What are the biggest challenges in ALS clinical trials according to pharmaceutical companies? Survey results

Why this study?

Developing effective treatments for ALS is difficult. Many treatments that look promising do not show a clear benefit in clinical trials. TRICALS is interested in understanding why this happens. When a study does not show a benefit, it is not always clear whether the treatment did not work or whether the way the study was set up played a role. Differences in how trials are designed and carried out also make it harder to compare results and learn from previous studies.

New ALS treatments are mostly developed by researchers and pharmaceutical companies working together. Companies have an important say in how new treatments are tested, but little is known about the challenges they face or how they think trials could be improved. Therefore, researchers at the UMC Utrecht explored the industry’s perspective on these challenges.

What did the researchers do?

The researchers developed an international survey, based on interviews with people working in the pharmaceutical industry. In total, 53 professionals from 42 different companies completed the survey. The results therefore show how the industry perceives the challenges in ALS trials.

What did they find?

According to the respondents, there are three main scientific challenges:

• People with ALS differ greatly in how quickly their disease progresses and how it affects them.
• Better biomarkers are needed to show how the body responds to a treatment. Biomarkers are measurable signs in the body that show how the disease is doing.
• The tests currently used to measure the effect of a treatment may not be sensitive enough to pick up small changes.

They also named practical challenges: not enough funding, many participants dropping out of trials, and a shortage of experienced research staff.

Respondents also differed in how they think ALS trials should be set up, for example in which outcomes to measure and how long a study should last. Most respondents believed that greater standardization of key aspects of trial design would improve the quality of trials and make it easier to compare results between studies.

What does this mean?

In the industry’s view, many of the challenges come down to one thing: ALS progresses at very different speeds and in different ways from person to person. Based on these perceptions, the researchers make two recommendations:

Improve how disease progression and treatment effects are measured. More accurate measurements could help show sooner, and more reliably, whether a treatment works.

Increase standardization of ALS trials, so results can be compared more easily and lessons from one study can be used to improve the next.

Knowing how the pharmaceutical industry sees these challenges helps researchers, companies, regulators and patient organizations work together to improve how new ALS treatments are tested. This could lead to more reliable answers about which treatments benefit people with ALS and support the development of effective treatments.