On some occasions, the ALS Association takes a public stance on the effectiveness of an experimental therapy. This is never easy, as there is rarely universal certainty about scientific research or the findings of individual research studies. We consult with outside independent experts before taking any position. If the drug sponsor is unwilling to provide the sufficient level of information to answer the questions we have and our independent reviewers have, we will not take a position on how well an experimental treatment works. 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 we, like most other rare disease organizations, receive a portion of our funding from the pharmaceutical industry as whole, and we may receive some funding from a specific sponsor that may also have a drug up for review. In addition, as the largest philanthropic funder in the ALS space, we directly support research in many different experimental drugs at any given time, and some of these grants include pay-back provisions or other financial interests. We follow the industry standard of publicly declaring any financial arrangements with any sponsor.

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, and we also need to manage this bias to make good decisions. We draw on the experience of our research program. We engage external experts to review hundreds of applications each year from commercial and academic scientists seeking research funding. To ensure the integrity of this process, we carefully consider our reviewers to ensure they are not conflicted for a specific proposal, and we only release the results of our reviews to the applicant. Both the applicant and the reviewer understand that they are working with proprietary information that must be kept strictly confidential.

Only after we have a full and independent assessment of a treatment’s effectiveness can we decide what our role needs to be in any approval process to best serve people with ALS. Our options range from private interaction with the FDA and other opinion leaders, engagement with the media and scientific community, collaboration with other organizations, and engaging the broader ALS community in specific campaigns. If the case for approval is strong, we may not need to take any action at all.

We have already had success with this approach. We asked independent reviewers to assess confidential information supplied by Amylyx on Relyvrio before taking a position on its effectiveness in 2020. Their reviews helped us develop a nuanced and evidence-based position we shared broadly and was reinforced by unusually strong public support by the scientific and clinical community during FDA review. We will continue to rely on external independent reviews should we be asked to comment on the effectiveness of an experimental treatment.