Members of ALS Community Gather with Representatives from FDA and Industry to Inform FDA Draft Guidance on ALS

The ALS community recently presented its recommendations to the U.S. Food and Drug Administration (FDA) regarding the Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry at a day-long event, called ALS Community Workshop: Therapy Development and Regulatory Pathways, which was held in Washington, D.C., on July 12. Over 90 people attended in person, with many more tuning in online.

Throughout the day, there were opportunities to make comments to the FDA and industry representatives, both in-person and online. The Association was proud to host the day to bring many ALS stakeholders together to provide targeted feedback and information to further inform the FDA’s Draft Guidance on ALS Drug Development.

The FDA’s Draft Guidance provides drug developers with recommendations for all aspects of clinical trials for ALS, from study design to risk-benefit considerations to patient selection. For many years, The ALS Association has worked closely with the FDA and members of the ALS community in the development of a comprehensive ALS Guidance to make clinical trials faster, shorter, and more responsive to special considerations inherent to people with ALS and their caregivers.

The Workshop was an opportunity to provide targeted feedback and information from the ALS community to further inform the FDA’s Draft Guidance on ALS Drug Development. After the Workshop, the Association composed a report to the FDA to address key points discussed at the Workshop and to make recommendations on how to improve the FDA’s final Guidance (listed below).

“The purpose today is to build a strong guidance together,” said Calaneet Balas, president and CEO of The ALS Association. “We will be taking the great ideas from this meeting and presenting them to the FDA for their consideration in developing the final Guidance document. We look forward to that document, and to seeing it used widely for making clinical trials faster, more effective, and more aligned with the needs of patients and caregivers. We believe that is the route to the fastest development of new treatments for ALS.”

Continue reading Members of ALS Community Gather with Representatives from FDA and Industry to Inform FDA Draft Guidance on ALS

Guest Post: We Don’t Have Five Months to Wait

By Mary Johnson, Caregiver – Western Pennsylvania

Note: Under current law, people disabled with ALS who qualify for Social Security Disability Insurance (SSDI) must wait five months before receiving SSDI benefits. Every person must wait, regardless of the level of disability or how fast the Social Security Administration (SSA) approves their claim.

The ALS Disability Insurance Access Act (S.379/H.R.1171) would eliminate that five-month waiting period for people with ALS to receive SSDI. People with ALS would receive their SSDI benefits immediately after being approved by the SSA.

The five-month waiting period for Social Security Disability Insurance (SSDI) after an ALS diagnosis has severely impacted my family and I’m mad as hell about it. There’s absolutely no reason people with ALS shouldn’t be getting both SSDI and Medicare benefits immediately.

I can tell you from my own experience – we don’t have five months to wait.

My family has the genetic form of ALS (familial ALS). As of June 2018, I’ve lost 14 family members — siblings, aunts and uncles, nieces and nephews, and cousins – to this horrific disease.

My 25-year-old niece, Amanda, died four months after her ALS diagnosis – before the SSDI waiting period was met. The same situation occurred for my niece, Shannon, who died at age 34. She was diagnosed in January 2013 and died just two months later.

Continue reading Guest Post: We Don’t Have Five Months to Wait

With ALS, Every Day Adds Up

Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, research project funded, story shared, discovery made, piece of legislation passed, and auction bid placed – it all adds up!

For people living with ALS and their caregivers and family, every day adds up.

ALS is a journey that begins months, even years, before a diagnosis. Because there’s no definitive test for ALS, doctors must run through a battery of tests, ruling out other potential syndromes, conditions, and diseases before making a diagnosis of ALS.

It’s a journey that, for many people diagnosed with ALS, continues through the five-month waiting period they qualify for Social Security Disability Insurance.

Continue reading With ALS, Every Day Adds Up

Support for ALS Disability Insurance Access Act Grows Following Advocacy Conference

We need your help! Now is the time to urge your representatives and senators to waive the five-month waiting period for Social Security Disability Insurance (SSDI) benefits for people living with ALS.

Under current law, people disabled with ALS who qualify for SSDI must wait five months before receiving SSDI benefits. Every patient must wait regardless of the level of disability or how fast the Social Security Administration (SSA) approves their claim.

The ALS Disability Insurance Access Act (S.379/H.R.1171) would eliminate that five-month waiting period for people with ALS to receive SSDI. People with ALS would receive their SSDI benefits immediately after being approved by the SSA.

Continue reading Support for ALS Disability Insurance Access Act Grows Following Advocacy Conference

ALS Community Workshop: How to Attend the Live Stream, Ask Questions, and Make Comments

On Thursday, July 12, the ALS community will have the opportunity to meet with representatives from the FDA and the pharmaceutical industry during an ALS Community Workshop in order to discuss the latest FDA guidance document related to ALS drug development.

The Workshop will be streamed live on our ALS Community Workshop webpage, Facebook page, and YouTube channel from 9:00 a.m. EDT to 3:00 p.m. EDT.

Both in-person and online attendees will be able to ask questions and comment. Continue reading for instructions on submitting questions and comments that may be addressed during the Workshop.

If you plan on attending the (online) live stream event, please register here: https://thealsassociation.formstack.com/forms/signup.

Continue reading ALS Community Workshop: How to Attend the Live Stream, Ask Questions, and Make Comments

Join Us at ALS Community Workshop: Developing Drugs for Treatment, Guidance for Industry – July 12

The ALS Association is hosting ALS Community Workshop: Developing Drugs for Treatment, Guidance for Industry on July 12 in Washington, D.C., and the entire ALS community is invited.

The goal of the workshop will be to provide targeted feedback and information to the Food and Drug Administration (FDA) from people with ALS, caregivers, and stakeholders to inform revisions of the FDA’s Draft Guidance on ALS Drug Development.

Continue reading Join Us at ALS Community Workshop: Developing Drugs for Treatment, Guidance for Industry – July 12

Guest Post: ‘ALS is What I Have, Not Who I Am’

By Alan Alderman

Note: In recognition of ALS Awareness Month, we’ve been sharing a lot of new stories about people living with the disease and their caregivers, the volunteers and health care providers who help make the lives of people with ALS better, the powerful fundraisers and the participants in Walk to Defeat ALS and Team Challenge ALS events, and the researchers fighting for a cure. This is the thirteenth profile in that series.

Bad things happen to good people every day. That’s just the nature of life. But it doesn’t mean we stop living and loving.

My ALS story officially started on a warm fall afternoon in September 2001. I had been having difficulties with speaking and swallowing for several months. After more than two months of seeing about a dozen different medical professionals, and having more tests than one can imagine, I sat in the small examination room of a local neurologist.

The doctor had just finished an EMG and asked if he could be excused for a minute. What was I to say? No?

He stepped out of the room. I sat alone on the examination table for what seemed like an eternity but was probably only a few minutes.

When he returned, he sat on the small rolling stool and rolled in front of me. He looked me in the eyes, placed his hands on my knees, and said, “Alan, I believe you have ALS.”

Continue reading Guest Post: ‘ALS is What I Have, Not Who I Am’