ALS Association to Host FDA, Industry in “We Can’t Wait” Action Meeting

Research

Later this month, the ALS Association will host a virtual session focused on the urgent need for timely access to new therapies. Leaders from the U.S. Food and Drug Administration (FDA) and industry companies will listen to people living with ALS speak directly about their expectations for their experiences with the disease and how important it is to have timely access to therapies that have the potential to provide incremental benefit in improving how people feel, function and live.

This event takes place at a critical time for the ALS community as we continue engage regulators as they review potential new therapies and companies as they decide if and when to provide expanded access to drugs in development. We continue to urge the FDA to exercise its regulatory authority as outlined in its final 2019 ALS Guidance to grant the earliest and widest possible access to drugs that have met their endpoints in well-designed clinical trials. We also continue to urge companies with drugs that have shown benefit in development to provide expanded access at the earliest possible time.

This month’s meeting, while taking place in that context, is about much more than a single company, drug or development program. This meeting is about ensuring that FDA reviewers and leaders understand how our community evaluates and values the potential for incremental benefit – extra months of life, slowed disease progression that could mean extra months of being able to talk, or walk, or even breathe on one’s own. These seemingly modest benefits are real to people living with ALS. They add value and quality to life – and when combined with potential incremental benefit from the next novel agents to demonstrate positive results in a well-designed trial – could lead to significant impact on the trajectory of this disease. 

We have invited multiple people living with ALS to speak during the session, and have asked several ALS organizations to invite additional speakers. We would like to provide multiple perspectives about the need for timely access to potential treatments. To ensure that a broader group of people can be heard from by the FDA, the Association invites all member of our community to submit their perspectives in writing and by video at this link. These submissions will be compiled and provided to the FDA and company officials, and may be shared with the public.

Officials from the FDA, as well as the companies, will be in “listening mode” during the meeting. The decision not to livestream the event was made by the FDA, but we have requested that the meeting be recorded and shared with the community so everyone can see and hear the discussion. We will share the recording and details following the meeting.

Comments

Submitted by: Marjie S. on Thu, 05/13/2021

We would love to be able to speak to the FDA

Submitted by: Jayne L. on Sun, 05/16/2021

I tired of begging. You are robbing my husband of his right to EXIST. Our granddaughters do not comprehend that their Papi is not long for this life. Almighty dollar...

Submitted by: Sandra M. on Thu, 05/13/2021

Please allow patients with ALS to access promising treatments. We are willing to take risk. The disease we fight is 100% fatal. I was diagnosed with Bulbar ALS one year ago. A competitive athlete for for decades, now the simplest task leaves me breathless. I require a ventilator to sleep at night. I eat through a feeding tube. My voice is almost gone. Please do not fail us by blocking access to ALS treatments that may help our symptoms or extend our lives. Thank you.

Submitted by: Cassandra H. on Fri, 05/14/2021

This must be open to the public and should be live-streamed to accommodate ALS patients.

Submitted by: Amy L. on Tue, 05/18/2021

Because of stipulations imposed by FDA, the meeting is limited to 60 minutes and cannot be live streamed. There will be speakers living with ALS nominated by Team Gleason, I AM ALS and MDA along with other leaders with ALS. The meeting will be recorded and shared with the community as quickly as possible.

Submitted by: Barry T. on Fri, 05/14/2021

I diagnosed with ALS on November 19th 2020. I have gone downhill in the past 6 months to the point I can no longer DRIVE, I can barely WALK, and I can barely speak. I have bulbar ALS and upper body neuron damage. I cannot wait for you guys to discuss what drug is effective and what drug is not. I should have compassionate release of any medicine that my doctor at U of M Medical neurology says I can take. I shouldn't have to DIE while waiting on you to make a decision. Please help me!!!

Submitted by: Barry T. on Fri, 05/14/2021

I was diagnosed with Bulbar ALS in November 2020. Anyone can get this disease. In 6 months, I can no longer drive, dress myself, lift my hands, cut my food, or walk. I shouldn't have to die because the FDA will not release, compassionately, drugs that could be used to arrest the disease progression or cure the disease. I am going to die because you do not care care about me. You only care about insurance money. WE NEED HELP NOW!!

Submitted by: Erin R. on Fri, 05/14/2021

My mom was diagnosed with Bulbar ALS 17months ago. She requires oxygen and she has to have a ventilator to sleep or even lay down. She eats through a feeding tube. She’s lost her ability to speak. I haven’t heard her voice in over a year. She uses a text to speech app on her tablet to communicate. Her hands have started contracting, before long she won’t even be able to type. Those who have ALS deserve more than this, she deserves more than this. For a chance at a few more months with their loved ones, they are willing to take the risks. Please don’t deny them a chance for more.

Submitted by: Amy L. on Tue, 05/18/2021

Hi Erin. My sympathies to you and your mom. Please share your thoughts with the FDA: https://bit.ly/FDAALS

Submitted by: Nicole C. on Sun, 05/16/2021

Please allow me to be part of this

Submitted by: Amy L. on Tue, 05/18/2021

FDA accepted The ALS Association’s invitation to listen to people with ALS on the issue of drug approvals. Its conditions for participation include holding the meeting to 60 minutes, which limits the number of live testimonials. We reached out before our public announcement to Team Gleason, I AM ALS and MDA to nominate speakers. However, we are gathering testimonials from others and will be sharing them with the FDA following the meeting. You can submit yours here: https://bit.ly/FDAALS

Submitted by: Justin P. on Fri, 05/21/2021

I was diagnosed with ALS in 2019 at the age of 36. I am no longer able to dress my self or give my self showers I am loosing my upper body movements more and more every day I have kids that ask me every day why I can’t play with them anymore and I can’t help them with anything it hurts me and I don’t understand how the government can approve other drugs for emergency use and spend billions of dollars on finding treatment or cures for any disease that threatens them but can’t help us that have ALS with more funding to find better treatments or cure and I don’t understand when there is a treatment that could help me slow down this disease and give me a little bit longer to live and see my kids grow up they want us to damn near have to beg them to approve it guess they don’t understand how it feels knowing that this disease is a death sentence and I am willing to try anything to help me live longer even if there are risks

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