Duke-Margolis Center for Health Policy and U.S. Food and Drug Administration Project: Advancing Therapeutic Development for ALS

Duke-Margolis Center for Health Policy and U.S. Food and Drug Administration Project: Advancing Therapeutic Development for ALS

Amyotrophic lateral sclerosis (ALS), sometimes known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects approximately 5 in 100,000 people in the United States. Despite some therapeutic advancements that are designed to relieve symptoms and improve the quality of life for people with ALS, there is no cure for this devastating disease. The lack of scientific understanding of these conditions and a lack of identification and validation of potential targets for therapy development has presented a significant challenge in the development of drug and biological product treatments. Given the significant scientific gaps in our knowledge about ALS and other neurodegenerative diseases, funding of basic research and studies to further characterize these diseases – including those to support the identification of potent targets for drugs, as well as biomarkers and clinical outcome assessments – is needed to ultimately move the field forward. A greater understanding of the basic science of ALS can help to expedite product development programs overall and be more effective than funding clinical trials of specific products.

Stakeholders across the public, private, and non-profit sectors have unique roles to play in addressing the challenges in the development of drug and biological product treatments for ALS. Increased collaboration and coordination between these stakeholders can help to facilitate the development of innovative medical products and increase patient access.

The Robert J. Margolis, MD, Center for Health Policy at Duke University (Duke-Margolis), through a cooperative agreement with the U.S. Food and Drug Administration (FDA), organized a private workshop on January 27th and 28th 2021 to explore the state of scientific advancements for ALS. The workshop agenda was informed by a series of listening sessions with patient groups and other stakeholders. The workshop event brought together experts from the patient, scientific, clinical, industry, and regulatory communities to discuss challenges and opportunities to advancing drug development for ALS.  Additional information on this workshop, including meeting materials can be access on the event’s web page.

Based on the insights shared during the workshop, the Duke-Margolis team developed a Collaboration Roadmap to Advance Drug Development for ALS that was shared with the FDA in September 2021. Read the Roadmap here.

The roadmap was delivered to FDA in September 2021 and is not reflective of recent developments in this disease area in the past year. In keeping with Duke-Margolis’s commitment to public transparency, this roadmap is being released now as a public record of the output from the private workshop. The Center is pleased to see progress being made in this space and continues to support the recommendations in this roadmap as providing a path to advance of drug development to address ALS.


Funding for this workshop was made possible in part by a cooperative agreement from the U.S. Food and Drug Administration Center for Drug Evaluation and Research. The views expressed in written workshop materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.