The ALS Association is committed to urgently finding new treatments and a cure for ALS. That means supporting a robust drug development pipeline and maximizing participation in clinical trials.
We highly encourage people with ALS to participate in trials whenever possible, because enrollment is key to making new therapies available for our community as quickly as possible. Enrollment in clinical trials is voluntary, and people must consent prior to participation and have the right to understand all pertinent details of the trial.
For a drug to be approved by the U.S. Food and Drug Administration (FDA), drugs go through the interventional clinical trial process or pipeline.
When drug sponsors, like pharmaceutical companies, believe they have enough evidence to demonstrate that an emerging therapy is safe and effective, they can file a New Drug Application (NDA) with the FDA. Once the FDA accepts an NDA from a drug sponsor, the agency begins a formal review to determine whether the drug shall be made commercially available in the United States. In most instances the review should be complete within 10 months of FDA accepting the NDA – a deadline commonly referred to as a PDUFA date – though in some instances the FDA can designate an NDA for a faster Priority Review that takes 6 months. During the review period, FDA is looking for the drug sponsor to demonstrate that the drug is safe and effective, that the benefits outweigh the risk and whether the proposed drug label is appropriate. Along the way, FDA may convene an advisory committee to issue nonbinding recommendations on whether to approve a New Drug Application.
For drugs that are derived from living material rather than chemically synthesized compounds, drug sponsors can file a Biologics License Application (BLA). Depending on the makeup of the drug candidate under consideration for a BLA, either the FDA’s Center for Drug Evaluation Research or the agency’s Center for Biologics Evaluation and Research will oversee the review and determine whether the drug meets the definition of a biologic, that it is safe and effective and that it meets certain purity thresholds. The process and timelines for BLA review are essentially the same as for NDA review. If FDA issues a BLA, the drug is approved for marketing in the United States.