ICER Issues Modified Framework for Assessing Value of Treatments for Ultra-Rare Diseases

November 14, 2017

Rare Daily Staff

The Institute for Clinical and Economic Review, an independent non-profit research institute that analyzes the evidence on the effectiveness and value of drugs and other medical services, issued final modifications to its framework for assessing the value of treatments for serious, ultra-rare diseases.

The modification of the framework follows a nine-month public process that included engagement with the rare disease patient community, the life sciences industry, and U.S. healthcare 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.

The framework applies to therapies that are eligible to treat no more than 10,000 U.S. patients including certain treatments for inherited eye disease, hemophilia A, and cystic fibrosis.

It is intended to help U.S. patients gain access to the therapies they need at a price they and the country can afford, ICER said.

“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 Pearson, 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.”

The modifications build on ICER’s general ICER value assessment framework that guides its economic analysis and stakeholder engagement. For ultra-rare disease, the modifications call for ICER to:

  • 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.
  • Adapt its analyses to provide cost-effectiveness results for the broader range of $50,000-$500,000 per quality-adjusted life year gained.
  • 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.
  • Provide a separate cost analysis alongside the health system perspective on cost-effectiveness and value-based price benchmarks if the treatment has a significant effect on broader societal costs, such as patient or caregiver productivity, education, or disability.
  • Allow manufacturers 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

November 14, 2017

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