The majority of patients with amyotrophic lateral sclerosis (ALS) report wanting new treatments to stop their disease’s progression and so improve breathing function, muscle strength, and mobility, according to the results from a self-reported online survey of ALS patients and caregivers.
These findings may help define future guidelines for the development of therapies for ALS and fulfill the needs of the ALS community, the IMPACT-ALS survey showed.
The results of the survey were discussed at the 29th International Symposium on ALS/MND in Glasgow, Scotland. The presentation, “Updated results from a US focused online survey of patient and caregiver perspectives on disease burden in ALS,” was given by Amy Laverdiere, director of new product planning and business analysis at Cytokinetics.
A total of 1,534 people participated in the U.S. online survey — including 813 ALS patients, 74 people who assisted ALS patients, and 647 caregivers — who provided their views on disease burden, functional outcomes, treatment, and clinical trial participation. They were recruited at patient meetings and clinics, and online by email and social media notifications to members of local support groups and patient registries. The average ALSFRS-R score reported by 242 participants was 32.
The online survey was anonymous and conducted between October and November 2017. People with ALS were represented from every state in the U.S., with representation across states comparable to the portion of the U.S. population in each state.
ALS patients were asked 30 questions about their experiences over the past two weeks, while caregivers were asked 43 questions about the experiences of their patients as well as about their burden over the same period of time.
The survey results showed that nearly every ALS patient experienced at least one symptom during the prior two weeks. These included weakness in limbs (89%), fatigue (79%), muscle strength loss (76%), cramps and twitches (63%), balance issues (58%), speech problems (57%), muscle stiffness (53%), and shortness of breath (52%).
When asked about the most wanted outcomes in a new treatment, respondents selected stopping the progression of the disease (48%) — in particular, they wanted improvements in breathing/respiratory function (86%), muscle weakness (70%), and mobility (67%).
“These results may inform drug development in areas of greatest patient burden and highest unmet need, decision-making by the FDA and health insurers and other policy issues,” the researchers wrote. “We plan to submit these results in a Voice of the Patient Report for ALS to the FDA (U.S. Food and Drug Administration).”
About 71% of ALS patients who were offered participation in a clinical trial did take part, with many stating as their reason a willingness “to contribute to the greater good.” Those who did not participate cited ineligibility as their reason.
More than half of caregivers (65%) rated their health as somewhat or much worse since they began caring for a person with ALS. In addition, 94% reported medium, high, or maximum stress levels during the previous two weeks.
The survey did not require patient and caregiver travel, compared with the in-person meetings of the U.S. Food and Drug Administration (FDA) patient-focused drug development (PFDD) initiative.
Biogen, Ionis Pharmaceuticals, and Cytokinetics provided financial support for the development of the survey, which resulted from a collaboration of the ALS Association with regulatory and methodology experts, ALS clinical leaders, an ALS patient, a caregiver, and industry representatives.
A similar survey is currently under development in Europe to study the experiences of people with ALS and their caregivers, and to inform global ALS drug development.
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