On September 23, 2019, the Food and Drug Administration (FDA) issued its most recent guidance document for the development of potential treatments for ALS. This document is not binding on the FDA or the public and encourages those within the industry to communicate with the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) to discuss alternative approaches not listed within the document.
“The final guidance provides industry with the FDA’s current scientific thinking so that effective treatments with a favorable benefit to risk profile can be most efficiently developed, studied and ultimately made available to patients. It is the result of collaboration among experts across the agency and incorporates important input from patients, researchers and advocates.”
The FDA’s full statement on the final guidance document can be read here.
This publication only addresses the clinical development of drugs intended to tackle the motor deficits in ALS and not of drugs developed for other symptoms related to ALS. The FDA guidelines focus on addressing trial design issues that are specifically unique to the challenges found when studying ALS. These include techniques that can be used to minimize the use of placebo when possible, and the potential use of other methods to collect patient data, like decentralized studies and remote monitoring, to help with faster enrollment and to minimize the burden of trial participation on patients and their families.
The document discourages sponsors from excluding patients from trials unless scientifically justified with the thought that broader inclusion criteria will help fill trials faster, which could accelerate drug development. With this approach, a sponsor could conduct the primary analysis in a small subset and then use the data from the whole group as secondary support.
In regards to effectiveness endpoints for future clinical trials, the document discusses the use of ALS Functional Rating Scale (ALS FRS) scores, patient-reported outcomes (PROs), and the FDA’s openness to the potential use of digital biomarkers (e.g. wearable sensors; computer-assisted measurements).
The guidance document can be read in its entirety here.
For those with questions about the guidance document, you can reach the FDA by phone at 888-INFO-FDA or reach the Center for Drug Evaluation and Research at firstname.lastname@example.org.
To learn more about how drugs are approved by the FDA, you can watch our webinar on the topic here.