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Michael Robinson out there doing awesome work for ALS. ❤️
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#PaCTD
Michael Robinson...
BIO...
Open label extension is basic in a clinical trial. We need to make sure we are insisting on humane trials to advance ALS science. #WeAreNotZebrafish #PaCTD #WhatWeAllowWillContinue
Every trial candidate should know to ask, "What happens to me if this stuff works for me?"
The right answer is for the sponsor to say in writing that you will be able to continue on the therapy if you want.
If you don't get the right answer, find another trial.
Thank you @Cytokinetics! We so appreciate your partnership of creating trials that humans want to join. People living with ALS want to be a part of advancing our science. Thank you for making that easier for us. #PaCTD @iamalsorg
There are now 3 clinical trials that have a ⭐️⭐️⭐️⭐️⭐️ rating! The ALS community created and celebrates patient centric trial design. @Cytokinetics Reldesemtiv incorporated humane and efficient trial design from the start. Check it out here:
https://t.co/QrLE5wF71V
COURAGE-ALS earned a 5-star #PaCTD rating! This will be up on ALS Signal Dashboard very soon.
cc & thx: @nadia_sethi @cadawno @iamalsorg @sandymorris333
It is the best thing we have going in this crazy disease! @MGHNeurology If you can join the Sean Healey Platform Trial, do so! If you can support it, do so! The fastest way to get investigational therapies into our bodies is clinical trials. This one is 5-star! #PaCTD
Please join @PaganoniMDPhD and special guest Lauren Elman, MD from @PennNeurology @PennMedicine this Thursday at 5:00 pm Eastern for updates and audience questions on the HEALEY ALS Platform Trial. https://t.co/fTEUHQmyEd
#HealeyHope
@kelsieswrites you impress me everyday. Your honest & clear words coupled w/ your bravery make me so proud to know you. EAPs need to be planned for in the clinical trial design stage. And do not use placebo participants as your excuse as frankly it's just not true. #PaCTD
Lisa is dying of the same kind of aggressive-progressing ALS Chris has. She wants to get the @biogen drug Chris is on, but the trial is done enrolling patients. She’s asked for compassionate use of the drug, called Tofersen. Biogen said no. Please read this thread. 1/11
I'm definitely not okay with this. We've partnered with 25 drug sponsors so EAPs are included in clinical trials. This is not a novel request. ALS always has to fight to merely catch up - why is that? Don't let Lisa die without trying for her. Don't let us die w/o a chance.#PaCTD
There is a therapy that could help. But, she isn’t allowed to have it. Instead, her kids and husband have to watch her die a brutal death. Lisa knows and has to watch too. You okay with this America? I’m not. Help get treatments into bodies at @iamalsorg
Thank you @Biospace and Heather McKenzie for sharing Lisa's story and fight for #tofersen. @Biogen @US_FDA @FDAMedia #tofersenforlisa #EndALS #BiogenGrantAccess #dyingwaiting @iamals @sandymorris333 https://t.co/LrgyKzRljr #jobs
Exciting news! @ApellisPharma has a 4-star PaCTD rating for their trial showing that they care about ALS families. If interested in advancing ALS science, please go check it out. #PaCTD
New PaCTD (Patient-Centric Trial Design) Rating!!
@ApellisPharma received a 4-star rating for their phase 2 trial testing pegcetacoplan in patients with sporadic ALS.
Read more about the trial and rating here: https://t.co/T0xhzFKB9F
And time is money for therapy developers.
Spend a few $ to make a humane trial that gets a five-star #Pactd rating, and you will save gazillions by filling trials faster, completing trials faster, and getting to market faster.
https://t.co/bA6JA8rwYn Look what can happen when we implement w/ humanity & efficiency! I was told that ALS trials were difficult to fill as patients weren't that interested, I found this baffling as ALS is 100% fatal. Turns out that wasn't true at all. #PaCTD #ActionNotWords
Thank you @US_FDA and @DukeMargolis for including me and @pjgreen as the voice of the patient. It was a lot of valuable discussion from some of the brightest people I’ve ever met. I’m all in for turning those great words into action. #NotSoPatientPatient #PaCTD
Thanks to @DukeMargolis for hosting a productive 2-day workshop on #ALS research and drug development. Gaining momentum working together with patients, academia, industry and federal partners.
Day 2 #ALSMNDSYMP Attendees! Live Poster Session B: Patient-Centric Trial Design Rating System (#PaCTD)
2)PaCTD rating: The Patient Centric Trial Design rating evaluates trial design based on nine criteria. Scores based on these criteria are translated into a maximum of five stars. This rating is not reflective of the safety or efficacy of the treatments in the trials.
The new path forward - humane and efficient clinical trials. #PaCTD
Some ppl w ALS moving the system in a better direction...
Working w sponsors to fill trials that
a. are humane &
b. offer OLE (for those in the trial) &
c. offer EAPs (for those who can't get in trial).
This is important.This is effective.This will speed up access & approvals.
2)PaCTD rating: The Patient Centric Trial Design rating evaluates trial design based on nine criteria. Scores based on these criteria are translated into a maximum of five stars. This rating is not reflective of the safety or efficacy of the treatments in the trials.
Humane trials will fill faster.
Every day saved filling a trial is as important as a day in a lab or a day of FDA review.
Every day.
Thanks to those with ALS and caregivers who found a tangible way to save days. #PaCTD ratings will matter!
#PaCTD ratings will mean more humane trials while advancing the science for the ALS community. #EndALS @iamalsorg
#PaCTD ratings will mean more humane trials while advancing the science for the ALS community. #EndALS @iamalsorg
Participating in a clinical trial is a decision we know people living with ALS do not take lightly so a team of people most impacted by ALS created a rating system to help you see if a trial has a patient-centric approach.
Every. Single. Day. They Lead.
@bsw5020 @CWOB_Ancient And with that, I would like to get back to work in making EAPs a reality for future ALS generations. Truly it is up to us to stop the madness. Please join us in the fight of our lives. @iamalsorg #PatientCentricTrialDesign #PaCTD
Please read the thread. Please understand the difference between being a clinical trial participant and being a part of an EAP. Your awareness and education will help move us in the right direction, with urgency. #PatientCentricTrialDesign #PACTD
In case you missed this last night, if you are at all involved in the fight against ALS please take 5 minutes to read👇release today. Everyone. Including you @alsassociation. The more you know about EAPs, the more you understand their importance in this fight.
@Shankapotomas @kimbrolyclaire Exactly! Yes it could! It’s our turn. #PatientCentricTrialDesign #PACTD
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