Abstract
The Food and Drug Administration (FDA) developed a draft guidance for drug
development in Amyotrophic Lateral Sclerosis (ALS) that was issued in
February 2018. The FDA draft guidance considered the recommendations
developed by the ALS community which incorporated the views of a large
group of clinical investigators, industry representatives, advocacy
groups, patients, and caregivers. This external input from the ALS
community reviewed the current state of clinical research in ALS and made
suggestions over a wide range of drug development topics and served as an
educational tool to provide the Agency with additional inputs about ALS,
the state of the science and the community’s views on key topics. In
parallel to this effort, there was an independent effort to revise and
update the ALS clinical trial guidelines. We discuss the areas of
agreement of these three documents, and the areas that provide
opportunities to improve the efficiency of drug development in ALS. It is
likely that further research into biomarkers, efficacy endpoints, and
predictive algorithms will provide greater alignment among community
stakeholders and increase clarity on drug development efforts going
forward. Continued patient engagement and inclusion of patient experience
data in every aspect of the drug development process will further
facilitate the approval of new treatments.