The ALS Association
ALS Ice Bucket Challenge Progress

Read What People are Saying

The ALS community, including a group of nearly 30 people living with ALS and their caregivers, has been given the rare opportunity to participate in this important FDA Guidance effort. As much as we commend the efforts of the scores of people, organizations and government agencies who have devoted decades of work trying to crack the code of this dreadful disease, and as much as we recognize that significant strides have been made in recent years, we believe that great improvements can be made in the way that drugs and treatments are discovered, tested and approved. Including us in this effort has added our unique blend of knowledge, experience and passion to the process.”

This ALS Guidance-- the first ever for the disease-- will be a big asset to physicians, researchers, industry, and most importantly, our patients. It contains the most up-to-date information on key ALS research areas including biomarkers, disease progression and heterogeneity, and clinical trials and outcome measures; includes important information on diagnosis and the natural history of the disease, and barriers to diagnosis; and represents the work of the foremost ALS experts in the world, leading industry representatives and the patients and families that live with ALS every day.”

I sincerely believe that we'll look back at the ALS Guidance Project as a seminal contribution to improving the conduct, efficiency and outcomes of ALS clinical trials.”

We are very interested in elevating the patient voice during drug development.”

The ALS Guidance document demonstrates how much can be accomplished when a diverse group of dedicated individuals and organizations come together to advance a singular common cause—to ensure that safe and effective treatments and cures are made available to those living with ALS as quickly as possible. MDA is proud to have been part of such an important undertaking and is grateful to all of the stakeholders who have invested their time and efforts in this endeavor.”

The ALS guidance project was unique in that the recommendations came from researchers as well as patients and people in industry. By following the recommendations made, we hope to greatly improve ALS clinical trials in the future and have a greater rate of success.”

By bringing together expert voices from academic, industry, advocacy organizations, patients and patient representatives, this document has gathered into one place how to approach developing novel therapies for ALS. We are enthusiastic that this guidance document will enhance ALS therapy development and provide a valuable resource for patients, caregivers and all of those engaged in ALS research.”

This landmark guidance represents a major milestone for the ALS community that will advance our shared mission to accelerate the pathway for development, review and approval, for much needed treatments for people living with ALS.”

It has been a pleasure to work side by side with patients, families, clinicians and scientists on the ALS FDA Guidance. This collaborative project should streamline the process of ALS drug development and speed our search for a cure.”

The ALS Guidance will act as a roadmap to the design of ALS trials, guiding investigators and trial sponsors as they develop novel therapies for ALS. The presence of this roadmap holds the promise of more rapid development, higher likelihood of success in late-stage trials, and a larger number of new clinical development programs in ALS.”

The ALS Guidance project is the most comprehensive document addressing all aspects of ALS diagnosis, management, and clinical trial design that I have seen. It’s been an exciting project that has allowed experts across multiple disciplines to coordinate ideas addressing the challenges of ALS care.”

The ALS Guidance Project represents the combined efforts of experts in ALS clinical trial methodology as well as those who study the natural history and underlying pathophysiology of the disease, patients with ALS and ALS caregivers. The document resulting from this effort will be incredibly useful in guiding regulatory pathways, but will also be a resource for those thinking about new treatment strategies and for patients and caregivers wanting to both understand and participate in the ongoing effort to improve care.”

Our focus right now is on a treatment that will slow or stop this disease. This Guidance document is a huge leap forward in that direction and our journey to find treatments will be accelerated thanks to everyone in the ALS community, who have participated in the process to develop this draft Guidance. It truly is a landmark effort that breaks new ground in the fight to end this disease.”

This Guidance will be of tremendous benefit, helping industry hasten drug development, bringing early discoveries in the lab all the way through to clinical trials and ultimately FDA approval. The ALS Ice Bucket Challenge was critical to making the Guidance a reality. We are so thankful for the collaboration at all levels to get it to this very important stage.”

The profound unmet medical need and lack of successful new therapies compels the entire ALS community to improve the ALS drug development process. The ALS Drug Development Guidance provides much needed clarity and predictability for the biopharmaceutical industry as companies consider entering the ALS space, plan their clinical trials, and work through the regulatory approval process.”

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