Mitigating Risk In ALS Clinical Development

The lack of available treatments for ALS has led many ALS patients to view clinical research as the best available care option. That can make it easier for sponsors to attract patients to their trials.

Though, while they may be generally eager to participate, researchers still have to take the time to understand the unique needs of these patients and their families so they can make participation feasible and strengthen engagement. That requires thoughtful upfront planning, and a clear understanding of the obstacles patients face when participating in an ALS trial.

Collaboration across key stakeholders, including patients, sponsors and regulators, is key to driving new research and advancements in ALS treatments. For example, increasing public awareness of ALS brought these groups together to inform new guidance, resulting in the September 2019 update from the U.S. Food and Drug Administration: Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry.² The guidance integrated feedback from ALS patient advocacy groups (PAGs), and it has largely been viewed as a positive development that is encouraging much-needed innovation in ALS clinical trial design.