Correspondence to Dr Rachel L Richesson, University of South Florida College of Medicine, Department of Pediatrics, 3650 Spectrum Boulevard, Suite 100, Tampa, FL 33612, USA; J Am Med Inform Assoc 2011;18:341e346. doi: /amiajnl

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RDCRN Data Management and Coordinating Center (DMCC) Technologies/tools/support for study design & data analysis On-line protocol management system – Patient enrollment/randomization – Data entry and collection (data standards) – Adverse event reporting Training Members’ website, network coordination & reporting Public website (>2 million hits/year) Contact Registry PRISM Library of Patient Registry Questions

Evolution of this paper…. Typology of standards Best practices Research focus – clinical focus (?) ……… Review of current relevant standards Characterization and evaluation of current standards 5 Received 5 November 2008 Accepted 8 February 2011

Case Report Forms Used for structured-data collection in clinical research studies – Interventional – Observational Natural history A collection of data points, observed or extracted from other data sources Historically paper 6

Types of CRF Standards 1)Structural features of forms 1)Forms 2)Sections 3)Questions 2)Content standards - Semantics - Format of Q&A 3)Specs for using terminologies 4)Content, structure, format (online, print, language) 7

Heterogeneous Standards Groups/Organizations – NIH (various), U.S. OMB, CDISC (& ~ FDA), HL7, HITSP, ISO, … Applications/Projects – PROMIS, PhenX, caDSR, other metadata repository (flavors of ISO 11179) Information Model – HL7 RIM, BRIDG Terminology – SNOMED CT, LOINC, SNOMED + LOINC, RxNorm, MeSH, … “Best Practice” – CDASH, caBIG (VCDE space) ; AHRQ registry guidance 8

“Standard Forms” “One concern about ‘standard’ CRF use is that users should not be pressured to collect parameters defined within the CRF that are not directly related to a given protocol’s research objectives: such collection costs resources and violates Good Clinical Practice guidelines.” Instructing research staff to ignore specific parameters constitutes unnecessary information overload presenting extraneous parameters onscreen is poor interface design. - Solution: Dynamic CRF-rendering  protocol-specific CRF customization allows individual investigators to specify, at design time, the subset of parameters that they consider relevant. 9

Item Banks Required for dynamic form generation Must be searchable – Terminology can enable Must link related items Discussion Points: Is a single central item bank feasible? – Healthcare centric or research-centric? Different perspective on use of terminologies like SNOMED CT and LOINC – Question representation? Coding unique items? Indexing? 10

Process The CDASH standards document, ‘Recommended Methodologies for Creating Data Collection Instruments,’ presents important and necessary features of the CRF development process. – adequate and ‘cross-functional’ team review – version control – documented procedures for design, training, and form updates. The FDA also requires rigor in the development, validation, and use of data elements related to patient-reported outcomes as study endpoints in investigational new drug studies. ISO

Dilemmas Protocol-specific nature of research – hard to “standardize” the structure or the content Research uses of clinical data – merging two separate worlds Paper versus electronic Versioning and change Related value sets Format (paper, web; language) Validations – should they be tied to the implementation or to the question Definitions, instructions, scripts, scope notes 12

Future Challenges For Clinical Research Informatics “As the field of clinical research informatics matures, it will need to move from a mode of primarily reacting to clinical researchers’ needs through service provision, to one of active leadership by suggesting directions for standardization.” Who? 13

RDCRN Acknowledgements The RDCRN is funded by the National Institute for Neurologic Disease and Stroke (NINDS), (NIH Grant Number 7U54-RR ), a component of the NIH, and supported by the Office of Rare Diseases Research. The contents are solely the responsibility of the authors and do not necessarily represent the official views of NINDS or ORDR or NIH. The views expressed in written materials or publications do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government. 14

Thank you for your attention! Rachel Richesson 15