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Launching Effectiveness Research to Guide Practice in Neurosurgery: A National Institute Neurological Disorders and Stroke Workshop Report.

ABSTRACT: This workshop addressed challenges of clinical research in neurosurgery. Randomized controlled clinical trials (RCTs) have high internal validity, but often insufficiently generalize to real-world practice. Observational studies are inclusive but often lack sufficient rigor. The workshop considered possible solutions, such as (1) statistical methods for demonstrating causality using observational data; (2) characteristics required of a registry supporting effectiveness research; (3) trial designs combining advantages of observational studies and RCTs; and (4) equipoise, an identified challenge for RCTs. In the future, advances in information technology potentially could lead to creation of a massive database where clinical data from all neurosurgeons are integrated and analyzed, ending the separation of clinical research and practice and leading to a new "science of practice."

SUBMITTER: Walicke P

PROVIDER: S-EPMC5808147 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature

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Launching Effectiveness Research to Guide Practice in Neurosurgery: A National Institute Neurological Disorders and Stroke Workshop Report.

Walicke Patricia P Abosch Aviva A Asher Anthony A Barker Fred G FG Ghogawala Zoher Z Harbaugh Robert R Jehi Lara L Kestle John J Koroshetz Walter W Little Roderick R Rubin Donald D Valadka Alex A Wisniewski Stephen S Chiocca E Antonio EA

Neurosurgery 20170401 4

This workshop addressed challenges of clinical research in neurosurgery. Randomized controlled clinical trials (RCTs) have high internal validity, but often insufficiently generalize to real-world practice. Observational studies are inclusive but often lack sufficient rigor. The workshop considered possible solutions, such as (1) statistical methods for demonstrating causality using observational data; (2) characteristics required of a registry supporting effectiveness research; (3) trial design ...[more]

PMID: 28362926

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Project description:BACKGROUND:In neuroscience clinical research studies, much time and effort are devoted to deciding what data to collect and developing data collection forms and data management systems to capture the data. Many investigators receiving funding from National Institute of Neurological Disorders and Stroke (NINDS), the National Institutes of Health (NIH), are required to share their data once their studies are complete, but the multitude of data definitions and formats make it extremely difficult to aggregate data or perform meta-analyses across studies. PURPOSE:In an effort to assist investigators and accelerate data sharing in neuroscience clinical research, the NINDS has embarked upon the Common Data Element (CDE) Project. The data standards developed through the NINDS CDE Project enable clinical investigators to systematically collect data and should facilitate study start-up and data aggregation across the research community. METHODS:The NINDS CDE Team has taken a systematic, iterative approach to develop the critical core and the disease-specific CDEs. The CDE development process provides a mechanism for community involvement and buy-in, offers a structure for decision making, and includes a technical support team. RESULTS:Upon conclusion of the development process, the CDEs and accompanying tools are available on the Project Web site - http://www.commondataelements.ninds.nih.gov/. The Web site currently includes the critical core (aka general) CDEs that are applicable to all clinical research studies regardless of therapeutic area as well as several disease-specific CDEs. Additional disease-specific CDEs will be added to the Web site once they are developed and vetted over the next 12 months. LIMITATIONS:The CDEs will continue to evolve and will improve only if clinical researchers use and offer feedback about their experience with them. Thus, the NINDS program staff strongly encourages its clinical research grantees to use the CDEs and is expanding its efforts to educate the neuroscience research community about the CDEs and to train research teams to incorporate them into their studies. CONCLUSIONS:Version 1.0 of a set of CDEs has been published, but publication is not the end of the development process. All CDEs will be evaluated and revised at least annually to ensure that they reflect current clinical research practices in neuroscience.

Project description:Importance:One major advantage of developing large, federally funded networks for clinical research in neurology is the ability to have a trial-ready network that can efficiently conduct scientifically rigorous projects to improve the health of people with neurologic disorders. Observations:National Institute of Neurological Disorders and Stroke Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) was established in 2011 and renewed in 2018 with the goal of being an efficient network to test between 5 and 7 promising new agents in phase II clinical trials. A clinical coordinating center, data coordinating center, and 25 sites were competitively chosen. Common infrastructure was developed to accelerate timelines for clinical trials, including central institutional review board (a first for the National Institute of Neurological Disorders and Stroke), master clinical trial agreements, the use of common data elements, and experienced research sites and coordination centers. During the first 7 years, the network exceeded the goal of conducting 5 to 7 studies, with 9 funded. High interest was evident by receipt of 148 initial applications for potential studies in various neurologic disorders. Across the first 8 studies (the ninth study was funded at end of initial funding period), the central institutional review board approved the initial protocol in a mean (SD) of 59?(21) days, and additional sites were added a mean (SD) of 22?(18) days after submission. The median time from central institutional review board approval to first site activation was 47.5 days (mean,?102.1; range,?1-282) and from first site activation to first participant consent was 27 days (mean,?37.5; range,?0-96). The median time for database readiness was 3.5 months (mean,?4.0; range,?0-8) from funding receipt. In the 4 completed studies, enrollment met or exceeded expectations with 96% overall data accuracy across all sites. Nine peer-reviewed manuscripts were published, and 22 oral presentations or posters and 9 invited presentations were given at regional, national, and international meetings. Conclusions and Relevance:NeuroNEXT initiated 8 studies, successfully enrolled participants at or ahead of schedule, collected high-quality data, published primary results in high-impact journals, and provided mentorship, expert statistical, and trial management support to several new investigators. Partnerships were successfully created between government, academia, industry, foundations, and patient advocacy groups. Clinical trial consortia can efficiently and successfully address a range of important neurologic research and therapeutic questions.

Project description:Several definitions have been proposed to distinguish clinically relevant from incidental cerebral hemorrhagic transformation after thrombolytic therapy for acute ischemic stroke. We investigated which definition best identifies cerebral hemorrhages that alter long-term functional outcome in the National Institute of Neurological Disorders and Stroke (NINDS) tissue-type plasminogen activator (tPA) trials.We analyzed 4 candidate hemorrhage definitions for which the NINDS tPA trials public data set had relevant data. For each, we identified tPA-treated patients having that hemorrhage type and compared their actual functional outcomes at 90 days with their predicted outcomes had they not received tPA and not had the hemorrhage. Projected outcomes without tPA were based on a 17-variable prognostic model derived from the NINDS tPA trials placebo group.Among the 312 patients treated with intravenous tPA, 33 (10.6%) experienced any radiological intracerebral hemorrhage <36 hours of treatment, 16 (5.1%) a radiological parenchymal hematoma, 20 (6.4%) a NINDS tPA trials-defined symptomatic intracerebral hemorrhage, 12 (3.8%) an European-Australian Cooperative Acute Stroke Study 2 (ECASS2)-defined symptomatic intracerebral hemorrhage, and 6 (1.9%) a modified version of the Safe Implementation of Thrombolysis in Stroke Monitoring Study (mSITS-MOST)-defined symptomatic intracerebral hemorrhage. The ECASS2 and mSITS-MOST definitions identified the largest hemorrhage-related change in 90-day modified Rankin Scale scores (2.26-0.32=1.94, P=0.0001; 2.81-0.63=2.18, P=0.0002, respectively). These definitions also distinguished the largest hemorrhage-related change in 90-day mortality (64.7%-7.6%=57.1%; P=0.0004 for ECASS2; 68.4%-19.5%=48.9%; P=0.0152 for mSITS-MOST).The ECASS2 and mSITS-MOST symptomatic intracerebral hemorrhage definitions, which combine radiological features and occurrence of substantial early neurological deterioration, best identify tPA hemorrhages that alter final patient outcome.

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Launching Effectiveness Research to Guide Practice in Neurosurgery: A National Institute Neurological Disorders and Stroke Workshop Report.

Publications

Launching Effectiveness Research to Guide Practice in Neurosurgery: A National Institute Neurological Disorders and Stroke Workshop Report.

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