The ALS Association

FDA-page

Patient-focused Guidance for ALS Drug Development

Overview

The resources made possible by the ALS Ice Bucket Challenge have enabled the ALS community to join together to create the first patient-focused Guidance for ALS drug development that will be submitted to the FDA. The Guidance will serve as a roadmap to help industry navigate the drug development process and provide the FDA with an ALS community-centered view of how the Agency should approach therapies for ALS. The goals are to increase the efficiency, predictability and speed of the drug development process, including clinical trials, and lead to a more effective and earlier assessment of efficacy. This will speed access, reduce costs, help ensure resources are most effectively utilized and incentivize industry to enter the ALS market and develop new treatments for ALS.

Additional information about the guidance, including the current draft, is available in the links below. The Guidance Steering Committee encourages all stakeholders to learn more about the guidance project, review the draft ALS drug development Guidance and provide comments.

Comments will be accepted until May 30, 2016.

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Step 1: Understand Why the Draft Guidance is Important

Patient and Caregivers

The Guidance will help get more potential therapies in the pipeline and move therapies to patients faster.

Drug Developers

The ALS Guidance provides greater clarity and predictability for industry when navigating the drug development and regulatory process, decreasing the time requiredand improving the prospects for success.

Clinicians

The ALS Guidance includes important information on diagnosis and the natural history of the disease, including barriers to diagnosis, disease progression, and heterogeneity.

Researchers

The ALS Guidance contains the most up-to-date information on key ALS research areas including biomarkers, disease progression and heterogeneity, and clinical trials and outcome measures.

Policy Makers

The ALS Guidance is a landmark, patient-led initiative to help drive innovation from the laboratory to the patient faster and more efficiently.

An FDA Guidance explains the Agency’s current views on a regulatory topic with the goal of making the drug development and regulatory process, including clinical trials, more efficient, predictable, and effective at assessing drug efficacy and safety.

There is no FDA Guidance for ALS Drug Development today, which means there is a lack of regulatory clarity when a drug developer considers entering the ALS space or designs clinical trials for a potential therapy. Thanks to a 2012 federal law which encourages and facilitates patient and caregiver involvement in the drug development process, the Draft Guidance being developed by the ALS Community will:

  • Clarify and improve the development and review process;
  • Attract more industry to focus on the unmet need in ALS; and
  • Help new therapies get to patients faster.

Step 2: Read What People are Saying

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.”

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

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.”

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.”

Step 3: Learn About the Participants

More than 100 individuals from throughout the ALS community worked together to develop the draft guidance. This includes 38 people with ALS and caregivers, 10 different ALS organizations, 45 of the world’s leading clinicians and researchers, 15 industry leaders from 9 different pharmaceutical companies and 5 government representatives from 3 centers at the National Institutes of Health and Centers for Disease Control and Prevention.

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Step 4: View the FDA Guidance Infographic

View and Share the Full Infographic

Step 5: Find Additional Resources Here

Find answers to frequently asked questions about the draft guidance, including what a guidance is, how it impacts drug development, and what are the next steps among other important questions.

Read the FAQ's

Learn about the timeline for developing the guidance.

Learn More about the Timeline

Read the glossary to understand terms and acronyms that are included in the draft guidance.

See the Glossary

Step 6: Download the FDA Guidance Document

Download the PDF

Final Step: Submit Your Comments

The Guidance Steering Committee encourages all stakeholders within the ALS community to submit comments about the guidance to include comments expressing support or concerns about sections of the draft guidance as well as suggestions for how it could be improved. The deadline for submitting comments is May 30, 2016. Submit comments or questions to: FDAALSGuidance@alsa-national.org.

Following the comment period, the Guidance Steering Committee will review comments, make appropriate edits and formally submit the draft guidance to the FDA. The ALS guidance, which will be a consensus-developed document, will be submitted to the FDA and will be accompanied by a “community imperatives” letter. That letter, which is currently under development, will include the real life experiences of people with ALS and place a special emphasis on the issues that are most important to the ALS community. Importantly, the community imperatives letter will include subject areas about which there is not consensus within the ALS community to make sure all stakeholders have a voice on these issues which are so critical to finding a treatment for ALS.

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