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Treatments

USDA Approves New Drugs to Manage Sickle Cell Disease

OxbrytaOne of several approved treatments for sickle cell disease is Oxbryta (voxelotor), developed by Global Blood Therapeutics. Oxbryta targets the abnormal hemoglobin that causes the disease. It prevents hemoglobin from sticking together, which destabilizes the large aggregates, or clumps of sickled cells that block blood vessels and prevent blood flow in people with sickle cell disease. An important message from Ted W. Love, M.D. , GBT President and CEO: Despite this challenging, rapidly changing time, GBT Source, our support program for those prescribed Oxbryta® (voxelotor) tablets, is fully functional and remains available to support you and your patients. We continue to be available to assist with new enrollments, reimbursement, financial and copay support, and adherence and refill support. Also, we believe we currently have sufficient supply of Oxbryta to sustain patient need through the remainder of the year and into 2021.
To learn more about this and other USDA Approved medications, click here

ICER

A new report from the Institute for Clinical and Economic Review (ICER) says the new sickle cell disease treatments may not be cost effective. As a result, ICER is reviewing the information gathered from the perspective of the patient and caregiver. See information below to stay updated regarding ICER acivities. Due to the Corona Virus, the public meeting planned for 3/26/2020 is cancelled. To keep you informed, the SCDAA has a committee available that is working on the coronavirus and community education. Connect to the SCDAA website for more information
ICER shares several updates related to the ICER review for sickle cell disease:"March 13, 2020, ICER published the updated Evidence Report for sickle cell disease. The report concludes that crizanlizumab, voxelotor, and Endari are not cost effective at their current list prices. Public comments on ICER’s Draft Evidence Report for sickle cell disease are now live. To read what the sickle cell disease community wrote to ICER, click here. To see ICER’s response to the community, click here. We are so proud of the community for making your voices heard. Results from the "My Life with Sickle Cell Disease" Survey are summarized within ICER's Evidence Report. Sick Cells will also present this information during the ICER Public Meeting. Our findings from the survey provided new evidence to inform gaps in the ICER report. Again, this is a huge accomplishment for our community. Due to the Corona Virus, our March 26th public meeting to review ICER findings has been cancelled. Please refer to the sickle cell disease evidence report for their most updated findings. In response, this task force will no longer meet on a weekly basis. We will use our task force call on Tuesday morning to think creatively about how the oral commenters and expert panelists can target their comments during the meeting to address gaps in ICER's assessment. In preparation for this discussion, please review ICER's responses to the community's comments. Our discussion will help prioritize information and plan the testimonies of our public speakers."
stay updated by learning more about ICER through past and future webinars: If you're interested in learning more about the ICER Review process, Sick Cells hosted a webinar series to educate the community on value assessments and drug pricing. View all of the webinars here:Please reach out to mjalowsky@sickcells.org if you have any questions and/or if you'd like to get involved.
Currently researchers are studying a number of new treatments with the potential to reduce complications of the disease. For example, hydroxyurea, decitabine and butyrate (a food additive) appear to cause the body to make a form of hemoglobin (fetal hemo-globin) that is produced in a baby before birth. Increased levels of fetal hemoglobin appear to help prevent red blood cells from sickling. View the videos below to learn more.