STATUS: Completed Project
Existing comparative effectiveness research (CER) often relies on data captured at the point of care, re-entered into clinical research systems, and then consolidated and transformed for analysis and research purposes. The process is viewed as complex, labor-intensive, and expensive, as it requires extensive data validation and normalization to assure accurate and effective evaluation. As a result, both study designs and study infrastructure for generating and appraising real-world evidence (RWE) are often limited. To improve the capacity to study medical devices in real-world settings, the FDA Center for Devices and Radiological Health (CDRH) launched a series of strategic efforts in this area. These efforts established the National Medical Device System Planning Board and the National Medical Device Registry Task Force, which produced landmark reports that set the direction for the development of the National Evaluation System for health Technology (NEST) and the establishment of the NEST Coordinating Center (NESTcc). FDA and its partners have invested significant resources into standing up a strategic Coordinated Registry Networks (CRN) infrastructure designed to facilitate the evolution of traditional registries into CRNs capable of producing relevant and reliable data sources as part of the evolving NEST. Coordinated by one of the NEST Data Partners—the Medical Device Epidemiology Network (MDEpiNet)—the CRN Learning Community (CRN-LC) was established to promote registries’ maturity through harmonization of their minimum core data sets, commitment to the incorporation of device identification, patient engagement, and facilitating the linking of registries to other data sources. The CRN-LC established a community of stakeholders including patients, providers, manufacturers, electronic health record (EHR) vendors, hospitals and integrated delivery networks, standards development organizations, researchers, government agencies, and others interested in working together to make system changes in women’s health device safety and effectiveness.
This project continued support for the CRN-LC partnership among the national registries (aspiring to be CRNs) capturing experiences of patients treated by medical device technologies in 12 clinical areas including, but not limited to, women’s, cardiac, and vascular health. This initiative provided an opportunity to collect structured, standardized, analysis-ready patient data at the point of care and to link those data to other data sources, including administrative claims, state all-payers data, patient generated data, and other sources. Strengthening the CRN-LC and each individual CRN resulted in a more strategic approach to addressing the needs of the entire patient-centered outcomes research (PCOR) stakeholder community via harmonized and interoperable infrastructure, potentially allowing for more complex study designs.
PROJECT PURPOSE & GOALS
The goal of this project was to strengthen the CRNs as a real-world data source for high-quality, relevant, reliable, timely, and actionable evidence to improve patient outcomes of medical devices, specifically for technologies affecting women’s health. This goal was achieved by advancing CRNs’ ability to capture standardized data, include patient-generated data, and link their registry data to additional data sources.
- Advance the CRNs’ capacity for PCOR use in 12 clinical areas through their development in seven areas (attributes): patient engagement, unique device identification, data quality, efficiency, governance, sustainability, and fitness for use during the total product life cycle (TPLC).
- Pilot test and refine the existing device-specific Fast Healthcare Interoperability Resources® (FHIR) profiles (produced as part of the FY17 project, “Developing a Strategically Coordinated Registry Network for Women’s Health Technologies”) in an expanded set of three to five CRNs to demonstrate the capture and exchange of CRN data using FHIR.
- Pilot test and refine the existing instrument for capturing patient-reported outcomes to capture patient preferences in the End Stage Renal Disease registry to evaluate scientifically valid data regarding patient uncertainty in accepting a variety of benefit/risk tradeoffs within a CRN.
- Advance CRN capacity to produce linked data sets and combine heterogeneous data and develop machine learning techniques to validate the linked data sets.
- Develop a gender- and sex-specific outcome measure framework for devices and test it in the most mature CRNs (e.g., in orthopedics, vascular, cardiac, abdominal hernia, and neurologic clinical areas).
PROJECT ACHIEVEMENTS AND HIGHLIGHTS
- The project team developed a CRN assessment framework consisting of a novel tool to assess CRN data infrastructure across seven domains and five levels of maturity, with input from a multistakeholder expert group of MDEpiNet collaborators from academia; clinical, industry, and regulatory settings; and the patient community.
- The project enhanced existing registry infrastructure for studying and evaluating medical devices by harmonizing a minimum set of common data elements across registries, incorporating the collection of unique device identifiers (UDIs) and data reflecting the patient perspective, and facilitating data linkages between registries and other data sources across 12 clinical domains.
- The project conducted a pilot to test and refine the instrument used to capture patient preferences with registry data, which facilitates linking patient-contributed data to clinical data sources, supports regulators in understanding device performance, and identifies patient-centric endpoints for patient-centered outcomes research.
- The project piloted and refined device-specific FHIR standards to demonstrate FHIR’s ability to capture and exchange CRN data.
PUBLICATIONS, PRESENTATIONS, AND OTHER PUBLICALLY AVAILABLE RESOURCES
- The MDEpiNet website summarizes the scope of activities and tools developed under each of the 12 CRNs, available here: https://www.mdepinet.net/. The website also provides a list of tools including data dictionaries, implementation guides, road maps, and lessons learned.
- • The CRN Maturity Framework, which serves as a guide for researchers and clinicians to develop and advance high-quality CRNs by describing proven approaches in seven core technical areas that contribute to CRN success, was published in BMJ Surgery, Interventions, & Health Technologies. The framework is available here: https://sit.bmj.com/content/bmjsit/4/Suppl_1/e000148.full.pdf.
- Published in BMJ Surgery, Interventions & Health Technologies, the MDEpiNet Supplement includes an editorial and seven papers, all focused on advancements in and research applications of CRNs. The supplement is available here: https://sit.bmj.com/content/4/Suppl_1/e000148.
- The project produced nine peer-reviewed journal articles covering a range of studies utilizing the enhanced CRN data:
- “Safety and Efficacy of Outpatient Surgical Procedures for the Treatment of Benign Prostatic Enlargement in New York State and California (2005-2016)”, published in The Journal of Urology, available here: https://sit.bmj.com/content/4/Suppl_1/e000148.
- “Long-Term Safety with Sling Mesh Implants for Stress Incontinence”, published in The Journal of Urology, available here: https://www.auajournals.org/doi/10.1097/JU.0000000000001312.
- “Safety and efficacy of TURP vs. laser prostatectomy for the treatment of benign prostatic hyperplasia in multi-morbid and elderly individuals aged ≥ 75”, published in World Journal of Urology, available here: https://link.springer.com/article/10.1007/s00345-021-03779-2.
- “Vascular Quality Initiative Surveillance of Femoropopliteal Artery Paclitaxel Devices”, published in JACC: Cardiovascular Interventions, available here: https://www.sciencedirect.com/science/article/pii/S1936879821016563?via%3Dihub.
- “Validation of an indirect linkage algorithm to combine registry data with Medicare claims”, published in Journal of Vascular Surgery, available here: https://www.sciencedirect.com/science/article/abs/pii/S074152142200338X.
- “Development and Usability Testing of a Mobile Application to Monitor Patient-Reported Outcomes after Stress Urinary Incontinence Surgery”, published in Urology, available here: https://www.sciencedirect.com/science/article/abs/pii/S0090429521009845.
- “Sex Disparities in Long-Term Mortality after Paclitaxel Exposure in Patients with Peripheral Artery Disease: A Nationwide Claims-Based Cohort Study,” published in Journal of Clinical Medicine, available here: https://www.mdpi.com/2077-0383/10/13/2978.
- “Association of Sex With Risk of 2-Year Revision Among Patients Undergoing Total Hip Arthroplasty”, published in JAMA Network Open, available here: https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2780551.
- “Long-Term Outcomes Following Elective Repair of Intact Abdominal Aortic Aneurysms”, published in Annals of Surgery, available here: https://journals.lww.com/annalsofsurgery/Citation/2023/04000/Long_Term_Outcomes_Following_Elective_Repair_of.56.aspx.
Below is a list of ASPE-funded PCORTF projects that are related to this project:
Developing a Strategically Coordinated Registry Network (CRN) for Women’s Health Technology – This collaborative project with the FDA, Office of the National Coordinator for Health Information Technology, and the National Library of Medicine aligned existing registries of women’s health technologies through a CRN. The registry network included two existing nationwide registries—COMPARE UF registry for fibroid treatments and the National Pelvic Floor Disorder (NPFD) registry—and the new Collaborative Registry of Sterilization Therapies. These registries were linked to other major data networks including partners in the FDA’s Sentinel system, Medicare and Medicaid data, PCORnet clinical research networks, and available state data. The CRN incorporated data from patient-reported outcomes and employed FHIR standards for structured data capture from EHRs, to both enhance these registries and enrich PCOR data infrastructure used to treat or manage women’s health conditions.