Skip to Content

I AM ALS Principles for Access to Promising Therapies

December 19, 2019  -  Danielle Carnival

A Note From Our CEO

There is a time when you must stand up and demand change and then work like mad with anyone who is willing to champion your mission to get that change to happen. That time is now in ALS.  

During the course of my career, I have seen successes in oncology and the life changing treatments and, yes, even cures that are already saving and extending lives. We just saw the same happen for people living with cystic fibrosis. After decades of commitment, research, and hope, patients got an answer other than, “I’m sorry there is nothing we can do.”  

We are at the same critical point in the fight against ALS where hopefully soon no patient will hear those words.  But we must do better for people living with ALS now to get there. 

As an organization working for a community facing a fast progressing, currently terminal, disease, we must approach everything we do with the urgency and intensity of someone who is in the fight of their life. This is what we will stand for and fight for as an organization. 

I AM ALS Principles for Access to Promising Therapies

The current treatment development pipeline for diseases like ALS is very promising. That provides great hope for people living with these diseases and their families. Realizing that hope requires that the clinical trial and health care systems adapt to provide access to treatments for those living with ALS now and in the future. We will fight with urgency for any improvement that aligns with the following principles: 

  1. I AM ALS supports the implementation of effective pathways for access to promising ALS therapies which have been proven safe.

Though there have been previous attempts to improve access to therapies for diseases like ALS, none have provided a clear, accessible, and funded pathway to a safe, investigational therapy.  One must be developed now.  

2. I AM ALS supports patient-centered therapy development for our community. 

The current clinical trial system does not meet the needs of the ALS community, especially for current drugs in the pipeline. It is necessary that we develop a patient-centered system that immediately provides reasonable access for any trial participant to the therapy after their participation in the trial and seeks to minimize, or remove, the number of people in the placebo arm of a trial and to expedite trial progress so new therapies are available far faster.

3. While the FDA ALS Clinical Trial guidance issued in September of 2019 is a positive step forward, I AM ALS supports clear and aggressive steps inside and outside of FDA to ensure that new trials are not forced to reflect conventional trial designs that are inappropriate for rapidly fatal diseases like ALS. 

Much has been said about the value of the new guidance from the ALS patient and advocacy community, including by us.  We believe that FDA should implement a program that clearly and publicly sets forth how the new guidance impacts any trial design developed or embarked on post-issuance date. 

4. With rising costs of novel therapies across a host of serious diseases, I AM ALS supports making all efforts to lessen the cost burden on patients - while preserving innovation - and their families who find themselves shouldering crushing prices of therapies that confront and defeat rare diseases. 

There are more than 7,000 rare diseases. Each disease on its own may be rare, but collectively they affect more than 400 million people worldwide. Said differently, a rare disease is likely to affect you or someone you love during your lifetime. We are in this fight together and must ensure that everyone can access and afford care that can extend or improve their lives.

Back to top