1. PEDSnet: Advancing Multi-Institutional Applied Clinical Research
For more information see
pedsnet.org

2. Doesn’t answer questions that matter most to people
Our national pediatric clinical research system is well-intentioned but flawed
We are not generating the evidence we need to support the healthcare decisions that patients and their doctors have to make every day.
High percentage of decisions are not supported by evidence
Health outcomes and disparities are not improving
Children rely on “hand-me down” evidence
Current clinical research system faces several problems:
Let’s start by taking a look at our current clinical research system and how it is faring.
Health care in America is at an exciting precipice
Recent explosion in knowledge and innovation
Yet, at the same time, our traditional clinical research paradigm continues to hold us back
Too slow
Unsustainable surges in cost
Shortfalls in quality and outcomes – many of which aren’t answering the questions that matter most to people.
The result is a massive missed opportunity.
Currently, we are not generating the evidence we need to support the healthcare decisions that everyday people and their doctors have to make every day.
FDA deputy commissioner Robert Califf agrees - he described a new “ecosystem” for clinical research as key to supporting the efforts by FDA to “put patients first.” The traditional clinical trial enterprise, he advised, “ignores these forces at its peril.”  (
Doesn’t answer questions that matter most to people
Too slow
Too expensive

3. But what if we could improve?
What if we could decrease the time it takes to get clinical insights?
What if we could achieve significant cost savings over a traditional clinical study?
What if we could improve the conduct of our clinical research with a national evidence generation system?
Consider the elements of a traditional study that typically impede faster insights – starting with the fact that traditional clinical research typically focuses on a very narrow question. From there, build up the study network, and, after a very long period of time, we get the study answered. But then what happens? We immediately break down the network that we’ve spent so much time building until the next question comes along - at which point we must rebuild once again. It is a redundant system. What if we had an infrastructure that operated more efficiently?
Now let’s look at what it is exactly that makes the conduct clinical research so expensive. We can roughly estimate that one half of the cost of a traditional clinical study goes into things like monitoring that are not directly getting to the answer we seek. What if we could eliminate that wasted spend and allocate those dollars to directly the research question? Could we potentially double our insights?
Finally, consider that a major inhibitor to the quality of our nation’s research is the undisputable fact that the majority of our clinical studies only represent a narrow slice of the population that we care for – we cannot be sure that our results are applicable broadly across the U.S. population. But what if we had at our fingertips a trustworthy and diverse knowledge repository from health systems, people and partnerships to bring real-world people the answers they seek?
What if we could have at our fingertips trustworthy, high-quality data from health systems, people and partnerships to bring real-world people the answers they seek?

4. PEDSnet: A National Pediatric Learning Health System
Our mission is to conduct multi-institutional pediatric research that informs clinical care and contributes to a national learning health system that enhances health for all children.
Our 2018 vision is to be the premier multi-specialty national pediatric clinical network for conducting research in routine healthcare settings.

5. Founding Institutions
PEDSnet

6. Standardized to a Common Data Model
PEDSnet Data
Standardized to a Common Data Model
Analysis-ready for observational research
Can be used for recruitment clinical trials and prospective research studies

7. Underpinned by a Common Data Model
Same data are represented differently at different institutions (e.g., Type of Encounter)
Common Data Model
Ambulatory Visit (AV)
Emergency Department (ED)
ED Admit to Inpatient (EI)
Inpatient Hospital (IP)
Non-Acute Inst. Stay (IS)
Other Ambulatory (OA)
Other (OT)
Unknown (UN)
No Information (NI)
(null)
SITE 1
Social Work Visit
Allied Health
Office Visit
Nurse Visit
Procedure Visit
Employee Health
Vascular Lab
Sleep Study Visit
SITE 2
Office Visit
Specimen
Postpartum Visit
Clinical Support
Initial Prenatal
SITE 3
Home Care Visit
Office Visit
Therapy Visit
Orders Only
Cardiology Testing
Hospital Encounter
For example, the left side of this slide illustrates how data from three different sites might be represented
Three different ways to represent the same event
On the right, you can see how the Common Data Model standardizes these into a single language
In order to be able to trust results of an analysis, need to have consistent representations

8. Currently Available Data Domains
PEDSnet has the capacity to pull unstructured data from EHR notes
Procedures
Demographic
Condition
Medications
Encounters
Lab Results
Patient
Reported
Data
Health Plan
Claims
Vital
Status
To make the data from these 110 million people useful, we had to put it into a standard structure.
That’s why we created our Common Data Model.
We have data in a common format for all of these domains.
Data Domains in the CDM (Blue) and Domains That Can Be Added (Purple)

9. Administrative Efficiencies
Common data sharing agreement
Single IRB arrangement
Streamlined contracting

10. Relationship network Engagement Communications Social Resource
Governance
Relationship network
Engagement
Communications

11. PEDSnet Data: Jan 2009 – May 2016
Data are from the January 2016 data refresh.
Children seen recently = past 18 months using refresh data as time 0
Full Population – available for secondary data analysis
Seen Recently – potentially recruitable population

12. PEDSnet Patients
N = 5.3 million

13. Here’s how PEDSnet’s data network works for researchers
The Researcher sends a question to the PEDSnet Coordinating Center through the Front Door
The Coordinating Center converts the question into a query with an underlying executable code, and executes it on partner data marts
PEDSnet reviews the query results and provides a response, which is sent back through the Front Door to the Researcher
Front Door
Response
Researcher
Question
PEDSnet
Coordinating Center
Query

14. Example of an observational study: Short- and Long-term Effects of Antibiotics on Childhood Growth
Will evaluate whether antibiotics prescribed in early infancy may cause obesity during childhood, taking advantage of PCORnet’s expansive data network.
Objectives:
Assess the effects of different types, timing, and amount of antibiotic use in the first two years of life with BMI and obesity at ages 5 and 10 years, and growth trajectories to age 5 years
Evaluate effects within subgroups (racial/ethnic, pregnant mothers, etc.)
CHANGE to antibiotic

15. Example of a clinical trial: COMBINE
In children with moderate-severe Crohn’s, is low-dose oral methotrexate in combination with anti-TNF therapy more effective than anti-TNF monotherapy in inducing and maintaining steroid-free remission for up to two years?
PCORI funded
N=425 patients enrolled over 2 years
PEDSnet sites have about 700 patients/year who meet eligibility criteria
Outcomes include disease activity and patient-reported outcomes

16. PEDSnet Front Door is now open for your research!
The power of data is exploding all around us, and now, with PEDSnet, we have finally an infrastructure that is allowing clinical research to be conducted as it always should have been…
Uniting clinicians, people, and health systems with the massive amounts of data that are being generated from people in their everyday health encounters.
Driven by the real-world needs of our diverse population – not just a small sliver of the pie
All in a way that is more efficient and less expensive than a traditional research model
Today, we are inviting you to be a part of this transformative research process at the outset.
The Front Door is now open, and we look forward to seeing you on the other side.

17. Accessing PEDSnet Request a Feasibility or Descriptive Data Query
Request a Feasibility or Descriptive Data Query
Determine feasibility of using PEDSnet data for a particular research study
Seek PEDSnet Collaboration
Determine if a research concept or early proposal is suited to being developed in partnership with PEDSnet
Identify sponsors and partners in the network
Launching a PEDSnet Study
Methodological and Data Science reviews of proposals
Accessing PEDSnet administrative efficiencies
Develop administrative materials needed for grant submission
Offer contracting, IRB reliance, and data-related resources
Produce research-ready data sets

18. PEDSnet is a Clinical Data Research Network in PCORnet
Accelerating Data Value Across a National Community Health Center Network (ADVANCE)  Oregon Community Health Information Network (OCHIN)
Chicago Area Patient Centered Outcomes Research Network (CAPriCORN) The Chicago Community Trust
Greater Plains Collaborative (GPC)  University of Kansas Medical Center
Kaiser Permanente & Strategic Partners Patient Outcomes Research To Advance Learning (PORTAL) Network  Kaiser Foundation Research  Institute
Research Action for Health Network (REACHnet) Louisiana Public Health Institute (LPHI)
Mid-South CDRN Vanderbilt University
PEDSnet: A Pediatric Learning Health System  The Children’s Hospital of Philadelphia
New York City Clinical Data Research Network (NYC-CDRN) Weill Medical College of Cornell University
OneFlorida Clinical Data Research Network  University of Florida
Patient-Centered Network of Learning Health Systems (LHSNet)  Mayo Clinic
Patient-oriented SCAlable National Network for Effectiveness Research (pSCANNER)  University of California, San Diego (UCSD)
PaTH: Towards a Learning Health System University of Pittsburgh
Scalable Collaborative Infrastructure for a Learning Healthcare System (SCILHS)  Harvard University

19. PCORnet
135 health systems across the country with data on over 110 million patients

20. For more information visit pedsnet.org
Thank you!
For more information visit pedsnet.org