On some occasions, we choose to take a public stance on the effectiveness of an experimental therapy. This is never an easy decision as there is rarely universal certainty about the outcomes of scientific research or the findings of individual research studies.

In addition, many of us at the ALS Association have connections to people with ALS, and we desperately want every experimental treatment to work. We are all emotionally invested so we need to manage this bias to make good decisions. Therefore, we only take a position on drug approvals after consulting with outside independent experts.

Specifically, we ask sponsors to share data that will be submitted to the Food and Drug Administration (FDA) and then conduct an independent peer review with a panel of external experts who are not connected in any way with the program or the sponsor.

It is only after we have a full and independent assessment of a treatment’s safety and effectiveness that we can determine what our role should be in any review process to best serve people living with ALS. Our options may include interacting with the FDA and other opinion leaders, engaging with the media and scientific community, collaborating with other organizations, and/or engaging the broader ALS community in specific advocacy campaigns. If the case for approval is strong, we may not need to take any action at all.

When a drug sponsor does not provide a sufficient level of data or does not provide data with adequate time to complete the peer review process, we are unable to assess how well an experimental treatment works and will not take a position on approval. We will simply continue to urge the FDA to move forward as quickly as possible, staying consistent with its 2019 Guidance on ALS clinical trials.

We should note that, like most other rare disease organizations, we receive a portion of our funding from the pharmaceutical industry, which could include some funding from a specific sponsor that may have a drug pending review. In addition, we directly support scientific investigations of many different experimental drugs at any given time, and some of these grants include pay-back provisions or other financial interests. However, it is important to emphasize that funding—either provided by us or received by us—does not factor into our decisions about whether to publicly support or advocate for a therapy. We also follow the industry standard of publicly declaring any financial arrangements with any sponsor.