–Broader societal considerations and expanded cost-effectiveness ranges will be included for treatments targeting fewer than 10,000 patients–

BOSTON, November 13, 2017 – Following a nine-month public process, the Institute for Clinical and Economic Review (ICER) today posted the final modifications to its value assessment framework for reviews of certain treatments for serious, ultra-rare diseases. The modified framework applies to therapies that, based on approved indications and planned clinical trials, will be eligible to treat no more than approximately 10,000 US patients. These modifications are effective immediately for all relevant ICER assessments, including the ongoing reviews of new treatments for biallelic RPE65-mediated retinal diseasehemophilia A, and cystic fibrosis.

“Incentivizing pharmaceutical innovation while ensuring affordable access to life-improving medicines creates a natural tension in any health care system, and this tension is magnified by the evidentiary and ethical challenges associated with diseases that affect very small populations,” said Steven D. Pearson, MD, MSc, President of ICER. “Our modified framework for ultra-rare diseases establishes a transparent process of incorporating these additional considerations, assessing the effectiveness and value of each therapy, and recommending a price that aligns with the full benefit a patient will receive. Given the pharmaceutical industry’s growing focus on ultra-rare diseases, ICER’s modified framework represents a major milestone toward helping US patients gain access to the therapies they need at a price they and the country can afford.”The modifications announced today complement and build upon the general ICER value assessment framework that guides ICER’s methods of economic analysis and stakeholder engagement.

Key modifications for treatments of ultra-rare diseases include:

  • ICER will provide context around potential evidence limitations – including the feasibility of conducting randomized controlled trials, validating surrogate outcome measures, and obtaining long-term data on safety and the durability of clinical benefit.
  • ICER will adapt its analyses to provide cost-effectiveness results for the broader range of $50,000-$500,000 per quality-adjusted life year (QALY) gained.
  • ICER will calculate value-based price benchmarks using the standard range of $100,000-$150,000 per QALY, but will indicate in all reports that decision-makers often give special weight to additional benefits and contextual considerations when determining coverage of more expensive treatments for ultra-rare diseases.
  • If the treatment has a significant effect on broader societal costs — such as patient or caregiver productivity, education, or disability — ICER will include these costs in a separate analysis presented alongside the standard health system perspective cost-effectiveness results and associated value-based price benchmarks.
  • Manufacturers will have the opportunity to provide information about the development and manufacturing costs for their product. This information will be included, unedited, in a new dedicated section of each ICER report.

The complete summary of framework modifications is available at http://www.icer-review.org/material/final-ultra-rare-adaptations/.

This modified framework is the culmination of a nine-month public process of engagement with the rare disease patient community, the life sciences industry, and US health care payers. As part of this process, ICER published a white paper discussing the evidentiary and ethical challenges associated with evaluating treatments for rare conditions, hosted a multi-stakeholder policy summit, and accepted public comment from more than 50 organizations and individuals regarding an initial proposal of framework modifications. ICER looks forward to ongoing input from the entire stakeholder community, and it plans to launch a formal update to these methods in 2019.

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.