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Statement from Danielle Carnival, CEO, I AM ALS

June 18, 2020  -  Danielle Carnival

From day one, I AM ALS has sought to listen to and empower people living with and impacted by this disease to fight for cures and a better life. We also have promised to urgently change the system where that system isn’t working for patients, to work vigorously on increasing research budgets, and to create pathways to access effective treatments and cures as soon as they are available.  

In fact, we put out our principles for access to investigational therapies in December and we stand by them today. I said then, “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.”

This fierce determination to bring about a more promising future for people living with ALS doesn’t mean we can’t have a dialogue about the best way to enact this change. In fact, it demands that we do just that. We welcome that exchange of ideas with everyone who is working to make a better today and future for people impacted by ALS. 

But first, we want to clear up one central claim included in the ALS Association’s response to the ACT for ALS (H.R. 7071) that we don’t agree with, in fact - namely, that “The current iteration appears to support underwriting access for a few people with ALS to expensive experimental therapies that have not yet been proven effective and that are therefore not covered by insurance or Medicare.” The grant program created by this bill rights the wrong that certain therapies remain out of the hands of patients who aren’t in a given clinical trial simply because they have been brought forward by small biopharmaceutical companies who have a more difficult time financing expanded access. Funding decisions for this program are in the hands of NIH, which can consider cost and reach as part of the evaluation process, and FDA, which validates expanded access programs on a series of criteria including safety and potential benefit to patients. This is not for the treatment of a few, but potentially creates access for the thousands or more who will currently live and die without even the chance at a therapy that could extend or save their life. It does not distract or detract from existing efforts to get approved FDA therapies for ALS - rather, it enables additional use of expanded access as part of clinical development, which creates options for more people living with ALS and adds critical, otherwise missing, data to our understanding of promising treatments.

We were happy to see a response to these proposals by the ALS Association and think a public exchange of ideas would be a great way to further develop strategies to make progress for ALS patients living today. We will be reaching out to them to find a time to schedule a public virtual townhall for me and their President & CEO Calaneet Balas to discuss our organizations’ vision on these topics and how we can work together to make them real.  

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