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

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


Amyotrophic lateral sclerosis (ALS), sometimes known as Lou Gherig’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. This approach to bolster our understanding of the basic science of ALS will 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 a unique opportunity to come together to discuss challenges in the development of drug and biological product treatments and explore opportunities to facilitate the development of innovative medical products and increase patient access.

The Robert J. Margolis, MD, Center for Health Policy at Duke University, through a cooperative agreement with the U.S. Food and Drug Administration, is organizing a two-part workshop series to explore the state of scientific advancements for ALS. This workshop series will consist of a private workshop, a public workshop, a research roadmap, informed by listening sessions with patient groups and other stakeholders. This work, supported by experts from the patient, scientific, clinical, industry, and regulatory communities, will highlight current progress in basic and clinical ALS research, identify opportunities to address scientific, policy, and infrastructure challenges in a manner that empowers patients and furthers innovation in this space to ultimately help advance the development of medical products that can benefit people with ALS.

Schedule of Events*

  1. Stakeholder informational calls (Summer 2020- Summer 2021)
  2. Private, invitation-only workshop: Improving Basic Research, Clinical Trial Infrastructure, and Community Engagement to Support Drug Development for ALS (Winter 2020)
  3. Release of research roadmap (Spring 2021)
  4. Public Meeting (Spring 2021)

*All dates are tentative and subject to change. Please stay tuned for additional meeting details and materials.
 

Contact Information:   ALS_margolisevents@duke.edu

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.