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Meeting Summary - ASK CHILDREN (Assess Specific Kinds of CHILDREN Challenges for Neurologic Devices) STUDY Workshop, September 13, 2010

Meeting Summary of the ASK CHILDREN (Assess Specific Kinds of CHILDREN Challenges for Neurologic Devices) STUDY Workshop

FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31, Conference Center, the Great Room, Silver Spring, M.D. 20993

Monday, September 13, 2010

The ASK CHILDREN Study Workshop was convened at approximately 8:00 a.m.

Workshop Participants

Carlos Peña, Office of the Chief Scientist, Office of the Commissioner, FDA

Kristen Bowsher, Division of Ophthalmic, Neurological, and Ear Nose and Throat Devices, Center for Devices and Radiological Health, FDA

John Chelonis, University of Arkansas for Medical Sciences-Arkansas Children’s Hospital/National Center for Toxicological Research, FDA

Ann Costello, Division of Ophthalmic, Neurological, and Ear Nose and Throat Devices, Center for Devices and Radiological Health, FDA

Malvina Eydelman, Director, Division of Ophthalmic, Neurological, and Ear Nose and Throat Devices, Center for Devices and Radiological Health, FDA

Suzanne Fitzpatrick, Office of the Chief Scientist, Office of the Commissioner, FDA

Charleene Frazier, National Spinal Cord Injury Association & United Spinal Association

Neil Friedman, Cleveland Clinic

George Jallo, The Johns Hopkins Hospital

Victor Krauthamer, Director, Division of Physics, Office of Science and Engineering Laboratories, CDRH, FDA

Warren Marks, Cook Children’s Medical Center

Debra Nussbaum, Cochlear Implant Education Center, Clerc Center

Phillip L. Pearl, Children’s National Medical Center

P. Hunter Peckham, Cleveland Functional Electrical Stimulation Center

Shenandoah Robinson, Rainbow Babies & Children’s Hospital, University Hospitals

Lauri Rush, Patient Representative

Joy Samuels-Reid, Division of Anesthesiology, General Hospital, Infection Control, & Dental Devices, Center for Devices and Radiological Health, FDA

Jeff Shuren, Director, Center for Devices and Radiological Health, FDA

Ann Tilton, Louisiana State University Health and Sciences Center

Susan Waltzman, New York University

Presentations and Discussions

Welcome
Jeff Shuren, Director, Center for Devices and Radiological Health (CDRH), FDA

Workshop Purpose and Questions
Kristen Bowsher, Expert Reviewer, Division Of Ophthalmic, Neurological, And Ear Nose And Throat Devices (DONED), CDRH, FDA

Pediatric Initiatives and Medical Devices
Joy Samuels-Reid, Chief Medical Officer, Division of Anesthesiology, General Hospital, Infection Control, & Dental Devices (DAGID), CDRH, FDA

Overview of Medical Device Regulation
Malvina Eydelman, Director, DONED, CDRH, FDA

The ASK CHILDREN Study-Purpose, Goals, and Progress-to-date
Carlos Peña, Senior Science Policy Advisor, Office of the Commissioner (OC), FDA

Clinical Perspectives on Pediatric Neurologic Devices
Neurosurgical Approaches to Medical Devices

Shenandoah Robinson, Rainbow Babies & Children’s Hospital, University Hospitals

Clinical Perspectives on Pediatric Neurologic Devices
Neurological Considerations

Phillip L. Pearl, Division Chief, Neurology, Children’s National Medical Center

ASK CHILDREN Study Perspectives
Neil Friedman, Staff Physician, Pediatric Neurology, Cleveland Clinic
John Chelonis, University of Arkansas for Medical Sciences-Arkansas Children’s Hospital/National Center for Toxicological Research, FDA

Patient Perspective
Lauri Rush, Patient Representative

Advocacy Community Perspective
Charleene Frazier, National Spinal Cord Injury Association & United Spinal Association
Debra Nussbaum, Cochlear Implant Education Center, Clerc Center

Science Perspectives-Invited Expert on Neurologic Devices Research and Development
P. Hunter Peckham, Cleveland Functional Electrical Stimulation Center

Summary of Workshop Presentations and Discussion

Welcome

Jeff Shuren, Director, Center for Devices and Radiological Health (CDRH), FDA

Workshop Purpose and Questions
Kristen Bowsher, Expert Reviewer, Division Of Ophthalmic, Neurological, And Ear Nose And Throat Devices (DONED), CDRH, FDA

  • Dr. Bowsher presented the workshop purpose questions for participants . She provided background on existing pediatric guidance, legislation, and the mechanism of support for the ASK CHILDREN Study. She also reviewed the five devices studied under the ASK CHILDREN Study. Dr. Bowsher presented the workshop questions to participants.

Pediatric Initiatives and Medical Devices
Joy Samuels-Reid, Chief Medical Officer, Division of Anesthesiology, General Hospital, Infection Control, & Dental Devices (DAGID), CDRH, FDA

  • Dr. Samuels-Reid presented an overview of CDRH- sponsored pediatric initiatives, surveillance systems, and other FDA pediatric-specific activities, including workshops. She provided a more detailed background on the pediatric medical device guidance. She also reviewed pediatric- specific considerations such as human factors, growth and development, and discussed adequate safeguards. and protections for pediatric patients.
  • Committee Discussion- Workshop participants discussed pediatric subpopulations, the pathophysiology of neurological disorders and conditions, behavioral and psychosocial factors, and the range of needs of different pediatric subpopulations. The Committee discussed the need to follow adolescents 18 to 21 years in pediatric settings and discussed challenges pertaining to transitioning them to adult medical services thereafter.

Overview of Medical Device Regulation
Malvina Eydelman, Director, DONED, CDRH, FDA

  • Dr. Eydelman presented an overview of CDRH’s mission, the definition of a medical device, and risk based modeling for medical devices. She also provided a more detailed discussion on medical device classification schemes, regulatory routes for clearance of a medical device, including the premarket approval route, premarket notification route, and humanitarian device exemption, and postmarket activities.

The ASK CHILDREN Study-Purpose, Goals, and Progress-to-date
Carlos Peña, Senior Science Policy Advisor, Office of the Commissioner (OC), FDA

  • Dr. Peña presented the purpose, origin, and a description of the ASK CHILDREN Study. He discussed the ongoing IRB review and study oversight for the trial. He also presented in more detail aspects of the ASK CHILDREN Study, including devices of interest, eligibility criteria, trial design, and progress to date. He reviewed short-term and long-term deliverables of the studies and next steps.
  • Committee Discussion-Workshop participants discussed progress to date, ways to increase study enrollment, and expanding eligibility criteria to increase the data to be gathered within this study.

Clinical Perspectives on Pediatric Neurologic Devices
Neurosurgical Approaches to Medical Devices

Shenandoah Robinson, Rainbow Babies & Children’s Hospital, University Hospitals

  • Dr. Robinson provided an overview of neurological disorders, including hydrocephalus & other abnormalities of fluid dynamics, movement disorders, epilepsy, neuromuscular disorders, cerebrovascular disorders, and deformational plagiocephaly. She discussed the application of medical devices to treat each condition and cross cutting issues within each disorder area, including comparison to other treatment options, efficacy, complications, and follow-up. She also discussed longevity, malfunction, and infection as important considerations for medical device development.
  • Committee Discussion-Workshop participants discussed cross cutting themes of pediatric medical device development including longevity, malfunction and infection in greater detail. Workshop participants also discussed additional considerations for pediatric medical device development.

Clinical Perspectives on Pediatric Neurologic Devices
Neurological Considerations

Phillip L. Pearl, Division Chief, Neurology, Children’s National Medical Center

  • Dr. Pearl presented an overview of pediatric neurological medical devices including the deep brain stimulator for dystonia, cochlear implants for sensorineural deafness, vagus nerve stimulator for epilepsy, sacral root stimulator for neurogenic bladder, shunts for hydrocephalus, and intrathecal baclofen pumps for spasticity. He presented a case study on the vagus nerve stimulator for epilepsy, conclusions from the study, and recommendations broadly applicable to pediatric medical device development. He also reviewed the pediatric organization and programs at the Children’s National Medical Center.
  • Committee Discussion- the committee discussed the importance of pediatric protections and understanding the natural history of the disorder being treated and the appropriate time of intervention. Discussions surrounding the appropriate age range to define as a pediatric population were also held.

ASK CHILDREN Study Perspectives
Neil Friedman, Staff Physician, Pediatric Neurology, Cleveland Clinic

John Chelonis, University of Arkansas for Medical Sciences-Arkansas Children’s Hospital/National Center for Toxicological Research, FDA

  • Dr. Friedman presented an overview of outcomes and measures for effectiveness in pediatrics, including definitions, questions, and outcomes. He discussed limitations of pediatric research outcome measures and the unique considerations of pediatric populations such as development age specific endpoints, surrogate markers, and efficacy outcomes. He also provided considerations for pediatric developmental considerations, device trials, Clinical Efficacy versus Functional Effectiveness, and considerations for specific neurologic disorders and conditions. He closed with clinical relevant endpoints and hindrances to device development.
  • Committee Discussion-Workshop participants discussed medical device regulation and the practice of medicine, efficacy versus effectiveness, and quality of life issues. Workshop participants also discussed the challenge in gathering sufficient data for the determination of safety and effectiveness.
  • Dr. Chelonis presented an overview of overview of pediatric behavioral assessments and instruments. He discussed regulatory decision making including risk benefit ratios and quality of life measures, secondary outcome measures, qualitative vs. quantitative methods, and how such issues relate the ASK CHILDREN Study. He also discussed assessing quality of life in children, issues when assessing children in clinical studies, and how the study design, parental support, and environment can affect clinical outcomes.
  • Committee Discussion-Workshop participants discussed device maintenance as it relates to pediatric quality of life. Workshop participants also discussed medical device patient selection.

Open Public Hearing Presenters

  • Angus A. Wilfong, MD, Associate Professor, Pediatrics and Neurology, Baylor College of Medicine, Medical Director, Comprehensive Epilepsy Program, Texas Children’s Hospital, Houston, Texas
  • Laurene McKilliop, Chief Executive Officer, Hydrocephalus Association

Patient Perspective
Lauri Rush, Patient Representative

  • Ms. Rush presented a cochlear implant pediatric medical device view from the parent perspective. She provided observations for at home and at school, medical treatment, and other considerations. She also discussed future needs in the areas of research needs, communication, education, equipment, new technology, and children vs. adult issues.

Advocacy Community Perspective
Charleene Frazier, National Spinal Cord Injury Association & United Spinal Association

  • Ms. Frazier presented the mission of the National Spinal Cord Injury Association & United Spinal Association, approaches for the protection and promotion of public health in children and adolescents, as it relates to the ASK CHILDREN Study, unmet needs including at home and at school considerations, and optimization of medical device for spinal cord injury. She also presented demand of medical treatment/care related to spinal cord injury devices.

Debra Nussbaum, Cochlear Implant Education Center, Clerc Center

  • Ms. Nussbaum presented considerations for cochlear implant patients, including decision making, factors impacting outcomes, outcome measurements, and post implant expectations. She discussed post implant maintenance throughout the lifetime of a pediatric patient. She also discussed considerations for habilitation services and the importance of the educational environment for the patient.

Science Perspectives-Invited Expert on Neurologic Devices Research and Development
P. Hunter Peckham, Cleveland Functional Electrical Stimulation Center

  • Dr. Peckham presented an overview of functional electrical stimulation devices and compared device use between adult and pediatric patient populations. He provided a more detailed background by using case studies on medical device considerations that are relevant to children and adolescents. He also discussed future applications and the importance of smarter devices for pediatric populations.
  • Committee Discussion-Workshop participants discussed the goal of eliminating external technologies, accelerating the research and development of devices for children, and the importance of orphan product development.

Workshop Conclusion & Next Steps
Carlos Peña, Senior Science Policy Advisor, OC, FDA
Joy Samuels-Reid, Chief Medical Officer, DAGID, CDRH, FDA

  • Dr. Pena presented common themes discussed during the course of the workshop proceedings including general considerations such as pediatric subpopulations, the importance of outcome measures, and different regulatory needs may exist for different device types. He acknowledged d evice specific considerations such as longevity, smarter devices, improved adaptability of device to the child, malfunction rates, unanticipated adverse events, and impact upon pediatric quality of life. He also reviewed t rial design considerations relevant to pediatric medical development including informed assent, multi-center opportunities, and awareness of pediatric subpopulations. He reviewed upcoming meeting outputs, the meeting transcript, meeting summary, and opportunity for public comments via the public docket for comment. He also noted the agency’s interest to c ontinue to evaluate pediatric guidance, continue evidence-based assessments to address unmet needs for the pediatric population, and d evelop pediatric registries to collect data on device use in children.

The meeting adjourned at approximately 4:30 p.m.

Please see transcript for details  

I certify that I attended the September 13, 2010 ASK CHILDREN Study Workshop and that this meeting summary accurately reflects what transpired.

Carlos Peña, Ph.D., M.S
Workshop Moderator

Kristen Bowsher, Ph.D.
Workshop Co-Moderator