Common Problems, Common Data Model Solutions: Evidence Generation for Health Technology Assessment

There is growing interest in using observational data to assess the safety, effectiveness, and cost effectiveness of medical technologies, but operational, technical, and methodological challenges limit its more widespread use. Common data models and federated data networks offer a potential solution to many of these problems. The open-source Observational and Medical Outcomes Partnerships (OMOP) common data model standardises the structure, format, and terminologies of otherwise disparate datasets, enabling the execution of common analytical code across a federated data network in which only code and aggregate results are shared. While common data models are increasingly used in regulatory decision making, relatively little attention has been given to their use in health technology assessment (HTA). We show that the common data model has the potential to facilitate access to relevant data, enable multidatabase studies to enhance statistical power and transfer results across populations and settings to meet the needs of local HTA decision makers, and validate findings. The use of open-source and standardised analytics improves transparency and reduces coding errors, thereby increasing confidence in the results. Further engagement from the HTA community is required to inform the appropriate standards for mapping data to the common data model and to design tools that can support evidence generation and decision making

Common Problems, Common Data Model Solutions: Evidence Generation for Health Technology Assessment

There is growing interest in using observational data to assess the safety, effectiveness, and cost effectiveness of medical technologies, but operational, technical, and methodological challenges limit its more widespread use. Common data models and federated data networks offer a potential solution to many of these problems. The open-source Observational and Medical Outcomes Partnerships (OMOP) common data model standardises the structure, format, and terminologies of otherwise disparate datasets, enabling the execution of common analytical code across a federated data network in which only code and aggregate results are shared. While common data models are increasingly used in regulatory decision making, relatively little attention has been given to their use in health technology assessment (HTA). We show that the common data model has the potential to facilitate access to relevant data, enable multidatabase studies to enhance statistical power and transfer results across populations and settings to meet the needs of local HTA decision makers, and validate findings. The use of open-source and standardised analytics improves transparency and reduces coding errors, thereby increasing confidence in the results. Further engagement from the HTA community is required to inform the appropriate standards for mapping data to the common data model and to design tools that can support evidence generation and decision making

Derivatives of erythropoietin that are tissue protective but not erythropoietic

Erythropoietin (EPO) is both hematopoietic and tissue protective, putatively through interaction with different receptors. We generated receptor subtype selective ligands allowing the separation of EPO's bioactivities at the cellular level and in animals. Carbamylated EPO (CEPO) or certain EPO mutants did not bind to the classical EPO receptor ( EPOR) and did not show any hematopoietic activity in human cell signaling assays or upon chronic dosing in different animal species. Nevertheless, CEPO and various nonhematopoietic mutants were cytoprotective in vitro and conferred neuroprotection against stroke, spinal cord compression, diabetic neuropathy, and experimental autoimmune encephalomyelitis at a potency and efficacy comparable to EPO

Derivatives of erythropoietin that are tissue protective but not erythropoietic

Erythropoietin (EPO) is both hematopoietic and tissue protective, putatively through interaction with different receptors. We generated receptor subtype-selective ligands allowing the separation of EPO's bioactivities at the cellular level and in animals. Carbamylated EPO (CEPO) or certain EPO mutants did not bind to the classical EPO receptor (EPOR) and did not show any hematopoietic activity in human cell signaling assays or upon chronic dosing in different animal species. Nevertheless, CEPO and various nonhematopoietic mutants were cytoprotective in vitro and conferred neuroprotection against stroke, spinal cord compression, diabetic neuropathy, and experimental autoimmune encephalomyelitis at a potency and efficacy comparable to EPO