Accelerating Access to Critical Therapies for ALS Act – ACT for ALS - Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS), Docket for Public Comment
What is the Accelerating Access to Critical Therapies for ALS Act, Action Plan for Rare Neurodegenerative Diseases?
On September 14, 2022, the FDA and NIH announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases.
FDA Action Plan for Rare Neurodegenerative Diseases
As part of its requirements under the ACT for ALS, the FDA has developed a five-year plan describing actions the FDA intends to take to:
1. Foster the development of safe and effective drugs that improve or extend or both, the lives of people living with ALS and other rare neurodegenerative diseases; and
2. Facilitate access to investigational drugs for ALS and other rare neurodegenerative diseases.
Your Voice Matters
We need your help! As of now, the five-year plan only focuses on ALS, even though ACT for ALS includes other rare neurodegenerative diseases such as HD. The Huntington’s disease community deserves a chance to have access to potentially life-changing therapies and treatments. As of now, the 5-year plan primary focus is on ALS; we need a primary focus to be HD/JHD as well.
Help the FDA understand time is simply something we don’t have and the importance of accelerating critical therapies to relieve the pain and suffering that our families are forced to endure.
The FDA opened a public docket to hear from individuals living with rare neurodegenerative diseases, and we hope you can take 5-10 minutes to submit a comment
2. Click on the blue button in the left-hand corner that says “comment”
3. Fill out the appropriate boxes
4. Press Submit
From the FDA: A comment can express simple support or dissent for a regulatory action. However, a constructive, information-rich comment that clearly communicates and supports its claims is more likely to have an impact on regulatory decision-making.
Here are things to consider when filling out the docket:
Why is accelerating access to critical therapies important to you and your family
Do you have any frustrations with the regulatory process? If so, in a constructive way, write about how that has affected you/your community and what you would like to see change.
Discuss the symptoms of Huntington’s disease that impact your family the most and why it is so important to have access to possible treatments and therapies that could help manage those symptoms.
Comment Example:
I write to you today as a member of a family that is impacted by Huntington’s disease. Currently, there are only three medications approved by the FDA for chorea only. As you know, Huntington’s disease is so much more than chorea. We need treatments for all symptoms associated with Huntington’s disease, including cognitive and psychiatric symptoms. Huntington’s disease takes away all of our loved one’s quality of life with little treatment options available. As of now, we have nothing to help slow down or halt the disease progression of Huntington’s disease. Please consider making Huntington’s disease and Juvenile Huntington’s disease a priority when it comes to accelerated access to life-changing, meaningful therapies and treatments. Without access to therapies, our community will continue to live generation after generation, like we have in the past, with limited options and finding it hard to find any hope of future relief from the pain and suffering our loved ones are forced to endure. With 4,000 people living with Huntington’s disease and 200,000 Americans at risk, we need more research and more symptom management treatment options. We need therapies to slow or halt the progression. Our future generations rely on the changes we make today.
I have seen first hand the effects of this terrible disease, My friend Nathan Wilmoth’s mother died from it. Nathan and his family now suffer from it. Anything that can be done , should be done.
I am 39 and have a CAG 49, diagnosed on 5/8/2023, I started progressing quickly, with five falls in 4 months. Every new progression happened every two months. I also fell everytwo months, as well. My movements are mostly in my legs and head and neck. my legs feel like they are moving side to side, and my my head movements have started causing migranes. I had appointment with my neurologist, on 12/4/2023, she also agress that i am progressing quicly, i belive we found an assisted living for me to go, since i have progressed this much in 8 months.