ALS survey shows patients value even modest treatment gains
Findings suggest small slowdowns could matter to many living with ALS
- Many people with ALS consider even modest slowing of disease progression clinically meaningful.
- More than a third of surveyed patients said a 5% to 10% slowdown in ALSFRS-R decline would be meaningful.
- Findings suggest trials and regulators may need to account for patient views on smaller treatment effects.
For many people with amyotrophic lateral sclerosis (ALS), even a slight slowing of disease progression is considered clinically meaningful, according to a new study based on a survey of hundreds of patients.
“These results highlight the need for clinical trial designs that are capable of detecting small but meaningful treatment effects, and for regulators to recognize even modest slowing of ALS progression as clinically relevant,” Thomas Meyer, MD, a co-author of the study and director of the ALS Center at Charité University Hospital in Berlin, Germany, said in a summary emailed to ALS News Today.
The study, “Minimum important slowing of disease progression as determined by the ALS functional rating scale – a survey of patient expectations toward disease-modifying drugs in ALS,” was published in Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.
Understanding how ALS progression is measured
ALS is a neurological disorder that causes muscle weakness and, over time, can lead to paralysis. It is a progressive disease, meaning symptoms often begin subtly and worsen gradually.
Treatments for ALS generally aim to slow disease progression. Doctors and researchers often track this using a standardized tool called the ALS Functional Rating Scale-Revised (ALSFRS-R), which measures how difficult everyday activities such as speaking, dressing, and walking have become.
But how much slowing on this scale would patients themselves consider meaningful? To find out, researchers conducted a survey that asked people with ALS directly.
“For regulators, pharmaceutical and biotech companies, and academia, understanding patients’ expectations toward future ALS therapies is of paramount importance. Despite the clear relevance of this topic, people living with ALS have rarely been asked directly about their perspectives,” Meyer said.
“In a large survey of people with ALS, [minimum important slowing] was assessed based on the monthly change in the [ALSFRS-R] by asking a seemingly straightforward question: ‘At what point of slowing of ALS, as determined by the ALSFRS-R, do you consider a drug to be important?'” Meyer added.
The survey included 522 people with ALS, though nearly a quarter said they were unable to estimate what level of slowing would matter to them.
Of the 395 people who provided an estimate, more than a third (36.9%) said a modest slowing of about 5% to 10% would be meaningful. Another 34.2% said a moderate slowing of around 20% to 40% would be meaningful, while the remaining 28.9% said ALS progression would need to slow by at least 50% to be considered meaningful.
How expectations differ among people with ALS
The researchers found a wide range of responses, likely reflecting differences in how people experience ALS. For example, patients whose disease was progressing more quickly were more likely to say that a major slowing would be meaningful to them than those with slower progression (36% vs. 25.2%).
Even so, more than half (51.7%) of participants said that a slowing of 20% or less would be meaningful for them. Meyer noted this figure closely matches results from a survey of physicians who treat ALS.
“A modest or moderate slowing of ALS progression was considered the [minimum important slowing] for disease-modifying drugs in ALS by half of the survey participants,” the researchers concluded, adding that this finding “highlights the need for trial designs capable of detecting small but meaningful effects and for regulators to acknowledge modest slowing of ALS progression as relevant.”
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