Cecilia Aguirre-Crow

This is great news for the #ALS community. Thanks @SecretaryPete #EndALS #DisabilityRights

No 22 yr old with #ALS should die when a drug could help her live. It’s inhumane for BCBS-TX to deny coverage for @Biogen’s ASO #Qalsody. 1. BCBS-TX is denying coverage alleging “clinical benefit has not been established.” But Shelby’s medical records document her ACTUAL clinical benefit and reduction in bad #ALS biomarkers. 2. Only 2% of people with ALS have Shelby’s SOD1 mutation. Thus, there are less than 500 Americans with ALS today who could be prescribed Qalsody ... even fewer Texans … and even fewer BCBS-TX insureds. Thus, the cost of covering her life-sustaining therapy would be an infinitesimal blip on BCBS-TX’s profits. 3. From ALS symptom onset, the median trach-free lifespan is appx 2.5-3 years. But there are people who have been on Qalsody for over 7 yrs and they are still not trached. Shelby deserves this same chance. 4. @BCBSTX please identify the expertise that your staff MDs and claim reps have to opine about the efficacy and clinical benefit of Qalsody on people with SOD1-ALS? Have they ever diagnosed or treated an ALS patient? Ever conducted a clinical trial? An ALS trial? Are they published in a peer-reviewed journal? Authored any ALS papers? Spoken at ALS conferences? What do they know about ALS genetics? ALS biomarkers? Do they know about the flaws in the COAs? Do they even know what a COA is? Can they explain what NfL is and how it correlates to ALS progression? Have they ever signed a death certificate for a patient with ALS? Now compare their lack of expertise to the ALS and clinical trial expertise of Lead PI & neurologist Dr Timothy Miller of WashU. He testified at the FDA AdComm that he had never seen people with ALS improve in his 20+ yr career in ALS but he does see “remarkable” improvements in his patients in the Tofersen (now Qalsody) trial, OLE and EAP. Has anyone on your team listened to the AdComm testimony? Because if you did, you would have “actual knowledge” that Qalsody works. youtube.com/live/K0k6Jfcc5uM… Dr Miller was the keynote speaker who also presented Qalsody data at the @alsone_official scientific conference. He told the crowd of neurologists that declines in biomarker neurofilament light resulted in a “substantial lowering of the neurodegenerative disease process.” Shelby’s NfL levels decreased substantially on Qalsody. youtu.be/_-KJjwSHWM4?si=PSqO… Now compare your team’s lack of expertise to the ALS and clinical trial expertise of the PIs who authored the peer-reviewed publication in the @NEJM. This is a Who’s Who list of internationally renowned ALS researchers. They know Qalsody works. youtu.be/_-KJjwSHWM4?si=PSqO… Unless your team’s expertise matches that of these neurologists, BCBS should not be supplanting your team's uninformed opinions for these experts’ opinions about efficacy in the trial and the clinical benefit to the patients these neurologists are treating in their ALS clinics. To do otherwise is tantamount to Bad Faith. Please reconsider your position. Your policy language and the actual evidence requires you to give Shelby coverage for Qalsody as long as her neurologist deems it medically necessary. Thank you @statnews for publishing this editorial. #EndALS #neurotwitter #neurology #RareDiseases @JohnCornyn @SenAmyKlobuchar @SenatorWicker @RepTonyGonzales @CEHaddadNP