Two large surveys that explore the views of adult and juvenile Huntington’s disease patients and their caregivers on symptoms and treatments may help direct research efforts toward issues that matter most to those affected.
Interestingly, the symptom-focused survey revealed large differences in how patients and caregivers perceive the frequency and severity of symptoms, with caregivers’ commonly describing worse scenarios than patients.
The initiative was part of a strategy to raise awareness and gather information for a meeting between the Huntington’s Disease Society of America (HDSA) and the U.S. Food and Drug Administration (FDA). The meeting was a step toward more patient-focused approaches in drug development for Huntington’s disease.
The symptoms survey was completed by 2,591 participants, and 1,040 people completed the treatments survey. Only 600 participants completed both.
The first survey, titled “FDA Topic 1: Disease Symptoms that Matter Most to Individuals with HD,” included 38 questions divided into two identical sections. One was completed by patients with Huntington’s disease or juvenile Huntington’s disease, and the second by caregivers.
“What is striking in these data are the marked differences in the experience and perception of symptoms of HD and their impact between individuals with HD/JHD and their caregivers,” George J. Yohrling, PhD, senior director of Mission and Scientific Affairs, HDSA, and the study’s lead investigator, said in a press release.
“The frequency and impact of symptoms appear to be felt more by caregivers than individuals with HD, especially when it comes to loss of abilities. There was also a large difference in the perception of symptom frequency between caregivers and individuals with HD.”
According to the survey, 90 percent of the caregivers thought patients experienced symptoms “frequently” or “constantly,” while only half of the surveyed patients felt that way. Caregivers also tended to view patients as more impaired than the patients themselves.
The second survey, “FDA Topic 2: Perspectives on Current Approaches to Treatment,” had a similar setup. First, a section with 167 questions gathered information on demographic characteristics, and then two identical sections with 58 questions each inquired about views on medical treatment from either patients or caregivers.
“With this information we can tailor efforts more effectively to treat the symptoms that are the most disruptive to individuals impacted by HD and target symptoms that degrade their quality of life,” Yohrling said.
“While there seems to be some effective treatments for symptoms like depression and anxiety, there are still cognitive, behavioral, and motor symptoms that lack effective treatments and severely impact the lives of both individuals with HD and their caregivers,” he said. “Taking this information into account, we can refocus efforts in research and development to offer more options, and hopefully more effective treatments for individuals impacted by Huntington’s disease.”
Approaches focusing on outcomes reported by patients and caregivers have recently been adapted as a tool to improve treatment development. Including patients in discussions give investigators a more comprehensive perspective on real-life benefits and risks of various treatments, as well as existing unmet needs.
Researchers admitted that the surveys had some limitations. To participate in the survey, it was not necessary for a patient and his or her caregiver to complete the survey together, meaning that some patient’s responses lack a corresponding caregiver response.
Also, the surveys likely included patients in earlier disease stages who were more able to participate.
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