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20 Years of Thought Leadership

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With more than 20 years of thought leadership in healthcare, Saira’s blog offers insights into health policy legislation and regulation, health advocacy, trends in rare and ultra rare diseases, and more.

Check back for valuable posts.

 
 

ICER - True to Form!

ICER is doing what they ALWAYS do.  After putting their “ultra-rare” framework out for comments, they are just going ahead and applying it to Hemophilia A.  Here’s a snippet from their draft scoping document:

“ICER has posted and asked for public comment on proposed changes to its value assessment framework for treatments of certain ultra-rare conditions (https://icer-review.org/material/odapsproposed-changes/).  While awaiting those comments, we are considering whether emicizumab should be evaluated under these proposals, recognizing that public comments received and further reflection may lead to some revisions to this modified set of methods and procedures. The proposed criteria are:

  • The treatment is envisaged for a patient population of fewer than 10,000 individuals

  • There is little chance of future expansion of indication or population that would extend the size of the treated population above 20,000 individuals

  • The treatment potentially offers a major gain in improved quality of life and/or length of life

While the population of hemophilia A patients in the US with inhibitors is likely much less than 10,000, and treatment with emicizumab offers potential major gains in quality of life, future expansion of use to the broader population of patients with hemophilia A could extend the size of the treated population to above 20,000 individuals.  As such, we plan to evaluate emicizumab under the usual ICER value assessment framework.”

NOTE:  At the very start of their document, ICER states that the estimated US prevalence is approximately 20K.

Saira Sultan