CEO Laura Dalle Pazze’s testimony to the FDA Advisory Committee for AMX0035

My name is Laura Dalle Pazze, and I am the CEO of I AM ALS. I have no disclosures.

For the past 14 years, I have poured my heart, mind and soul into service at medical research nonprofits. At the core of my work lies a question that could not be more urgent: How do we get safe and effective treatments to people in need as quickly and efficiently as possible? Through my career, I have contended with three horrendous diseases: Parkinson’s, Duchenne muscular dystrophy and now ALS – conditions that rob people of their physical abilities, their dignity and their lives. I have worked on developing more rigorous preclinical testing standards to weed out poor drug candidates. I have helped develop and validate more sensitive outcome measures to better tease out a drug’s benefit. I have used data-driven modeling to improve clinical trials so it doesn’t take decades and billions to get help.

Here today is a chance to fulfill a promise made to people with ALS. We will listen to you and consider your input. We will be flexible and consider data from one controlled study. We will consider the risk/benefit profile of the people who are affected. We will do a better job of using advancements in technology, our understanding of your disease and regulatory flexibility to get you the help you need. The FDA has said all of this. Here is a chance to put action to those words.

Unlike many neurological conditions under this panel’s purview, ALS is not a disease of decades. It is measured in moments, like an aggressive cancer. A 2017 JAMA Oncology review found that of 62 cancer drugs approved from 2003 – 2013, the average survival benefit was 3.5 months. For people facing ALS’ median time to death from diagnosis of 2-3 years, this drug offers an average of 6 and a half more months on this earth. Please conduct your review and analysis in the ALS context. Focus on the functional outcome measures and analysis we do have rather than biomarkers and tools we aim to make possible in years to come. When considering p-values in ALS, remember the heterogeneity of our disease. Value this hazard ratio the way the cancer community would.

Please also remember that the testimony from people with ALS is critical to contextualize the data presented today. People living with ALS are experts in how their disease affects their lives and what constitutes meaningful effect. Their testimony delivers tangible examples of what two points on the ALSFRS-R and 6.5 more months actually mean in real life: another baseball game with the kids. The dignity of being able to feed oneself. Time to record a grandmother’s voice for her new grandchild.

I am sure this panel is concerned about possibly making a type 1 error — supporting a drug that does not work. I ask you to be equally concerned about the danger of a type 2 error – NOT approving a drug that DOES work. If we wait for the phase 3 data, three years will pass. That means 20,000 presently living with ALS die while waiting, while an additional 20,000 will be newly diagnosed, decline and some also die without access to this drug.

This drug is not a cure, but it does not need to be for its effects to be transformative for people living with ALS. And these effects are. You have the opportunity here to help take a step toward making ALS a chronic, manageable disease. Please take it.

We will find cures for ALS. The only question is when. That answer depends on you.

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