Final modified framework for assessing ultra-rare disease drug value released by ICER

Following a nine-month public process, the USA’s Institute for Clinical and Economic Review (ICER) yesterday 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 around 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 disease, hemophilia 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 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. 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.”