Patients living with sickle cell disease could run into more insurance barriers as a result of a pending report on two potentially groundbreaking SCD treatments.

The Institute for Clinical and Economic Review, which receives support from insurance companies, has begun its financial analysis of how two new sickle cell disease treatments, crizanlizumab and voxelotor, will affect insurance profits. Patient advocates are worried that ICER’s review will mean more insurance obstacles, delays and barriers for patients desperately seeking access to new and improved treatments.

“The SCD community needs access to effective and innovative treatments,” says Terry WIlcox, co-founder and executive director of Patients Rising, a non-profit organization that helps patients overcome insurance barriers. “Insurance companies use ICER’s insurance profitability reports to deny patients access to the right treatment.” 

She adds, “Doctors should decide the right treatment for every member of the SCD community, not insurance profits.”

Clear Need for New Sickle Cell Disease Treatments

Sickle cell disease, which affects approximately 100,000 Americans, describes a group of painful and incurable disorders in which the body produces irregular hemoglobin cells.

Hemoglobin is an essential molecule for delivering oxygen to red blood cells throughout the body. Especially prevalent among patients of color, SCD causes distortions in red blood cells into a sickle, or crescent, shape.

“Painful episodes can occur when sickled red blood cells, which are stiff and inflexible, get stuck in small blood vessels,” according to the National Institutes of Health. “These episodes deprive tissues and organs of oxygen-rich blood and can lead to organ damage, especially in the lungs, kidneys, spleen, and brain.”

To manage the pain associated with sickle cell disease, the SCD community is often prescribed opioids that don’t cure the disease, but merely treat the pain. That leaves some SCD patients vulnerable to opioid addiction and other patients struggling to overcome the barriers and stigma associated with responsible opioid use for pain management.

Other than opioids, SCD patients are left to choose between just two FDA-approved treatments.

“There is clearly a large unmet need for additional treatments for SCD,” ICER writes in its background and scoping document, “Crizanlizumab, L-Glutamine, and Voxelotor for Sickle Cell Disease: Effectiveness and Value.”

Promising New SCD Treatments: Crizanlizumab & Voxelotor

New treatments on the horizon are changing the landscape of for sickle cell disease. This summer, the Food and Drug Administration granted priority review status for crizanlizumab, an innovative treatment that works to prevent vaso-occlusion, or recurrent acute pain, crises. 

Another equally promising treatment, voxelotor, is “an HbS polymerization inhibitor that reversibly binds to hemoglobin to stabilize the oxygenated hemoglobin state, thus shifting the oxyhemoglobin dissociation curve.” Because of its potential to help SCD patients, the FDA has accepted the treatment into its rolling New Drug Application program, which allows for an accelerated approval process.

Flawed Timeline: ICER Decides Before FDA Approval


Although the FDA won’t reach its approval decision on both drugs until 2020, that hasn’t stopped the private, unregulated ICER from conducting its own insurance profitability report. 

Experienced patient advocates like Wilcox of Patients Rising point out that ICER initiated its review and opened public input on August 30th — months before the FDA. 

“The order is totally wrong,” explains Wilcox of Patients Rising. “Before obtaining safety information from the FDA, ICER will have produced its Draft Scoping Document, Public Comments, Stakeholder List, Revised Scoping Document, Research Protocol, Model Analysis Plan, Draft Evidence Report, and Draft Voting Questions.”

ICER’s review of new sickle cell disease treatments not only precedes the FDA’s safety evaluations, but also prevents patients from sharing their voices on the value of new treatments. 

“We’re standing with the SCD community and taking action,” says Wilcox of Patients Rising. “The SCD community should not be singled out by ICER. Their voices must be heard.”

Take Action: Share Your Patient Input on New SCD Treatments

Are you a patient living with sickle cell disease? Here are three ways that you can take action:

  1. Sign Up for ICER Updates: Join the Patients Rising email list for more updates on the latest news from ICER at
  2. Post on Social Media #ICERWatch: Share your patient voice via social media using #ICERWatch.
  3. Share Your Story: Contact Patients Rising to share your personal patient story.