SILVER SPRING, MD. — The ASK Children study, a Food and Drug Administration—led clinical trial designed to gather data about the use of neurologic devices in children, has enrolled 18 patients since launching in March 2009.
Through 2011, the FDA wants to enroll 100 pediatric and adolescent patients aged 7-15 years who have been implanted with a neurologic-related medical device for up to 1 year.
Despite the slow progress, the agency signaled at the workshop that the trial remains an important priority. The gathering was called to collect information on how to improve regulators' approach to evaluating pediatric neuroprostheses.
The ASK Children (Assess Specific Kinds of Children, askchildrenstudy.org
Through interviews with the children and adolescents, the agency will gather data about scientific and medical device—related issues. Regulators hope the data will lead to more efficient approaches in evaluating the devices and the patients' experiences with them, as well as the development of similar, new technologies.
Requirements Are an Ongoing Issue
Including pediatric needs in the device evaluation process is an ongoing issue for the FDA, and one that has gotten more attention as the agency strives to implement pediatric-focused provisions of the FDA Amendments Act of 2007.
This is an “emerging science area as we continue to learn about the nervous system,” said Dr. Peña, who is leading the trial with Kristen Bowsher, Ph.D., an engineer in the FDA's Office of Device Evaluation.
Study organizers hope to enroll 20 children and adolescents each who have been implanted within the last year with five kinds of neurologic devices: deep brain stimulator, spinal cord simulator, cerebral spinal fluid shunt, vagus nerve stimulator, and cochlear implant.
The children will be required to participate in two 1-hour in-person or telephone interviews about 6 months apart. Three sites have been chosen for the study: the FDA Parklawn Building in Rockville, Md.; the Arkansas Children's Hospital in Little Rock; and the Cleveland Clinic. Patients also will be required to answer questionnaires about general quality of life.
Regulators hope to obtain information on human factors, safety, usability, adverse events, and possible postmarket issues immediately following implementation of high-risk devices. The initial study will be expanded in the future into other pediatric-related device areas.
Unique Considerations Are Needed
Dr. Warren Marks, medical director of the movement disorder and neurorehabilitation program at Cook Children's Medical Center in Fort Worth, Tex., said the FDA should consider quality of life factors in evaluating neurologic device use, as well as safety and efficacy, because “there is no really good quality of life measure out there right now.”
He argued the trial would gain more relevant data on quality of life if it were to include patients who have had device implants beyond 12 months, rather than limiting the time frame to within 12 months.
Dr. Philip Pearl, chief of the division of child neurology at Children's National Medical Center in Washington, said the upper-age cut-off for classifying child neurology device implant patients as “adolescent” should be extended from 18 to 21 years because the unique emotional needs in dealing with the implants and their related health conditions are still prevalent at that older age.
Workshop attendees pushed for children's devices that are smarter, smaller, and, ideally, self-contained, without exposed wires that children could play with and damage. Lauri Rush, who spoke about her daughter's experience with the device, emphasized the need to factor in a child's active lifestyle with devices such as cochlear implants. “How do you keep on a device while [a child] is in gymnastics?” she asked.
The workshop participants agreed that it is difficult to encourage companies to develop devices for the pediatric population because they do not always see profit potential. The 2007 Amendments Act included a provision allowing device firms to profit from pediatric-targeted indications of humanitarian use devices, though industry says it needs more incentives.
Dr. Marks and Dr. Pearl said they had no conflicts of interest.
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