ALS ACCESS

Heartbroken to share the tragic news that @ScottsFight with #ALS ended tonight. Please pray for his family. Please honor Scott's legacy by remembering his fight. Scott received #NurOwn in the P3 trial in 2020. Years before the trial was unblinded, Scott repeatedly told the world that #NurOwn halted his progression & helped him regain some function: "My ALSFRS scores stabilized, my breathing jumped 10 pts & my walking stengthened to the point where I was constantly forgetting my walking cane." ~ Scott's ALS Uncensored Podcast Nov 2022 buzzsprout.com/2034679/episo… @MartyMakary Scott #DiedWaiting. Our inhumane regulatory system ignored the opinions of people who know best -- the people who received NurOwn & the renowned neurologists who watched it work on their patients. The paternalism & hubris at the FDA allowed Scott to die. When people are dying, they know when a drug helps them live. Believe them. #NurOwnWorks Our regulatory system doesn't. #EndALS #artist #artwork #DisabilityTwitter @SenatorLankford @SenMullin @TomColeOK04 @tulsaworld @TheOklahoman_ @GOPHELP @SenBillCassidy @RogerMarshallMD @RandPaul @lisamurkowski @SenatorCollins

If only @FDACBER had listened to the opinion of world renown neurologist & stem cell expert from @MayoClinicNeuro who had his patients' best interests in mind when he testified at the AdComm that people on #NurOwn had stabilized in a way he had never seen in any other #ALS trial in his 40+ year career. @MartyMakary the #ALS community needs your help. People are #DyingWaiting. @SenAmyKlobuchar @SenTinaSmith @RepAngieCraig #EndALS

As the President-elect is choosing his Cabinet Secetary for HHS, what cannot get lost in the mix is why people with terminal rare diseases desperately need DISRUPTIVE policy change at HHS. We need the new HHS Secretary, a new FDA Commissioner, the White House and all Republicans & Democrats in Congress to help correct these FDA's failures: 1. Disregarding the opinions of world renown rare disease experts/ clinical trial investigators, biomarker experts who testified in Advisory Committee meetings that drugs worked on their dying patients -- especially egregious when the FDA has no similar expertise in the rare disease. This has recently happened in #Duchenne, #ALS & #amyloidosis with experts from Stanford, Mayo, MassGen, Vanderbilt, Boston U, Nationwide Childrens & Cleveland Clinic to name just a few. 2. Disregarding the opinions of rare disease treating physicians who opined that a drug helped their patients live and have a better quality of life. One example: regulations.gov/comment/FDA-… 3. Ignoring the testimony, videos & medical records from patients in the trials and expanded access who received therapies and experienced how a drug improved how they "felt, functioned & survived." When patients are dying they know when a drug helps them live. Believe them. youtu.be/vrr3uB1TP4o?si=XP5o… 4. Opposing a Conditional Approval pathway like Senators' Braun & Gillibrand's Promising Pathway 2.0 bill. Dying Americans are traveling around the world & spending hundreds of thousands dollars, to take unproven & possibly harmful scam therapies rather than getting therapies in the US & collecting that data to further the science. This bill would accomplish that. braun.senate.gov/news/press-… 5. Failing to mandate adequate representation of people of color in clinical trials as advocated by @RepLBR @RepRobinKelly @RepBarragan and @RepYvetteClarke on @EnergyCommerce. 6. Failing to follow both the "plain language" & intent of the 21st Century Cures Act sponsored by Democrat @RepDianaDeGette & passed in 2016. 7. Failing to recognize #RealWorldEvidence and Patient Reported Outcomes that weren't prespecified despite efforts by Rare Disease Caucus chairs like Democrats @DorisMatsui, @SenAmyKlobuchar & GOP @SenatorWicker. The Duchenne community & Rep Matsui introduced the Benefit Act because the FDA is ignoring RWE & RWD. parentprojectmd.org/advocacy… 8. Failing to consider post hoc evidence as "supporting evidence" of a drug's efficacy that has valid confirmation in other aspects of the clinical trial -- despite Dr Pazdur's Oncology Center of Excellence considering similar data. In his 32-page Public Comment, Dr. Ardalan Minokadeh gave examples of divisions who had properly used Post Hoc evidence. regulations.gov/comment/FDA-… 9. Failing to consider how hetereogeneity effects trial endpoints & p-values then denying efficacy when a drug may work on some but not all. When people are dying, #SomeisEnough. 10. Failing to give people with terminal rare diseases a statutory right to request an Advisory Committee meeting. prnewswire.com/news-releases… 11. Refusing people dying of Rare Diseases their Constitutional Due Process rights to testify in person at AdComms. 12. Depriving patients at Advisory Committee meetings an adequate opportunity to present the first-hand CLINICAL EVIDENCE from taking the therapy, then failing to have any dialogue about that evidence. Some parents with boys dying of Duchenne were denied a chance to speak. Even for those given a chance to share their evidence, three minutes is not ample time to share seven years of evidence in the fight for your life or your child's life. 13. Failing to mandate adequate representation of people of color in clinical trials as advocated by @RepLBR @RepRobinKelly @RepBarragan and @RepYvetteClarke on @EnergyCommerce. 14. Failing to ensure its "Fast track" progam works when many drugs still aren't approved a decade later despite evidence of efficacy for YEARS. 15. FDA CBER's failure to use RMAT progam to expedite biologic development and approval. 16. Failing to take into account how flawed, subjective "clinical outcome assessments" are impacting trial endpoints & accurate conclusions in assessing efficacy. 17. Applying different approval standards, AdComm Questions & Instructions among different FDA divisions such as CBER, CDER & Oncology Center of Excellence. 18. Taking years to draft & implement 10 pg Guidance Documents. 19. Failing to follow statutes & Guidance about exercising "Regulatory Flexibility" in terminal diseases with critical unmet needs. 20. Failing to act with urgency & triage the underfunded terminal rare, diseases that are killing people. 21. Failing to consider the inhumane risk of Type II errors in heterogeneous terminal diseases. This must be factored into the statutory risk-benefit decision. 22. Failing to exercise flexibility to get drugs in bodies of #Veterans who are dying of diseases they get from their service to our country. militarytimes.com/opinion/co… The patient community has tried all mechanisms to change this broken regulatory system for the last decade. We've had 1000s of Congressional calls, meetings, and worked with the #RareDisease Caucus. Patients met with the White House, Domestic Policy Counsel, and FDA Commissioners & Directors under Presidents Obama, Trump & Biden. We passed $500M legislation. People wth ALS and leading researchers spoke at multiple Congressional hearings discussing the need for a new approach to terminal rare diseases. youtu.be/mjDwdMpraxY?si=4yNO… Patients wrote OpEds. ALS widow @nicolecimbura wrote an OpEd in the @WSJ about how a slow FDA was delaying and denying access to drugs. wsj.com/articles/a-slow-fda-… Physician and pediatric oncology reseacher Bill Woods wrote how the FDA applied different standards in ALS compared to Oncology: "As a pediatric cancer researcher, I admired the FDA. Then I got ALS." statnews.com/2022/08/16/fda-… People battling ALS engaged with the media across the country and NBC affiliate WLWT did multiple stories with anchor @ShereeWLWT. wlwt.com/article/cincinnati-… We have top Democratic allies who advocated for change including Brian Wallach & Sandra Abrevaya who worked for Barack Obama; health care advocate Ady Barkin, and Dan Tate one of the country's top lobbyists. Ady died of ALS. Dan, Brian & 32k others have ALS and are #DyingWaiting for the FDA. Dying patients like Brian Wallach made documentaries such as the Katie Couric produced No Ordinary Campaign in 2023. amazon.com/Love-Life-No-Ordi… and Angelina Fanous made #DieTrying on HBO Vice in 2017. youtu.be/arDpeOm2s_4?si=nyrZ… We participated in protests, Town Halls, AdComms, PFDDs, public meetings, patient advisory boards & industry meetings. We sent private emails to FDA Commissioners & Directors. We spoke at industry conferences on panels with FDA officials. We started non-profits & Facebook groups, paid for billboards and made our own press releases with pleas for our lives. prnewswire.com/news-releases… No one has listened. I've cried too many tears about family & friends who died when drugs could have helped them live. I can't speak for any other Democrats but I will work with anyone of any party who will help us in the fight for our loved ones' lives. Congress will you fix this problem and make it part of the questions at any Senate Confimation hearing of the HHS Secetary nominee? @GOPHELP @HELPCmteDems @johnthune @SenBillCassidy @SenJohnBarrasso @RogerMarshallMD @lisamurkowski @SenatorCollins @MarkwayneMullin @SenTedBuddNC @SenSanders @ChrisMurphyCT @SenatorHick @SenMarkey @SenTinaSmith @SenatorHassan @SenatorBaldwin @ChrisCoons @DickDurbin @SenSchumer @RepSchakowsky @RepPaulTonko @RepAngieCraig @RepFletcher @RepMikeQuigley @RepAndyHarrisMD @RepGuthrie @RepGregMurphy @RepMarkGreen @RepJohnCurtis @RepJohnJoyce @RepGusBilirakis @HouseCommerce