the cornerstone of pcori’s vision for big, real-world … · the cornerstone of pcori’s vision...
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The Cornerstone of PCORI’s Vision for Big, Real-World Data Research
Maryan Zirkle, MD, MS, MA Program Officer, CER Methods and Infrastructure
Internet2 Global Summit 2015
• An independent research insDtute authorized by Congress in 2010 and governed by a 21-‐member Board of Governors represenDng the enDre healthcare community
• Funds comparaDve clinical effecDveness research (CER) that engages paDents and other stakeholders throughout the research process
• Seeks answers to real-‐world quesDons about what works best for paDents based on their circumstances and concerns
About PCORI
PCORI’s Mission and Strategic Goals
PCORI helps people make informed healthcare decisions, and improves healthcare delivery and outcomes, by producing and promoDng high-‐integrity, evidence-‐based informaDon that comes from research guided by pa8ents, caregivers, and the broader healthcare community. Our Strategic Goals:
Increase quanDty, quality, and Dmeliness of useful, trustworthy research informaDon available to support health decisions
Speed the implementaDon and use of paDent-‐centered outcomes research evidence Influence research funded by others to be more paDent-‐centered
Why Did We Establish PCORnet?
• NaDonal clinical research system is well-‐intenDoned but flawed
• We’re not generaDng the evidence needed by paDents and clinicians as they make decisions about their healthcare
• Health outcomes and dispariDes are not improving
• Our current system is great except: ― Too slow, too expensive, and not reliable ― Doesn’t answer quesDons that ma[er most to paDents ― Una[racDve to clinicians & administrators ― Doesn’t harness the true poten)al of health data
Virtuous cycle of a learning healthcare system: Real World DATA as the backbone
Our Vision for PCORnet
PCORnet will enable rapid, large-scale, patient-centered clinical research in real-world care delivery systems and communities.
“Research Infrastructure Done Differently”
7
SCALE:
Na8onal Infrastructure
for CER
SIZE: Includes large, diverse populaDons from real-‐world care se_ngs
STRUCTURE: Engages paDents &
clinicians, systems in all aspects of research SCOPE:
Creates efficient & effecDve processes for all aspects of conducDng
research
Our Vision for PCORnet How is PCORnet different from other research networks?
Pivotal $100M Infrastructure
Coordina8ng Center Provides technical and logisDcal assistance under the direcDon of a steering commi[ee and PCORI staff
18 Pa8ent-‐Powered Research Networks PaDents with a single condiDon form a research network; $16.8 million awarded
11 Clinical Data Research Networks Health system-‐based networks, such as hospital systems; $76.8 million awarded
Pivotal $100M Infrastructure
18 Pa8ent-‐Powered Research Networks PaDents with a single condiDon form a research network; $16.8 million awarded
11 Clinical Data Research Networks Health system-‐based networks, such as hospital systems; $76.8 million awarded
Complimentary and synergistic
Lead Organiza8on (Network name)
Partnered Health Systems
PaDent Outcomes Research To Advance Learning (“PORTAL”)
1. Denver Health 2. Group Health CooperaDve 3. Group Health Research InsDtute 4. HealthPartners Research FoundaDon 5. KP Center for EffecDveness & Safety Research 6. KP Colorado (InsDtute for Health Research) 7. KP Georgia (Center for Health Research) 8. KP Hawaii (Center for Health Research) 9. KP Mid-‐AtlanDc States (Mid-‐AtlanDc Permanente Research InsDtute) 10. KP Northern California (Division of Research) 11. KP Northwest (Center for Health Research) 12. KP Southern California (Department of Research & EvaluaDon)
Louisiana Public Health InsDtute (“Louisiana Clinical Data Research Network – LACDRN”)
1. Greater New Orleans Health InformaDon Exchange (GNOHIE) 2. Louisiana State University 3. Louisiana Public Health InsDtute 4. Tulane University
Children’s Hospital of Philadelphia (“PEDSNet”)
1. Children’s Hospital of Philadelphia 2. CincinnaD Children’s Hospital Medical Center 3. Children’s Hospital Colorado 4. Nemours Children’s Health System 5. NaDonwide Children’s Hospital 6. St. Louis Children’s Hospital 7. Sea[le Children’s Hospital 8. Boston Children’s Hospital
PaTH: Towards a Learning Health System in the Mid-‐AtlanDc Region (“PaTH”)
University of Pi[sburgh/UPMC and UPMC Health Plan Penn State College of Medicine/Hershey Medical Center Temple University School of Medicine/Temple Health Johns Hopkins University/Johns Hopkins Health System and Johns Hopkins Health Care
Lead Organiza8on (Network name)
Partnered Health Systems
Harvard (“SCILHS”)
1. Boston Children’s Hospital 2. Partners (Mass General and Brigham and Women’s Hospital) 3. Wake Forest BapDst University Medical Center 4. CincinnaD Children’s Hospital 5. University of Texas Health Science Center 6. Columbia University 7. Morehouse School of Medicine 8. University of Mississippi Medical Center
Vanderbilt University (“Mid-‐South CDRN”)
1. Vanderbilt Health System 2. Vanderbilt Healthcare Affiliated Network (VHAN) 3. Greenway Medical Technologies
University of California San Diego (“pSCANNER”)
1. University of California Research eXchange (UC-‐ReX) network 2. VA InformaDcs and CompuDng Infrastructure (VINCI) 3. UC San Diego (UCSD), 4. VA Tennessee Valley Healthcare System
University of Kansas Medical Center (“Great Plains CollaboraDve”)
1. University of Kansas Medical Center 2. Children’s Mercy Hospital 3. University of Wisconsin-‐Madison 4. Medical College of Wisconsin 5. Marshfield Clinic 6. University of Health Sciences at San Antonio 7. University of Texas Southwestern Medical Center 8. University of Iowa Healthcare 9. University of Minnesota 10. University of Nebraska
Network name Lead Organiza8on/PI
Partnered Health Systems
NYC-‐CDRN Weill Medical College
1. New York-‐Presbyterian Hospital 2. Weill Cornell Medical College 3. Columbia University Medical School 4. Montefiore Medical Center 5. NYU Langone Medical Center 6. Mount Sinai Health System 7. Clinical Directors Network (FQHC)
Chicago Community Trust (“CAPriCORN”)
1. Loyola University 2. Northwestern Medicine 3. Northshore University Health System 4. University of Chicago 5. University of Illinois Hospital & Health Sciences System 6. Cook County Health and Hospital System 7. Alliance of Chicago’s Federally Qualified Health Centers 8. Hines VA 9. Jesse Brown VA 10. Lurie Children’s Hospital 11. Children’s Hospital of University of Illinois 12. Comer Children’s Hospital
AcceleraDng Data Value Across a NaDonal Community Health Center Network (“ADVANCE”)
1. OCHIN 2. Health Choice Network 3. Fenway Clinic
71 Participating Health Systems in 11 CDRNs
Organiza8on PI Condi8on Popula8on Size
Duke University Laura Schanberg Juvenille RheumaDc Disease 9000
ALD Connect, Inc Florian Eichler Adrenoleukodystrophy 3000
Phelan-‐McDermid Syndrome FoundaDon
Megan O’Boyle Phelan-‐McDermid Syndrome 737
Immune Deficiency FoundaDon
Kathleen Sullivan
Primary Immunodeficiency Diseases 1250
University of Pennsylvania
Peter Merkel VasculiDs 500 (Pilot)
Parent Project Muscular Dystrophy
Holly Peay Duchenne and Becker muscular dystrophy 4000
Arbor Research CollaboraDve for Health
Bruce Robinson Primary NephroDc Syndrome (Focal Segmental Glomerulosclerosis [FSGS], Minimal Change Disease [MCD], and Membranous Nephropathy [MN] MulDple Sclerosis
1250
Epilepsy FoundaDon Janice Beulow Aicardi Syndrome, Lennox-‐Gastaut Syndrome, Phelan-‐McDermid Syndrome, Hypothalamic Hamartoma, Dravet Syndrome, and Tuberous Sclerosis
1500
GeneDc Alliance, Inc Sharon Terry Alström syndrome , Dyskeratosis congenital, Gaucher disease, HepaDDs, Inflammatory breast cancer, Joubert syndrome, Klinefelter syndrome and associated condiDons, MetachromaDc leukodystrophy, Pseudoxanthoma elasDcum (PXE), Psoriasis
50-‐ 50,000
Organiza8on PI Condi8on Popula8on Size
University of California, San Francisco Mark Pletcher Cardiovascular Health 100,000
CincinnaD Children's Hospital Medical Center
Peter Margolis Pediatric Crohn's Disease and UlceraDve ColiDs
15,000
Crohn’s &ColiDs FoundaDon of America R. Balfour Sartor Inflammatory Bowel Disease (Crohn’s disease and ulceraDve coliDs)
30,000
Global Healthy Living FoundaDon Seth Ginsberg ArthriDs (rheumatoid arthriDs, spondyloarthriDs), musculoskeletal disorders (osteoporosis), and inflammatory condiDons (psoriasis)
50,000
American Sleep Apnea AssociaDon Susan Redline Sleep Apnea 50,000
COPD FoundaDon Richard Mularski COPD 50,000
Accelerated Cure Project for MulDple Sclerosis
Robert McBurney MulDple Sclerosis 20,000
University of South Florida Rebecca Sutphen Hereditary Breast and Ovarian Cancer (HBOC) 17,000
Massachuse[s General Hospital Andrew Nierenberg Major Depressive Disorder, Bipolar Disorder 50,000
80 Organizations Participating in 18 PPRNs
Clinical & TranslaDonal
Science Awardees
Health InformaDon Exchanges
Federally-‐Qualified Health Centers
Integrated Care Delivery
Systems
Academic Health Centers
PaDent-‐Powered Registries
Disease Advocacy Groups
Pharmacy Data
Vendors
Data from Payers
(e.g., CMS)
Diverse Contributors + Rich Data = High Potential
PCORnet will bring together the exper8se, popula8ons, resources, and data of its par8cipa8ng organiza8ons to create a na8onal infrastructure that enables more efficient, pa8ent-‐centered research.
Hallmarks of PCORnet’s success will include:
• Highly engaged paDents, clinicians, health systems, researchers and other partners
• A collabora8ve community supported by robust governance
• Analysis-‐ready standardized data with strong privacy protecDons • Oversight that protects pa8ents, supports the Dmely conduct of research, and builds trust in the research enterprise
• Research that is sustainably integrated into care se_ngs and with communiDes of paDents
PCORnet Phase I Aims (April 2014-September 2015)
What makes data “Big?”
Volume: So much data, so li[le Dme
Velocity: AccumulaDng at incredibly fast rate
Variety: Different from source to source
Big Data Attributes & Relevance To Research
Other “V” words have emerged, per8nent to research
Veracity: Are the data true and correct?
Validity: Can we draw reliable conclusions?
Most important “V” may be… Value: Do the data produce something that paDents, clinicians and decision-‐makers want and need?
• When it succeeds, PCORnet will be able to leverage:
― Health system informaDon (diagnoses, procedures, EHR, etc.) ― Mobile health data (FitBit, Ginger.io, symptom trackers) ― Biologic data from stored blood, saliva, Dssue ― Online paDent communiDes and social media
PCORnet as a Resource for Big Data
• Not turn-‐key or automa8c!
― Managing the volume & variety, while determining veracity and validity of data, AND transforming the data into research-‐ready form requires resources and experDse
• Privacy and security are paramount
– Partnering with paDents to devise data-‐sharing pracDces
• A data plazorm is needed to support PCORnet—the imperaDve is to ensure that the data has immediate uDlity in real-‐world healthcare.
• Through PCORnet, we will have ongoing paDent and clinician input about high priority research topics.
• Sustained engagement of paDents, clinicians, and health systems is the key differen8ator that will enable PCORnet research to provide answers to important healthcare quesDons quickly, efficiently, and at lower cost than previously possible.
Beyond the Big Data Capabilities
• Finding ways that the data can simultaneously support healthcare research and healthcare improvement
― Need to arDculate a value proposi8on that resonates with paDents, clinicians, healthcare leaders, policy-‐makers, employers, payers
• Leveraging myriad data sources that can help us a[ain greater precision in the absence of interoperability
• Using that greater precision to achieve the holy grail of healthcare—targeDng treatment to paDent’s unique traits and preferences (eventually!)
“Challenge-tunities” Inherent in the Use of Big Data for Research
With Many Advocates for Freeing the Data, Are We Near an Inflection Point?
Can a new research network with >150 partners a[ain maximum velocity in 18 months?
Chaos
Learning
Change Transforma8on
“Speed without a purpose is chaos. Velocity is speed toward a purpose.”
David Kenny, The Weather Company
• “PCORnet has created a new sense of collabora8on among the many partners in Chicago and beyond. Our faculty have become much more engaged with their colleagues at other sites and are realizing that studies previously unfeasible are now viable with much less Dme and effort…” (CDRN)
• “Broad surfacing of widely recognized, long-‐standing problems faced by cross-‐insDtuDon paDent informaDon sharing-‐-‐suggesDng that it may be truly possible to study millions of pa8ents at once through PCORnet’s infrastructure”. (PPRN)
• “Enabling us to accelerate engagement approaches and increased a[enDon at insDtuDonal level, including paDent and family parDcipaDon, and what it means to be a learning health system. (PPRN)
• “CreaDon of [our] CDRN and parDcipaDon in PCORnet has been fundamentally transforma8ve. It has sparked conversaDon surrounding collaboraDve research at an unprecedented level, and engaged health system CEOs, senior leadership, trustees in ongoing CDRN conversaDons.” (CDRN)
Feedback from all 29 Networks Suggests that We Can!
“PCORnet holds the promise to transform clinical research—but many challenges lie ahead. For ultimate success, all those involved in shaping this revolutionary dream must maintain the bold and visionary attitude that enabled its creation. This is not your father’s clinical trial network.”
PCORnet is Reality
Thank You
Maryan Zirkle MD, MS, MA Program Officer, CER Methods and Infrastructure
Contact me: [email protected]