PCORnet: turning a dream into realityFrancis S Collins,1 Kathy L Hudson,1 Josephine P Briggs,2

Michael S Lauer3

Many modern physician specialists like tothink of their work as grounded in strongscience. Yet 5 years ago, a group of cardiol-ogists published their findings on thescience underlying over 2700 practicerecommendations issued by their specialtysocieties.1 Only 314 (or 11%) were basedon ‘level A’ evidence, that is, evidencebased on multiple well-done randomizedtrials. Nearly half of the recommendationswere based solely on ‘expert opinion.’Even more disconcerting is the fact thatdespite the activities of many researchers,the vast majority of ongoing clinical trialsare too small to provide evidence relevantto patients and clinicians.2 Robust trialsthat can support recommendationsgrounded in solid science are few and farbetween, in part because they have becometoo expensive and complicated to run.Our biomedical enterprise is conductingmany clinical trials, yet we may not begetting all that much for what we spend.3

No wonder that too often, patients andcaregivers seeking information on how bestto improve their health or the health oftheir loved ones find that biomedicine doesnot have answers for questions they ask.Too often, clinicians cannot tell patientswhich therapies are likely to work best forindividuals like them. Too often risks, bene-fits, and impact on quality of life are uncer-tain. Providing accurate answers based onthe highest levels of scientific evidence forthe majority of unresolved clinical questionsis a revolutionary dream shared by patients,providers, payers, health plans, researchers,and policy makers alike. PCORnet, theNational Patient-Centered Clinical ResearchNetwork, promises a transformativeplatform that will turn this revolutionarydream into reality.4 The Patient-CenteredOutcomes Research Institute (PCORI)vision begins with patient-centeredness,which is fundamental to PCORnet’s struc-ture and aspirations. This model promisesto engage patients intimately in the

prioritization, design, and conduct ofresearch, a collaboration that will helpresearchers develop more accurate andmeaningful information for patients, theircaregivers, and clinicians—information thatwill allow our healthcare systems to achievethe best possible clinical outcomes, and theoutcomes that matter most to patients.PCORnet aims to build a national

research network, linked by a common dataplatform and embedded in clinical caredelivery systems. This network will enablestudies, and in particular randomized trials,that have been impractical to conduct todate—and do so with economies of scale.For patients, participating in research willbe simplified: studies will be easier to locateand privacy protections will be consistent.Participants will be true partners in theresearch. For researchers, a network of elec-tronic medical records representing over100 million covered lives will makelarge-scale observational and interventionaltrials faster to launch, more representativeof diverse real world populations, andcapable of providing much-needed answersto comparative effectiveness research ques-tions with greater accuracy.Just a few examples of the kinds of

questions that can be asked and answeredusing the PCORnet platform shouldexcite anyone interested in improvinghealthcare: What are the best manage-ment strategies for localized prostatecancer? Which of the available primarycare treatment strategies for children withattention deficit hyperactivity disorder aremost effective? What are the best treat-ment strategies for low back pain? Whichinterventions are most effective for redu-cing disparities in hypertension outcomes?For years, many of us have dreamed

about what would be possible if we hadthe research infrastructure to conductlarge cohort studies to understand genetic,behavioral, social, and environmentalfactors that contribute to health andillness,5 and to implement large rando-mized trials at affordable cost, to drivecontinuous improvement in the standardsof best clinical care. PCORnet can providethese capabilities, and do so in a realworld setting. As a result, clinicians andhealthcare systems will benefit from animproved evidence base for their practicesand recommendations, getting the mosteffective therapies into the hands of

patients and improving healthcare deliv-ery. Furthermore, the timetable for trans-lation of research results into improvedcare can be significantly shortened sincethe network carrying out the research isactually responsible for the care of almostone third of Americans.

Under the leadership of PCORI’sExecutive Director, Joe Selby and thePCORI Board of Governors, on which oneof us (FSC) serves, the PCORnet initiativehas been launched. PCORI designed theinitiative’s Phase I details and issued PCORIfunding announcements for establishing thecoordinating center, clinical data researchnetworks (CDRNs), and patient-poweredresearch networks (PPRNs). TheCoordinating Center, announced inSeptember 2013, worked quickly to kickstart planning with the PCORnet SteeringCommittee, on which two of us ( JPB andKLH) participate, setting the stage for thearrival of the CDRNs and PPRNs. Diversepanels of reviewers carefully evaluated 28CDRN and 61 PPRN applications, recom-mending 11 CDRNs and 18 PPRNs forfunding. The announcement of the CDRNsand PPRNs in December 2013 launched aflurry of activity to finalize this innovativenetwork’s governance and address funda-mental challenges to achieving a function-ing distributed research network within thenext 18 months.

There are still many challenges to bemet in order for PCORnet to reach itsambitious goal of launching a simple prag-matic clinical trial by September 2015.PCORnet is establishing the fundamentaldata architecture and data standards (eg,the adoption of a data model) in a mannerthat will facilitate rapid implementationwhile leaving room for other approachesto grow and mature. Implementation willrequire incorporation of patient-generatedoutcomes, interoperable methods to querythe electronic medical record, andcommon standards for biospecimens.Simultaneously, PCORnet is addressingkey policy questions concerning theresponsible conduct of research in this newenvironment, including informed consent,the use of central institutional reviewboards, and the protection of patientprivacy. By addressing these and othermajor challenges, PCORnet has the poten-tial to improve the conduct of, and provideguidance for, large pragmatic trials con-ducted within PCORnet and beyond.

Then the real work begins: conductingresearch. Building on the work of thePCORI Methodology Committee,6 onwhich one of us (MSL) sits, the research con-ducted through PCORnet will strengthenthe research community’s understanding of,

1National Institutes of Health, Bethesda, Maryland,USA; 2National Center for Complementary andAlternative Medicine, National Institutes of Health,Bethesda, Maryland, USA; 3National Heart, Lung andBlood Institute, National Institutes of Health, Bethesda,Maryland, USA

Correspondence to Dr Kathy L Hudson, NationalInstitutes of Health, 9000 Rockville Pike, Bethesda,MD 20892, USA; kathy.hudson@nih.gov

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and capacity for, patient-centered research,including a heavy emphasis on randomizedtrials that focus on patient-centered out-comes. The network will be faced with thechallenging yet exciting prospect of identify-ing the highest priority projects for thePCORnet platform to take on. Other orga-nizations will be invited into the tent andresearchers not directly affiliated withPCORnet will be able to conduct researchin the network through collaborations.Ultimately, if this platform is able to demon-strate its value and revolutionary potential,PCORnet will face the test of sustainability:it will need to establish partners that wish touse this platform for research, and developself-sustaining sources of support in acomplex and challenging budgetary envir-onment. Speaking as one such potentialpartner, the National Institutes of Health iseager to support studies that will be con-ducted in PCORnet and to develop initia-tives that we hope will be among the first tomake use of this important resource.

PCORnet holds the promise to trans-form clinical research—but many chal-lenges lie ahead. For ultimate success, allthose involved in shaping this revolution-ary dream must maintain the bold andvisionary attitude that enabled its creation.This is not your father’s clinical trialnetwork.

Acknowledgements The authors express sinceregratitude to Gwynne Jenkins for assistance inpreparation of this manuscript.

Contributors All authors contributed sufficiently.

Competing interests None.

Provenance and peer review Commissioned;internally peer reviewed.

Open Access This is an Open Access articledistributed in accordance with the Creative CommonsAttribution Non Commercial (CC BY-NC 3.0) license,which permits others to distribute, remix, adapt, buildupon this work non-commercially, and license theirderivative works on different terms, provided theoriginal work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/3.0/

To cite Collins FS, Hudson KL, Briggs JP, et al. J AmMed Inform Assoc 2014;21:576–577.

Accepted 8 April 2014

Published Online First 12 May 2014

▸ http://dx.doi.org/10.1136/amiajnl-2014-002747▸ http://dx.doi.org/10.1136/amiajnl-2014-002758▸ http://dx.doi.org/10.1136/amiajnl-2014-002764▸ http://dx.doi.org/10.1136/amiajnl-2014-002744▸ http://dx.doi.org/10.1136/amiajnl-2014-002746▸ http://dx.doi.org/10.1136/amiajnl-2014-002743▸ http://dx.doi.org/10.1136/amiajnl-2014-002827▸ http://dx.doi.org/10.1136/amiajnl-2014-002740▸ http://dx.doi.org/10.1136/amiajnl-2014-002727▸ http://dx.doi.org/10.1136/amiajnl-2014-002751▸ http://dx.doi.org/10.1136/amiajnl-2014-002745▸ http://dx.doi.org/10.1136/amiajnl-2014-002759▸ http://dx.doi.org/10.1136/amiajnl-2014-002756

J Am Med Inform Assoc 2014;21:576–577.doi:10.1136/amiajnl-2014-002864

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2 Califf R, Zarin D, Kramer J, et al. Characteristics ofclinical trials registered in ClinicalTrials.gov, 2007–2010. JAMA 2012;307:1838–47.

3 Devereaux P, Yusuf S. When it comes to trials, do weget what we pay for? NEJM 2013;369:1962–3.

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