normative guides to pricing and will not replace ICER's established approach for determining value-based price benchmarks.

The complete list of methods adaptations is available within the final document. These adaptations were developed through a year-long public process that involved engagement with all relevant US stakeholder groups, as well as international health technology assessment agencies. As background to these methods changes, ICER previously posted a draft set of proposed methods adaptations, as well as 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 NICE and CADTH but, as noted earlier, do not reflect the official position of either collaborating organization. The specific methods adaptations in this document will be implemented by ICER alone, and they do not necessarily reflect the judgments of NICE or CADTH about if or how they will adapt their own assessment methods going forward.

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

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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 (http://www.icer-review.org/).