Recommendations for Trial Sponsors

Trial sponsors have a responsibility to people living with ALS/MND to do the right thing, given the sacrifice and toll it takes to participate in often multi-year trials.

Five recommendations for trial sponsors

1. Disseminate trial results quickly and responsibly
2. Share data and samples with the research community
3. Give clear guidance on access to treatment outside the blinded trial
4. Share short-term and long-term development plans
5. Unblind participants

1. Disseminate the results of the trial quickly and responsibly

Trial sponsors should promptly convey the results of the trial to the community, regardless of whether the results are positive or negative.

Most sponsors provide top-line results through a press release. However, we recommend that additional approaches, such as directly contacting trial participants, publishing in peer-reviewed journals, presenting at scientific meetings, and holding patient webinars, be undertaken to inform trial participants of the results of the study.

Communicating trial results in ALS/MND requires a balance of transparency and sensitivity. We recommend that sponsors:

• Present findings clearly and factually, avoiding overly optimistic language that can unintentionally foster false hope.
• When sharing biomarker results, clearly communicate the biomarker’s context of use, its potential relevance to disease progression or treatment response, and whether it is a novel discovery, ensuring expectations are grounded in the current stage of validation and practical applicability.
• Emphasize what the data definitively show, clearly acknowledge limitations and where post hoc analysis is used, and frame early-phase results as part of an ongoing scientific process rather than a breakthrough.
• Include patient advocates or clinicians in communication planning to help guide messaging that resonates with the ALS/MND community while fostering realistic expectations. While social media has a place in creating awareness of clinical research and its outcomes, the use of influencers by sponsors to promote investigational therapies is strongly discouraged.

2. Share data and samples with the research community

Many people living with ALS/MND participate in trials with the expectation that their samples and data will be widely used for research. Data and sample sharing is not just good practice. In the health research context, it allows us to replicate, validate, or develop new hypotheses, and it leads to more research engagement and improved patient care.

Sponsors should ensure that appropriate consent and de-identification processes are in place so that data and samples can be shared, and that data ownership and data security are safeguarded. Sponsors should also promptly share data to open-source platforms and share biosamples through biorepositories whenever possible and authorized by the participants.

3. Give clear guidance on access to treatment outside the blinded trial

When clinical trials end, many people living with ALS/MND continue to get access to experimental treatment under an open-label extension (OLE) or compassionate use (also known as Expanded Access, or EA). EA may also be available, unattached to a specific trial, in any phase of drug development.

Sponsors need to be clear up front if they will be providing long-term access to experimental therapy through an OLE or EA pathway, and for how long. They also need to be clear as to how participants will be selected, regardless of whether they were participants in the blinded trial or not. Clinics and telehealth organizations offering EA should inform prospective participants in advance if EA spots are limited, helping them avoid unnecessary time and expense in becoming eligible through that site or organization.

We recognize that for many sponsors, OLE and EA programs may be cost-prohibitive. We recommend that sponsors take these costs into consideration when they are fundraising or building capital for the blinded trial, so that such programs can be offered in a transparent, equitable, and objective manner. Sponsors should also inform participants, prior to starting the blinded trial, if OLE and EA programs will not be offered.

4. Share short-term and long-term development plans

ALS/MND is a devastating disease with few options for disease-modifying treatments. As such, people living with ALS/MND are always hopeful that new experimental treatments will continue to move forward in the clinical trial pipeline toward approval.

It is important for sponsors to describe the regulatory next steps that the company will undertake, regardless of whether the trial met its endpoints, did not meet them, or included subgroups that may have benefited from the therapy.

We recommend that sponsors give transparent, lay-friendly presentations (see recommendation 1) on what the results were and how they affect the sponsor’s regulatory strategy. Sponsors should state whether they will pursue regulatory approval, in which countries, and through which pathways.

5. Unblind the participants

Blinding is essential for clinical trials to remove bias that can be caused if participants or the research team is aware of who is receiving an active or placebo treatment. Blinding is also important if a trial sponsor continues to collect data through an OLE period to assess the longer-term safety and therapeutic potential of their treatment.

However, once the trial and OLE period are officially closed, or the sponsor has an approval decision from regulatory agencies, participants should be promptly unblinded and informed as to their original treatment (e.g., drug or placebo) in the blinded portion of the trial.

With the addition of numerous platform and adaptive trial designs in ALS/MND, there may be challenges in unblinding participants promptly. In these cases, participants should be informed in advance of unblinding timepoints and expectations.