ICER Seeks Comments for Proposed Changes for Orphan Drugs Assessments

The Institute for Clinical and Economic Review (ICER) is seeking public comments on its recently released outline of proposed adaptations involving the assessment of certain treatments for ultra-rare conditions, also known as orphan drugs.

The proposed changes are intended to provide information for decision makers regarding the practical and ethical challenges involved in potential major advances for ultra-rare conditions. The adaptations are also intended to complement the ICER’s recent update to its value assessment framework.

In order to be considered for an adapted approach, a treatment must be expected to be used in a population of less than 10,000 with a minimal chance of future indication, and must demonstrate potential to majorly improve the quality of life or the length of life for the patient.

The key statements and proposed changes in the proposal include:

1. ICER will continue with the same standard of evidence when rating comparative clinical effectiveness, but will offer context regarding the challenges of producing evidence for the treatments and for identifying data for long-term safety.
2. ICER will continue developing cost-effective models for new treatments, but models for ultra-rare conditions will have a broader range of cost-effectiveness results—from $50,000 per quality-adjusted life year (QALY) to $500,000 per QALY—and will calculate a value-based price benchmark from the standard range. However, these reports will indicate that decision-makers often give special weighting to other benefits, causing coverage and funding decisions at higher price—resulting in higher cost-effective ratios for the treatments.
3. The report sections “other benefits and disadvantages” and “contextual considerations” will include evidence and perspectives of the potential positive treatment effects on family and the community.
4. ICER will conduct a collaborative process to develop a template for information in reports on research, development, and relative costs for ultra-rare condition treatments.

“Ultimately, the purpose of these proposed adaptations to the ICER value framework is to provide a backbone for rigorous evidence reports that, within a broader mechanism of stakeholder and public engagement, can help the United States address these tensions through an open, collaborative process,” the proposal said.

ICER is opening the discussion of these proposed updates for 60 days, until Monday, September 25, 2017; however, the National Pharmaceutical Council (NPC) suggested that this amount of time is not sufficient and cited other concerns.

“We are just at the start of this important dialogue, and given the ethics of how best to treat orphan diseases, we don’t want to rush through the conversation about how we address these challenges in the United States,” NPC published in a blog post. “There may be a temptation to look to the ICER framework as the first option or only approach to considering how to value orphan drugs, but we don’t want to further limit options, especially when so few already exist for patients with rare conditions.”

Following the 60-day period, ICER will reflect on the public’s comments and seek more feedback from stakeholders before releasing a final version of the adaptations in fall 2017.