The ALS Association

Ice Bucket Challenge Progress

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 has been submitted to the FDA. The community-developed Guidance aims to 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. The ALS Guidance Document has the potential to 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 version submitted to the FDA, is available in the links below.

Read More

Understand Why the Guidance is Important

Patient and Caregivers

The ALS Guidance aims to increase the number of potential therapies in the pipeline and move therapies to patients faster.

Drug Developers

The ALS Guidance has the potential to provide greater clarity and predictability for industry when navigating the drug development and regulatory process and could decrease the time required and improve 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.

FDA guidance documents explain the Agency’s interpretation of or policy on a regulatory issue and are developed within the Agency. Most FDA guidance documents are not binding on the Agency or industry sponsors like law or regulation, but can provide helpful recommendations in the drug development and approval process.

Currently, there is no FDA guidance document focused specifically on ALS drug development. We believe that availability of an ALS guidance document might be able to help decrease the amount of time it takes a sponsor to move a potential therapy from initial development to approval.

The ALS community’s guidance document, which was submitted to the FDA in August 2017, is intended to provide a roadmap to help industry navigate the development process and provide the Agency with an ALS community-centered view of the disease and how it should approach potential therapies for ALS.

Thanks to a 2012 federal law which encourages and facilitates patient and caregiver involvement in the drug development process, the Guidance being developed by the ALS Community is intended to:

  • 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.

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.”

Read More

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.”

Learn About the Participants

More than 100 individuals from throughout the ALS community worked together to develop the 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.

Read More

Find Additional Resources Here

Find answers to frequently asked questions about the 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 ALS Guidance Development process and timeline.

Learn More

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

See the Glossary

View the ALS Community’s Guidance Submitted to FDA

Read More

What Happens Next?

With support made possible by the ALS Ice Bucket Challenge and in coordination with the Food & Drug Administration, The Association convened a landmark patient-focused drug development (PFDD) effort across the ALS community to prepare and submit a draft guidance, in part, to advance the Agency’s work and considerations. Together with an imminent update to the complementary ALS Clinical Trial Guidelines, also supported by The ALS Association and authored by leading clinical experts, the community-led draft guidance represents an unprecedented initiative to improve the efficiency, reliability, and speed of the therapy development process for this devastating disease.

In February 2018, the FDA issued their draft guidance on ALS Drug Development (Docket ID: FDA-2013-N-0035-0273). As expected, this document is considerably shorter than the community-developed document. The ALS community guidance purposely includes content that goes far beyond what typically is found in an FDA guidance document, and will serve an educational purpose. We are using the guidance process to provide much broader and in depth input to the FDA on topics that go beyond a guidance document, as most FDA reviewers are not ALS experts. This will help to ensure that we do not simply inform the Agency about the development of a guidance, but also provide them with additional inputs about ALS, the state of the science and the community’s views on key topics. This additional input is critical as it is intended to inform Agency decision-making and may be a valuable resource to the Agency beyond the finalized guidance itself. In fact, the FDA has communicated that the Agency expects the community-submitted guidance to be a vital resource for the scientific community regardless of what is included in the FDA version of the guidance. The extensive, community-wide work product is posted in the Food & Drug Administration (FDA or Agency) docket (Docket ID: FDA-2017-D-6503) where it continues to serve as a resource to all stakeholders.

The ALS Association is actively engaged in advocating for the most useful FDA Guidance and other regulatory improvements. To respond to the FDA’s draft guidance document, we gathered feedback from across our community, including the leaders from the community’s ALS Draft Guidance Steering Committee, and other participants in this initiative. While a number of the community’s recommendations are included in the FDA’s more concise document, we recognized room for improvement on topics such study design and endpoints, addressing the burden of trials on patients, accelerated approval considerations and benefit risk. The ALS Association’s comments on the FDA draft guidance can be found here.

Finally, as part of the guidance development process, the FDA has committed to attending an ALS community workshop to gather additional community input on their draft guidance document. A finalized workshop date, most likely in early summer, will be shared with the community soon. Please check back for additional information.

Powered by Blackbaud
nonprofit software