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Real-World Evidence: The Future of Data Generation and Usage


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As data is captured through electronic health records, registries and unique device identifiers, the generation of evidence based on this data is expected to play a crucial role in informing orthopedic manufacturers’ decisions before and after regulatory approval. While regulators, payors, hospitals and manufacturers support this shift, they acknowledge that gaps remain in its optimal execution. Priority considerations include how to generate evidence to expedite regulatory market decisions, device indication expansion, postmarket studies, postmarket surveillance and reimbursement decisions. The National Evaluation System for health Technology Coordinating Center (NESTcc), an initiative of the Medical Device Innovation Consortium (MDIC), is leading the conversation with various stakeholders, including FDA and orthopedic device companies to support the sustainable generation of Real-World Evidence (RWE) using Real-World Data (RWD).

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Real-World Evidence: The Future of Data Generation and Usage

  1. 1. Rachel R. Rath, MBA, MPH Chief of Staff, NESTcc Wednesday, June 12, 2019 Real-World Evidence: The Future of Data Generation and Usage through NESTcc
  2. 2. MDIC RECEIVED INITIAL FUNDING FOR NESTcc IN 2016 MDIC is a public-private partnership that facilitates programs and activities to advance the medical device regulatory process for patient benefit. These programs are housed within four core initiatives of MDIC. HEALTH ECONOMICS AND PATIENT ACCESS CLINICAL SCIENCE DATA SCIENCE AND TECHNOLOGY - Case for Quality (CfQ) - Computational Modeling and Simulation (CM&S) - External Evidence Methods - Cybersecurity NATIONAL EVALUATION SYSTEM FOR HEALTH TECHNOLOGY COORDINATING CENTER (NESTcc)
  3. 3. Regulators Health Systems Patient Groups Clinician Groups PayersIndustry NESTcc NESTcc’s MISSION & VISION Mission To accelerate the development and translation of new and safe health technologies, leveraging Real-World Evidence (RWE), and innovative research. Vision To be the leading organization within the health technology and medical device ecosystem for conducting efficient and timely high-quality Real-World Evidence (RWE) studies throughout the Total Product Life Cycle (TPLC).
  4. 4. NESTcc DEVELOPMENT BEGAN IN 2012 4 201520162017 NESTcc envisioned as a voluntary data network of collaborators by Planning Board FDA awarded funding for NESTcc to Medical Device Innovation Consortium (MDIC) NESTcc Executive Director named and Governing Committee selected NESTcc Strategic and Operational Plan developed Initial NESTcc Data Network formed and testing initiated through Round 1 Test-Cases 2018 NESTcc Data Quality and Methods Subcommittees formed 2012 FDA proposed the development of a national system 20192022 Concept Building Capacity Network Development Utilization & Expansion Interim and Final Results from Round 1 and Round 2 Test-Cases NEST Version 1.0 is operational NESTcc fully launched and operational
  5. 5. MULTI-STAKEHOLDER GOVERNING COMMITTEE ADVISES NESTcc Trade Association Nominees NAOMI ARONSON Blue Cross Blue Shield Association (BCBSA) KATHLEEN BLAKE American Medical Association (AMA) MARK DEEM – MDMA Nominee The Foundry, LLC PAMELA GOLDBERG Medical Device Innovation Consortium (MDIC) BILL HANLON – ACLA Nominee LabCorp/Covance ADRIAN HERNANDEZ Governing Committee Vice Chair Duke Clinical Research Institute (DCRI) HARLAN KRUMHOLZ Yale University JENNIFER LURAY Research!America MICHELLE MCMURRY-HEATH – AdvaMed Nominee Governing Committee Chair Johnson & Johnson Medical Devices VANCE MOORE Governing Committee Treasurer Mercy Health JEFFREY SHUREN CDRH, FDA SHARON TERRY Genetic Alliance DIANE WURZBURGER – MITA Nominee GE Healthcare MARC BOUTIN National Health Council TAMARA SYREK JENSEN Center for Clinical Standards and Quality, CMS
  6. 6. FRAMEWORK STRATEGY TO ACHIEVE ESTABLISHED GOALS To achieve success, NESTcc has four strategic priority areas for 2019: 1 2 3 Establish NESTcc Governance Develop NESTcc’s Data Network Establish NESTcc’s Sustainability Ensure NESTcc Stakeholder Engagement Ensure NESTcc Stakeholder Engagement 4 Ensure NESTcc Stakeholder Engagement
  7. 7. Establishing the NESTcc Data Network
  8. 8. REAL-WORLD DATA & REAL-WORLD EVIDENCE Real-World Evidence (RWE) Insights gleaned from Real-World Data (RWD) that have the potential to inform and empower patients, accelerate medical device innovation, and improve health care outcomes. Real-World Data (RWD) Data generated in the routine course of care, rather than during pre-market research or clinical trials, that takes into account real patient experiences.
  9. 9. NESTcc has established relationships with 12 Network Collaborators to advance evaluation and use of high-quality RWD from various sources. Profiles of each Network Collaborator can be found on NESTcc’s website. ESTABLISHING THE NESTcc DATA NETWORK
  10. 10. BUILDING NESTcc’S DATA NETWORK NESTcc surveyed its Network Collaborators to determine current capabilities, gaps, and priority areas. 12 Duke University Health System • HealthCore • Lahey Clinic • Mayo Clinic • MDEpiNet • Mercy • NYC-CDRN • OneFlorida • PEDSnet • STAR • Vanderbilt University • Yale New Haven Health System Network Collaborators represent Hospitals 195 Outpatient Clinics Network Collaborators report regular data refreshes 4 Quarterly 3 Mixed Rates 3 Monthly 2 Daily Most cited expertise  Cardiovascular and Cardiac Surgery  Women’s Health  Neurosurgery  Gastroenterology  Orthopedic Patient Records 494M+* Patient data represents Network Collaborators Common data models  I2b2  OMOP  PCORnet  Sentinel *Does not account for duplicate records Numbers reflect data as of February 2018
  11. 11. The collaborators comprising the NESTcc Data Network have access to a range of available data sources, including those listed below. ACCESS TO A RANGE OF RWD SOURCES EHR Public Claims Pharmacies Registries* Private Claims Patient-Generated Data *Registries Include (but are not limited to): • Anesthesia Quality Institute’s National Anesthesia Clinical Outcomes • Cardiac Catheterization • Cardiogenic Shock • Immunization • Implant registries • Integrated tumor • International Consortium Lower-GI • American College of Surgeons National Surgical Quality Improvement Program • Oncology • Pediatric Cardiomyopathy • Prostate Ablation‐Related Energy Devices • Robotic Surgery • Society of Thoracic Surgeons National Database • Society for Vascular Surgery • Thalassemia Clinical Research Network - Thalassemia Registry • Vital Records (Birth and Death) UDI 0 12# collaborators 0 12# collaborators 0 12# collaborators 0 12# collaborators 0 12# collaborators 0 12# collaborators 0 12# collaborators incorporating fully or demonstrated capability Available Data Sources UDI Implementation
  12. 12. Utilizing the NESTcc Data Network
  14. 14. TWO ROUNDS OF TEST-CASES ARE UNDERWAY The following process outlines the Test-Case process: Organization submits question Network Collaborators opt-in NESTcc matches manufacturer and collaborators Project is executed Lessons and gaps are sent to NESTcc Collaborators submit project short forms Collaborators submit full project proposal NESTcc awards funding Call for submissions posted 1. FDA and MDIC staff review 2. Preliminary Selection Team approval 1. Final Selection Team project-level approval 2. GC slate approval 3. MDIC Board slate approval 4. FDA project-level approval ROUND 1 & 2 ARE HERE START HERE
  15. 15. SELECTED TEST-CASES SUPPORT THE TOTAL PRODUCT LIFE CYCLE NESTcc Test-Cases are intended to explore the Network Collaborators’ ability to capture the data needed to support a range of studies and analyses. NESTcc Test-Cases Advance Across the Medical Device Total Product Life Cycle (TPLC) Pre- Market Label Expansion Post- Market Surveillance Coverage
  16. 16. NESTcc TEST-CASES ADDRESS A RANGE OF DEVICE QUESTIONS NESTcc’s Round 1 Test-Cases span a wide range of devices classes, regulatory pathways, TPLC stages, data sources, and disease areas. Device Classes Class I Class II Class III Regulatory Pathway 510(k) PMA TPLC Alignment Pre-Market Label Expansion Post-Market Active Surveillance Data Sources Claims Electronic Health Records (EHRs) Registries Disease Area Cardiology Dermatology Orthopedics Surgery Vascular
  17. 17. Eight Test-Cases were selected in the initial round of Test-Cases: NESTcc ROUND 1 TEST-CASES TOTAL-PRODUCT LIFE CYCLE (TPLC) ALIGNMENT REGULATORY PATHWAY PRODUCT(S) PARTICIPATING NETWORK COLLABORATORS (n) DISEASE AREA Pre-market Submission 510(k) Wound Closure Devices (topical skin adhesives, staples, sutures) 2 Dermatology Label Expansion PMA Endovascular stent 3 Vascular Label Expansion PMA Catheters used in Rx of Cardiac Arrhythmias 3 Cardiology Label Expansion PMA Mechanical Aortic Heart Valves 2 Cardiology Label from General to Specific Indication 510(k) Microwave Ablation Device 4 Surgery Post-market Surveillance 510(k) Total Knee Arthroplasty 2 Orthopedics Post-market Surveillance 510(k) Craniomaxillofacial Bone Distractors 1 Orthopedics Post-market Surveillance 510(k) Intervertebral Lumbar Body Fusion Devices 2 Orthopedics
  18. 18. ORTHOPEDIC TEST-CASE – TOTAL KNEE ARTHROPLASTY Project Title Testing the Feasibility of Registry and Claims Data Linkages Data Sources Electronic Health Records, Public Claims, Private Claims, Pharmacies, Registries Project Duration 12 months Disease Area Orthopedic Technology of Interest Total Joint Arthroplasty (TJA); Primary Total Knee Arthroplasty Project Aims • This project aims to conduct anonymous data linkages of Registry data with Real-World Data (RWD) sources, including private claims databases within the NESTcc Network Collaborators, from Mayo Clinic (Optum Labs) and HealthCore. • The specific aim is to conduct a descriptive study to evaluate the clinical outcomes of total knee arthroplasty (TKA) implant survivorship, mortality, revision/reoperation, readmission, and Emergency Department visit following TKA 2012-2017 for private health plan members. Pre- Market Label Expansion Post-Market Participating Network Collaborators Surveillance Coverage TotalProductLifeCycle(TPLC)Alignment Regulatory Pathway 510(k)
  19. 19. ORTHOPEDIC/ENT TEST-CASE – CRANIOMAXILLOFACIAL DISTRACTORS Project Title Developing Capacity to Conduct Proactive Post Marketing Safety Surveillance of Craniomaxillofacial Distractors Using Electronic Health Record Data Data Sources Electronic Health Records, Registries Project Duration 9 months Disease Area Orthopedic/ENT Technology of Interest Craniomaxillofacial Distractors Project Aims • This test-case will assess the feasibility of using Real-World Data (RWD) captured through the NESTcc Data Network to conduct proactive post-market surveillance for safety with devices used in pediatric populations. • This test-case seeks to determine the feasibility of using RWD captured through PEDSnet, a NESTcc Data Network Collaborator, to conduct proactive post-market surveillance for safety and effectiveness for CMF distractors. Pre- Market Label Expansion Participating Network Collaborators Surveillance Coverage TotalProductLifeCycle(TPLC)Alignment Regulatory Pathway 510(k) Post-Market
  20. 20. ORTHOPEDIC TEST-CASE – INTERVERTEBRAL LUMBAR BODY FUSION DEVICES Project Title Developing Capacity to Conduct Proactive Post Marketing Safety Surveillance of Intervertebral Body Fusion Devices Using Electronic Health Record Data Data Sources Electronic Health Records, Registries Project Duration 9 months Disease Area Orthopedic Technology of Interest Intervertebral Body Fusion Devices Project Aims • This test-case will assess the feasibility of using Real-World Data (RWD) captured through the NESTcc Data Network to conduct proactive post-market surveillance for safety for class II medical devices. • Specifically, this test-case will conduct proactive post-market surveillance for safety and effectiveness of lumbar interbody systems captured within Lahey Hospital and Medical Center (Lahey), a NESTcc Data Network Collaborator. Pre- Market Label Expansion Participating Network Collaborators Surveillance Coverage TotalProductLifeCycle(TPLC)Alignment Regulatory Pathway 510(k) Post-Market
  21. 21. ROUND 2 TEST-CASES SHOWED AN INCREASE IN SUBMISSIONS The second round of Test-Cases, announced in June 2019, included a more diverse set of concept submissions from multiple stakeholders. Announcements Submissions • Round 2 included the first targeted Test-Case announcement and multiple announcements posted together. • First announcement to specifically target patient-generated data (PGD). • Round 2 received more than 4 times as many submissions as Round 1. • 40 submissions were received in Round 2 from 26 organizations. • Submissions were received from a diverse set of organizations—health systems, government entities, non- profits including patient advocacy organizations, and industry. • More than twice as many industry groups submitted concepts in Round 2 compared to Round 1.
  22. 22. NESTcc TEST-CASE WEBSITE DESCRIPTIONS Round 1 and Round 2 Test-Cases are outlined on NESTcc’s Test-Case webpage, including information on project titles, duration, and abstracts, as well as technology of interest, disease area, and participating network collaborators.
  23. 23. LAUNCHING ACTIVE SURVEILLANCE ACTIVITIES NESTcc received $3m in targeted funding from FDA and formed a Task Force which will establish a Roadmap for advancing NESTcc’s active surveillance work • Multi-stakeholder Task Force announced late 2018. • Roadmap under development and issued for public commitment in Fall 2019. Task Force Members Name Perspective Institution Kathy Blake NESTcc Governing Committee/Providers American Medical Association Owen Faris FDA FDA Kevin Haynes Network Collaborators/Payers HealthCore Harlan Krumholz NESTcc Governing Committee/Network Collaborators Yale Brad Malin Network Collaborators/Privacy Vanderbilt Michelle McMurry- Heath NESTcc Governing Committee/Industry Johnson & Johnson Bray Patrick-Lake Patients DCRI Fred Resnic Network Collaborators/Integrated Health System Lahey
  24. 24. The NESTcc Active Surveillance Roadmap will be developed to lay out the high- level foundation for Version 1.0 of Active Surveillance activities. Items will include: o Initial users (FDA and medical device manufacturers) o Products and services (signal detection and signal discernment) o User experience o Infrastructure and operations o Data quality and methodology aspects o Future directions (future users, products and services) NESTcc ACTIVE SURVEILLANCE ROADMAP The Task Force is working on a Roadmap that will undergo public comment.
  25. 25. LAUNCHING NEST 1.0
  26. 26. 1. ENGAGE 2. LAUNCH 3. EXECUTE 4. COMPLETE Engage with NESTcc to develop a project and gain access to: Data Network Assets Pricing Structures Terms and Conditions Launch through collaborations with identified Network Collaborators: Execute required agreements Communicate with the FDA point of contact Execute the project through collaboration with the project team of Network Collaborators while engaging with NESTcc to ensure project progress Complete the engagement with NESTcc through the receipt of the final report, while participating in: Publications and dissemination opportunities Engaging directly with regulators and coverage providers for product-specific discussions and submissions NEST IS PREPARING FOR A PUBLIC LAUNCH By the end of 2019, NESTcc will be operationally capable of intaking unsolicited projects from external stakeholders to utilize the capabilities of the NESTcc Data Network.
  27. 27. Ensuring High-Quality Data & Analysis Methods
  28. 28. ADVANCING DATA QUALITY & METHODS In 2018, NESTcc established multi-stakeholder subcommittees to support its efforts to conduct real-world evidence studies for medical devices, leveraging ongoing initiatives including expertise from MDEpiNet, PCORnet, and Sentinel. DATA QUALITY SUBCOMMITTEE • Chaired by Dr. Lesley Curtis, Duke University School of Medicine • 12-person subcommittee includes representation from: • 6 health systems, including Network Collaborators • 3 medical device manufacturers • FDA • Chaired by Dr. Sharon-Lise Normand, Harvard Medical School • 9-person subcommittee includes representation from: • 3 health systems, including Network Collaborators • 4 medical device manufacturers • FDA METHODS SUBCOMMITTEE
  29. 29. DATA QUALITY SUBCOMMITTEE & FRAMEWORK Charge & Vision • Develop Data Quality Framework for NESTcc Network Collaborators • Design a process by which NESTcc Network Collaborators can demonstrate their aptitude with the NESTcc Data Quality Framework • Develop first, simple, pragmatic, iteration of NESTcc Data Quality Framework that will apply to a “first wave” of NESTcc Network Collaborators Data Quality Framework Overview • Initial version lays out the foundation for the capture and use of high-quality data for post-market evaluation of medical devices • Grounded in the use of real-world data (RWD) gleaned from the clinical care setting and the electronic health record (EHR) • Data Quality Framework will evolve for a “second wave” of data vendors or similar collaborators with large de-identified datasets Framework Organization 1. Governance 2. Characteristics of Data 3. Data Capture & Transformation 4. Data Curation 5. NESTcc Data Quality Maturity Model
  30. 30. METHODS SUBCOMMITTEE & FRAMEWORK Framework Organization 1. Background: Disease, Available Therapies, and Device Risk 2. Device Description 3. Study Specific Objectives 4. Target Population and Patient Selection 5. Outcomes: Primary, Secondary, Procedural, and Device 6. Device Exposure 7. Study Design 7.1 Specific Design 7.2 Blinding (Masking) 7.3 Units of Randomization and Observation 7.4 Mechanism of Treatment Assignment 7.5 Other Covariates 8. Study Procedures 8.1 Patient Consent 8.2 Randomization 8.3 Protocol Deviation Handling 9. Required Sample Size 10. Study Registration 11. Monitoring Plan 12. Statistical Analysis Plan Charge & Vision • Develop a “living” Methods Protocol Framework for NESTcc addressing device-specific considerations in benefit/risk studies and safety signal detection. • Develop a research agenda identifying critical issues in Methods for device, imaging, and other diagnostic technologies studies across the TPLC • Consult on an ad hoc basis to NESTcc to ensure that NESTcc activities employ the most appropriate and rigorous methods of analysis Methods Framework Overview • Develop a methodological framework to include device-specific considerations • A single protocol is being utilized for both randomized trials and observational studies
  31. 31. LAUNCHING THE SUBCOMMITTEE FRAMEWORKS NESTcc Data Quality & Methods Subcommittee framework development will include a public comment period before the first iterations are released. July 2019 Version 1 of NESTcc Frameworks Released June 2019 Public Comment Period Closes May 2019 Public Comment Period Opens Implementation and Iteration NESTcc Frameworks Utilized to Inform Test- Cases and Active Surveillance
  32. 32. Engage with NESTcc
  33. 33. NESTcc QUARTERLY NEWSLETTER The NESTcc Quarterly Newsletter is distributed on the first Tuesday of each quarter. • The Newsletter is in response to feedback from NESTcc stakeholders for a high-level update on NESTcc activities. • The Newsletter contains upcoming dates and links to news items and publications from the previous quarter. • The newsletter is available to the public and can be subscribed to here.
  34. 34. CONNECT WITH NESTcc Explore opportunities to connect with NESTcc online with the following resources:
  35. 35. 35 @NESTccMedTech @RachelRRath