#ALSFDA: Become Trend on twitter at (2018-07-12 15:34)


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This Tag appearing in: United States: ( Indianapolis - ).
Popular tweets tagged with #ALSFDA:
#ALSFDA ALS Advocacy @alsadvocacyπŸ”.⁦@srfinger⁩ on this panel. It's bound to be good. #alsfda
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I love my wife Tammy #F*ck ALS @MyWifehasALSπŸ”How much does it cost to put on a big meeting in DC?
How much does it cost to do this ?
I rest my case als-advocacy.blogspot.com .

The ALS Association @alsassociationπŸ”Our Chief Scientist Dr. Lucie Bruijn & Board Chair Stephen Winthrop are speaking now at the Community Workshop. Watc twitter.com h live:
The ALS Association @alsassociationπŸ”How are clinical studies designed? Hear insights from researchers & clinicians live from the Community Workshop. Wa twitter.com tch now:
ALS Advocacy @alsadvocacyπŸ”This problem can and should be addressed tomorrow. drive.google.com …
It's not rocket surgery. It will simply take some effort.
twitter.com
ALS Advocacy @alsadvocacyπŸ”How much does it cost to put on a big meeting in DC?
How much does it cost to do this als-advocacy.blogspot.com ?
I rest my case.
#ALSFDA
ALS Advocacy @alsadvocacyπŸ”Sometimes we make it too hard. This isn't s best practice, but it's a better practice. als-advocacy.blogspot.com Somebody just do it pls! twitter.com
ALS Advocacy @alsadvocacyπŸ”@alsassociation @US_FDA Glad he spoke. That format was weird imo. #ALSFDA
Mark Berardi @marksberardiπŸ”Nancy Frates comments to The ALS Workshop held in Washington D.C. on 7/12/2018. Like ALS Advocacy said, cites the b twitter.com urden of stress and its effects on patient health. Trial design 101 from a caregiver.

Jeff Kuhn, MBA @jmkuhn99πŸ”Our Chief Scientist Dr. Lucie Bruijn & Board Chair Stephen Winthrop are speaking now at the Community Workshop. Watch live:
b'🌈 @bjcrochetπŸ”Today's Community Workshop is ending. Thank you for tuning in & submitting comments & questions.
The ALS Association @alsassociationπŸ”Today's Community Workshop is ending. Thank you for tuning in & submitting comments & questions. twitter.com
b'🌈 @bjcrochetπŸ”Dr. Billy Dunn, director of the Division of Neurology Products at , is sharing some final thoughts on today's Community Workshop now.
The ALS Association @alsassociationπŸ”Dr. Billy Dunn, director of the Division of Neurology Products at , is sharing some final thoughts on today's Commun twitter.com ity Workshop now.
ALS Advocacy @alsadvocacyπŸ”I feel like we get some really good input from panelists and then we take 15 step backwards 3 minutes later. We constantly fight collaboration and portray roadblocks as a single warrior fighting an army. Unite with others that have already started the battle.


Cory Burell @No_Quarter_DadπŸ”Every ALSA workshop I end up closing and reopening my computer over and over. It's like looking out the window for s twitter.com now on Xmas Eve if you lived in Key West but actually getting upset that it's not snowing.


Cory Burell @No_Quarter_DadπŸ”I feel like we get some really good input from panelists and then we take 15 step backwards 3 minutes later. We const twitter.com antly fight collaboration and portray roadblocks as a single warrior fighting an army. Unite with others that have already started the battle.


ALS Advocacy @alsadvocacyπŸ”Ok, everyone. How is tomorrow different from Feb 26, 2013? #ALSFDA
ALS Advocacy @alsadvocacyπŸ”.@momfrates cites the burden of stress and its effects on patient health. Trial design 101 from a caregiver. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Caregiver perspective added... they need to consider the family and the caregiver in finding ways to fill trials more twitter.com quickly. Cost for family is significant. Caregiver is essential in trials.
ALS Advocacy @alsadvocacyπŸ”.@srfinger Cites that data from 3 month run-ins are much less informative than average progression data for the patient. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”.@DrOzdinler Asks why not get patients enrolled from day 1 and use each patient's data as his or her own control. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Ferguson on communicating the "why" of trial design is huge to me. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Ferguson - Best practices. What is the burden on patients. How many visits. All should be considered thoughtfully. twitter.com Decisions on trial design should be communicated thoughtfully w patients.
b'🌈 @bjcrochetπŸ”How are clinical studies designed? Hear insights from researchers & clinicians live from the Community Workshop. Watch now:
ALS Advocacy @alsadvocacyπŸ”I'm hearling a lot today that Ph 2 trials aren't mini Ph 3 trials. Ennist speaks to virtual controls... inappropriat twitter.com e and appropriate use.
BbLlUuEe @Blueman17XTπŸ”Ennist on promoting use of large data sets - Because of old ways of doing trials, have efficacious drugs failed the trial designs? - Need to leverage more data more.
ALS Advocacy @alsadvocacyπŸ”Ennist on promoting use of large data sets - Because of old ways of doing trials, have efficacious drugs failed the t twitter.com rial designs? - Need to leverage more data more.
ALS Advocacy @alsadvocacyπŸ”Cudkowicz - Get rid of survival as the primary measure in ALS trials. She thinks guidance should take survival off the table. #ALSFDA
Sandy Morris @SandyMo77787733πŸ”. Has been a force at ... Dr Andrews clearly got the benefit of her interaction. And we've heard questions from her that should make us all cringe because the answers are poor.
Sandy Morris @SandyMo77787733πŸ”So far today, the home run Q came from ... Why not just replay the 2013 hearing. And she didn't have ALS in 2013. We are going over such familiar comments today, five years later. What will change???
ALS Advocacy @alsadvocacyπŸ”@momfrates @srfinger He applied boot to some hinders. I hope some here realized that. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Cudkowicz - Access to therapy problem. Her answer is not broad inclusion in a trial. It may be a companion study tha twitter.com t will contribute to safety data. Also open label. Also EAPs.
ALS Advocacy @alsadvocacyπŸ”Somebody record ... He's giving us lessons on stratification. He's giving us lessons on leveraging data we have no twitter.com w. Oh, I just heard Bayesian Dynamic Updating, too.
ALS Advocacy @alsadvocacyπŸ”. There is informative value in open label extension and in crossover trials, too. You can encourage patients and en twitter.com hance robustness. Guidance should encourage that.
ALS Advocacy @alsadvocacyπŸ”.@srfinger Placebo does not guarantee success. He now speaks to the use of placebo arm in safety trials. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”. The problem of shoulders shrugged by investigators when they encounter overachieving placebo groups is the antithes twitter.com is of PFDD.
ALS Advocacy @alsadvocacyπŸ”. Has been a force at ... Dr Andrews clearly got the benefit of her interaction. And we've heard questions from he twitter.com r that should make us all cringe because the answers are poor.
ALS Advocacy @alsadvocacyπŸ”Andrews - Speaks enthusiastically about the people w ALS she has heard from who are not her patients who reached out twitter.com to her as a result of this panel. AND she heard about the differences between cancer trial designs and ALS trial designs. Nice.
ALS Advocacy @alsadvocacyπŸ”Andrews - Trial inclusion should be as broad as possible. #ALSFDA
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ALS Advocacy @alsadvocacyπŸ”Andrews - Guidance has to be structured in a way that keeps in mind that things will change. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”I'm trying to keep an open mind about but the discussion constantly returns to lack of funding or "can not". The purpose of this should be converting the status quo not using it as crutch for impossibility.
Haepehstin @haephestinπŸ”I said this five years ago and I say it today.... every ALS trial is an efficacy trial regardless of phase. Coffey points out the underpowering of Ph 1,2 w references to efficacy.
Cory Burell @No_Quarter_DadπŸ”I'm trying to keep an open mind about but the discussion constantly returns to lack of funding or "can not". The pur twitter.com pose of this should be converting the status quo not using it as crutch for impossibility.
ALS Advocacy @alsadvocacyπŸ”I still don't like a format where we are talking at the FDA and not with the FDA! #ALSFDA
ALS Advocacy @alsadvocacyπŸ”I said this five years ago and I say it today.... every ALS trial is an efficacy trial regardless of phase. Coffey p twitter.com oints out the underpowering of Ph 1,2 w references to efficacy.
ALS Advocacy @alsadvocacyπŸ”Finger brings up target engagement endpoints in Ph 1 or 2 trials. How do we validate that THEY are working and valid. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Mitsumoto bringing up the concept of a Goal Attainment Scale in clinical trials. Franson chimes in as such things b twitter.com eing important for the use of Accelerated Approval. The challenge is how to validate.
ALS Advocacy @alsadvocacyπŸ”#alsfda "Interrogating" is probably not the best word for asking patients questions. Gee!
Wendy Selig @wendyseligπŸ”Key challenge in : figuring out how to incorporate patients' feelings when they take a medication even when this is s twitter.com ubjective.
ALS Advocacy @alsadvocacyπŸ”Sherman distinguishes natl history studies from historical controls. Different concept to provide formal data. #alsfda
ALS Advocacy @alsadvocacyπŸ”Sherman - Asks about natural history studies that could be looked upon w favor by FDA. He proposes the long-standin twitter.com g goal of getting natural history started for every clinic patient from day 1.
ALS Advocacy @alsadvocacyπŸ”Hebron challenges all Ph 2 trials, the effect of randomization, the hetergeneity of the disease, and the lack of guid twitter.com ance in any of the drafts on how to handle the basic statistical challenge of Ph 2 ALS trials.
ALS Advocacy @alsadvocacyπŸ”Again a woman w ALS speaks to the cruelty of the extended observation periods in ALS trials. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”.@DrOzdinler "We fail to learn from our failures." #ALSFDA
ALS Advocacy @alsadvocacyπŸ”.@DrOzdinler Speaks to responder groups in Ph 3 trials who, if given continuing access to drug, could teach us a lot. #dingdingding #alsfda
Wendy Selig @wendyseligπŸ”Profound, articulate and inspirational comments from patients and caregivers at workshop on therapy development. S twitter.com o much unmet need!
ITF Pharma, Inc. @ITF_PharmaπŸ”. 's Dr. Jeremy Shefner, co-founder, is leading a discussion on measuring drug development outcomes at the ALS Community Workshop. Join live:
ALS Advocacy @alsadvocacyπŸ”Are we just resetting the clock for another five years? #ALSFDA twitter.com
Andrew Frates @AndrewFrates_πŸ”.@momfrates holding people accountable 5 years later! #ALSFDA twitter.com
ALS Advocacy @alsadvocacyπŸ”Kennedy - Woman w ALS brings up the Type 2 error! #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Hersch suggests that people interested in ALS contribute comments to the rare disease guidance during its comments p twitter.com eriod which is open now.
ALS Advocacy @alsadvocacyπŸ”Hersch makes reference to announcement of draft guidances. He cites that rare disease guidance has items that are v twitter.com ery applicable to ALS (e.g. historical controls).
ALS Advocacy @alsadvocacyπŸ”Hersch from Voyager speaking to unique challenge of gene therapy trials that were not addressed in draft guidance. twitter.com Small trials, duration of effect, test dosing for a one-treatment therapy, sham placebo procedure concerns, participants not elig for oth trials.
b'🌈 @bjcrochetπŸ”Expert MDs from , & are joining ' chief medical officer to discuss patient experience & benefit-risk balance at the Community Workshop. Watch now:
ALS Advocacy @alsadvocacyπŸ”Impt topic... Shefner speaking to endpoints that FDA will accept for Accelerated Approval. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”So far today, the home run Q came from ... Why not just replay the 2013 hearing. And she didn't have ALS in 2013. twitter.com We are going over such familiar comments today, five years later. What will change???
ALS Advocacy @alsadvocacyπŸ”Shefner talks of the risk (incl community risk) of drugs being approved that don't work. I hope he speaks of the ris twitter.com ks of a drug that does work being rejected..
ALS Advocacy @alsadvocacyπŸ”Note - Community Guidance is the document formerly known as Airlie House Guidelines. Potato, potahto. #ALSFDA
The ALS Association @alsassociationπŸ”. 's Dr. Jeremy Shefner, co-founder, is leading a discussion on measuring drug development outcomes at the ALS Comm twitter.com unity Workshop. Join live:
ALS Advocacy @alsadvocacyπŸ”For heaven's sake, don't set expectations for long clinic visits with patients and caregivers, shorten the %^&*( visits! #ALSFDA
ALS Advocacy @alsadvocacyπŸ”They tried to tapdance away from the cannabis Q, but a scientist brought it back w specific Qs related to FDA. It t twitter.com ruly is important for people with ALS.
ALS Advocacy @alsadvocacyπŸ”Brooks bringing up delay to dx... need to dx promptly to get people into trials more quickly. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Yes, . Clinical trials are a legitimate therapy option for people with ALS. I personally think it's even more than twitter.com hope. It's something tangible. Maybe it will work. Maybe not, but maybe it will.
ALS Advocacy @alsadvocacyπŸ”.@momfrates Important point bringing up the subjectivity of the clinical measures done during those marathon 8 hour trial visits. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”.@momfrates brings up the deja vu to 2013 hearing... #ALSFDA The lack of action from 2013 is stunning... we spin our wheels.
ALS Advocacy @alsadvocacyπŸ”Woman w ALS suggests we just replay the 2013 FDA hearing. That should give us all pause. Are what we doing here rea twitter.com lly going to result in action?
ALS Advocacy @alsadvocacyπŸ”#alsfda Standard of care issues come up from a caregiver... riluzole and radicava.
ALS Advocacy @alsadvocacyπŸ”#alsfda The importance of caregiver perspective.... ! Caregiver speaks to the onerous visits and time spent "under the hood."
ALS Advocacy @alsadvocacyπŸ”There's that "expected" word and the problem of the overachieving placebo group. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Wolff from @Cytokinetics is trying to address the placebo issue. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Wolff - Cytokinetics in trial is tracking vital capacity at home and in clinic and the results track well. #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Here you go... Stephen Winthrop's kind reference... drive.google.com #ALSFDA
ALS Advocacy @alsadvocacyπŸ”Oskarsson is pleased w what he sees so far in guidance document? #ALSFDA
Andrea Pauls Backman @AndreaBackman05πŸ”Pleased to be with 100 researchers, physicians, leaders, representatives and many people living with ALS, convening twitter.com at the workshop today in Washington, DC. We need to move the needle forward on clinical trials.

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