The Institute for Clinical and Economic Review 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 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:
“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.