The disease is progressive, meaning the symptoms get worse over time. Voluntary muscles are those we choose to move to produce movements like chewing, walking, and talking. Our lives, and the lives of millions of others, depend on it.What is amyotrophic lateral sclerosis (ALS)?Īmyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a rare neurological disease that affects motor neurons-those nerve cells in the brain and spinal cord that control voluntary muscle movement. We cannot adequately stress to you the need and importance for immediate action by the FDA to combat ALS. We recognize the Guidance alone will not cure ALS, but it is an absolutely critical first step.
LOU GARRISON DISEASE TRIAL
Without it, we find ourselves handicapped by antiquated trial designs - designs that hinder the development of creative approaches to testing novel compounds and slow the development of critical, disease-altering therapeutics.
We focus on this document not because we enjoy writing letter after letter, but because it is critical to the fight against ALS.
LOU GARRISON DISEASE UPDATE
We demand you make a commitment to publicly and regularly update us on the progress toward release, and an assurance that your staff will explain the ways you have incorporated our feedback and our lived experiences into the Guidance. So we write to request that you move past well-intentioned words and finally provide a firm date by which the Guidance will be released. At a certain point, words, no matter how well-intentioned, become meaningless. Last we heard, FDA has now indicated that it “hopes” to publish the Guidance before the end of the year. Time and again the FDA either failed to respond or punted the deadline for publication. In May, Senators James Inhofe and Ron Johnson wrote about the Guidance. In April, Representative Jason Crow followed up about the Guidance. In February 2019 some of the undersigned met with senior FDA leadership about the Guidance. In November 2018, ten Members of Congress sent a letter asking about the Guidance. The Draft Guidance document was released in February 2018 and the public comment period closed on April 21, 2018.
Indeed, despite its words, its actions display a true indifference to the plight of those living with ALS. There are, however, an increasing number of potential breakthroughs that provide us hope, not only for those living with ALS, but also for those living with a myriad of neurological diseases that affect over 100 million Americans, including multiple sclerosis and Parkinson’s disease.ĭuring the multi-year process related to this Guidance, FDA repeatedly professed to be “very much aware of, and sympathetic to, the needs of patients with debilitating diseases such as ALS.” Yet despite telling Members of Congress that it is “actively working to finalize the ALS Draft Guidance and hope to do so expeditiously,” FDA has repeatedly kicked back the date for the publication of this Guidance.
We are among the thousands of Americans suffering from ALS. We write to you as individuals and families who suffer from Amyotrophic Lateral Sclerosis (ALS) to request final revisions to and publication as soon as possible of the Food and Drug Administration’s (FDA) Draft Guidance document: “ Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment. Collectively, the letter and the petition included more than 20,000 signatures. The letter seeks to break the cycle of delay by requesting the FDA Commissioner publicly provide a firm date for the release of the guidance and a clear explanation of how the ALS community’s concerns will be addressed in the guidance.īelow is the letter that I AM ALS hand-delivered on July 24th, 2019 to the FDA along with an additional petition calling on the FDA to approve pending ALS treatments as expeditiously as possible. The I AM ALS Clinical Trials Committee, made up of 15 patients and advocates, wrote a letter to compel action on the ALS clinical trials guidance. Time matters in a fight where 90 percent of patients die five years after diagnosis. For years, people living with ALS and additional ALS advocates have been asking the FDA to publish a revised ALS clinical trials guidance.
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