It is on us—those who have experienced this disease firsthand, those of us who are living with the disease, those who are serving as caregivers—to determine the value and quality of life with ALS. We need to stand up and object to discriminatory assessments that overlook the most important things that give life value.
Leaders in the fight against ALS held 250 virtual meetings with members of Congress last week to press for an exponential expansion of federal funding for ALS research and programs next year as part of The ALS Association’s annual fly-in conference.
Peter Sawyer of Mechanicsburg, PA is a military veteran and has been living with ALS for five years. He and his wife and caregiver, Lura, are tireless advocates for The ALS Association’s mission and exemplify true ALS heroes.
The ALS Association has formally objected to the use of controversial measures to evaluate ALS drugs that can make it harder to find effective new ALS treatments and get them to the ALS community as quickly as possible. These measures have been identified by the National Council on Disability as being inherently discriminatory against people with disabilities.
In 2016, Troy Fields had everything going for him. A beautiful and growing family. A highly successful and satisfying career that allowed him to travel internationally. But he also started to notice signs that something wasn’t quite right. After treatment for cancer and a battery of other tests, his ALS diagnosis was eventually confirmed. Instead of focusing on what he was going to lose, Troy opted to channel his energies to be an agent of change for the ALS community.
The National Institute of Neurological Disorders and Stroke (NINDS) issued a request for input recently to help guide its work supporting ALS research. The ALS Association is submitting input telling the agency that speed matters. We are encouraging NINDS and the rest of NIH to focus to find ways to use research to advance the health of people with ALS as quickly as possible.
The Institute of Clinical and Economic Review, commonly known as ICER, has opened a review of AMX0035 to determine the cost-effectiveness of the drug. The ALS Association is committed to making sure ICER’s review does not discriminate against people with ALS and that its analysis does not prevent people with ALS from accessing promising treatments.
Thanks to the hard work of ALS advocates, Congress has passed the Accelerating Access to Critical Therapies (ACT) for ALS Act after a unanimous vote in the Senate. The bill previously passed in the House 423-2. The bill is expected to be signed into law by President Biden.
We asked the FDA to treat the approval review process of AMX0035 with urgency. Specifically, we sent a letter to FDA asking the agency to conduct a Priority Review of Amylyx’s New Drug Application (NDA) for AMX0035 and then approve it. The Priority Review is an expedited review process, as opposed to the Standard Review process, which can take upwards of a year after the agency accepts submission of the NDA.
Amylyx recently filed a New Drug Application for AMX0035, a promising new drug that has proven safe and effective at slowing progression of ALS and extending the life of people living with the disease. The ALS Association has called on the FDA to approve the application with urgency.
Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537) was approved by a unanimous vote on Thursday, November 4 in the House Energy and Commerce Subcommittee on Health. Thanks to everyone within the ALS community who has advocated for passage and who has worked on Capitol Hill to help make this important step happen. The strong bipartisan support for this bill means smooth sailing as the bill goes to the full House Energy and Commerce Committee and then the full House for approval.
Unlike most Medicare recipients who need extensive home care and rehabilitative services, people with ALS do not improve, and the intensity of their service needs increase over time. That means standard Medicare cost control approaches don’t work well. I will discuss two examples.
After Amylyx’s announced that it intends to file a new drug application for AMX0035, The ALS Association immediately called on the U.S. Food and Drug Administration to approve the treatment for all people with ALS as soon as possible. Connecting ALS talked to the team at Amylyx to learn about the path ahead for access to AMX0035.
There is a lot to do, and this grounded focus of making ALS livable helps us hold everyone—ourselves, the FDA, and the research community—accountable to real impacts on real people with ALS and the time it takes to deliver those impacts. This week has been a big step forward for the ALS community, and we will continue urgently working to keep the momentum going.
Thanks to the tireless efforts of ALS advocates across the country, the House included many of our aggressive requests for new and increased investments in research to find treatments and a cure, slow symptom progression, reduce the number of new cases, and increase the length and quality of life for every American living with ALS.
In very powerful testimony, members of the ALS community asked members of Congress to ensure the FDA acts with greater speed and regulatory flexibility during a hearing before the House Energy and Commerce Health Subcommittee.
Following our We Can’t Wait Action meeting with the FDA, we sent a letter to the agency asking it to reaffirm its commitment to the 2019 ALS Guidance and provide us with a detailed report showing how it has implemented the guidance. This week, the ALS Association received a response from Dr. Patrizia Cavazzoni, Director of the Center for Drug Evaluation and Research.
ALS advocates are pursuing an aggressive set of public policy priorities for people living with ALS and their families. These priorities include increased federal funding for ALS research, creating new pathways for expedited approval of promising treatments including the Promising Pathway Act and ACT for ALS, making permanent expanded access to telehealth, and increasing access to high-quality health care and veteran's benefits.