title: Barriers to investigator-initiated deep brain stimulation and device research.
abstract: The success of device-based research in the clinical neurosciences has overshadowed a critical and emerging problem in the biomedical research environment in the United States. Neuroprosthetic devices, such as deep brain stimulation (DBS), have been shown in humans to be promising technologies for scientific exploration of neural pathways and as powerful treatments. Large device companies have, over the past several decades, funded and developed major research programs. However, both the structure of clinical trial funding and the current regulation of device research threaten investigator-initiated efforts in neurologic disorders. The current atmosphere dissuades clinical investigators from pursuing formal and prospective research with novel devices or novel indications. We review our experience in conducting a federally funded, investigator-initiated, device-based clinical trial that utilized DBS for thalamic pain syndrome. We also explore barriers that clinical investigators face in conducting device-based clinical trials, particularly in early-stage studies or small disease populations. We discuss 5 specific areas for potential reform and integration: (1) alternative pathways for device approval; (2) eliminating right of reference requirements; (3) combining federal grant awards with regulatory approval; (4) consolidation of oversight for human subjects research; and (5) private insurance coverage for clinical trials. Careful reformulation of regulatory policy and funding mechanisms is critical for expanding investigator-initiated device research, which has great potential to benefit science, industry, and, most importantly, patients.
authors.affiliation: From the Departments of Neurosurgery (M.L.K., A.G.M.) and Psychiatry (D.M.) and the Center for Neurological Restoration (A.G.M.), Neurological Institute, and the Center for Clinical Research (J.B.), Cleveland Clinic, OH; MacLean Center for Clinical Medical Ethics (M.L.K.), Department of Medicine, The University of Chicago, IL; and the Departments of Neurology and Neurosurgery (M.S.O.), University of Florida Center for Movement Disorders and Neurorestoration, Gainesville.
authors.forename: Michael L
authors.initials: ML
authors.lastname: Kelly
authors.forename: Donald
authors.initials: D
authors.lastname: Malone
authors.forename: Michael S
authors.initials: MS
authors.lastname: Okun
authors.forename: Joan
authors.initials: J
authors.lastname: Booth
authors.forename: Andre G
authors.initials: AG
authors.lastname: Machado
mesh_terms: D035843:Biomedical Research / Q000191:economics*; D002986:Clinical Trials as Topic / Q000191:economics*; D046690:Deep Brain Stimulation / Q000295:instrumentation*; D018795:Device Approval; D004867:Equipment Design; D005380:Financing, Government; D005381:Financing, Organized; D006801:Humans; D006930:Hyperalgesia / Q000503:physiopathology / Q000628:therapy*; D019458:Insurance Coverage / Q000191:economics; D009434:Neural Pathways / Q000503:physiopathology; D010292:Paresthesia / Q000503:physiopathology / Q000628:therapy*; D013786:Thalamic Diseases / Q000503:physiopathology / Q000628:therapy*; D013788:Thalamus / Q000503:physiopathology; D014481:United States