All posts by julie.schulman@duke.edu

PCORnet Community Rallies to Launch New Version of Common Data Model

cdmv3.1
Click Image to Enlarge

PCORnet, the National Patient-Centered Clinical Research Network, is pleased to announce the release of the newest iteration of its Common Data Model (CDM), CDM v3.1. Initially released in three deployments between 2014 and 2015, the CDM is a way of organizing PCORnet’s broad and diverse data into a standard structure to enable a more rapid response to research-related questions. Now on its fourth installment, the CDM continues to reflect the hard work and input of stakeholders across the spectrum of the PCORnet community; in fact, this latest iteration of the CDM was developed in just 11 weeks, achieving an aggressive timeline with key participation by its stakeholders.

“This quick turnaround reflects the PCORnet community’s engagement and cohesion in its efforts to furthering patient-centered research, and I am proud of us for achieving this rigorous CDM development timeline,” said Shelley Rusincovitch, project leader in Applied Informatics & Architecture, Duke Clinical and Translational Science Institute (CTSI). “Ultimately, it is this type of collaborative spirit that will help PCORnet achieve its goal of bringing real-world insights to patients with greater speed and agility.”

Recognition of the need for early stakeholder engagement has been critical to the efficient and trustworthy development of the CDM. To ensure all stakeholders had a voice, PCORnet has established four different venues for feedback—a CDM Modeling Working Group, CDM Stakeholder Sessions, a CDM Implementation Forum and CDM Interest Groups—all dedicated to developing and improving each iteration of the CDM.

Unlike major releases such as CDM versions 1.0, 2.0 and 3.0, CDM v3.1 is a minor release with incremental, yet important, improvements made over the 3.0 specification. The v3.1 minor release includes:

  • Four new fields (sexual orientation, gender identity, diagnosis origin, and medication prescribing unit)
  • An expanded “encounter types” value set to include observation stays and institutional professional consults
  • A collapsed value set of procedure terminologies so that current procedural terminology (CPT) and healthcare common procedure coding system (HCPCS) medical codes are grouped into a single category
  • Clarification around the expected number of digits for relational database management system (RDBMS) number formatting
  • Elimination of the date of death requirement field for death table
  • An enrollment table basis that now includes drug coverage

CDM v3.1 has been formally released and is publically available here; in addition, a lay version of the 3.0 model can be accessed here. This is an important milestone, and will be followed by development for implementation by PCORnet Clinical Data Research Networks (CDRNs). No immediate action needs to be taken by the PCORnet CDRNs and Patient Powered Research Networks (PPRNs), who will continue to use CDM v3.0 with the current cycle.

 

About PCORnet

PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s capacity to conduct clinical research by creating a large, highly representative network that directly involves patients in the development and execution of research. More information is available at www.pcornet.org.  

About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.

PCORI Annual Meeting Sheds Light on PCORnet’s Role in Furthering Patient-Centered Research

Participant morale was high at the Patient-Centered Outcomes Research Institute (PCORI) Annual Meeting, which took place November 17-19, 2016 at the Gaylord National Resort and Convention Center in National Harbor, Maryland. Over the course of three informative days, over 300 patients and patient advocates, 200 clinicians, 400 researchers and other key stakeholders from over 100 health systems gleaned valuable insights into PCORI’s dedication to furthering patient-centered research. Notable sessions on PCORI’s revolutionary and innovative initiative, the National Patient-Centered Clinical Research Network (PCORnet), included presentations by researchers and an update on progress-to-date.

Day one kicked off with an opening address by PCORI’s Executive Director, Joe Selby, before shifting gears into a moving address by Stephanie Buxhoeveden of PCORnet’s Multiple Sclerosis (MS) Patient Powered Research Network (PPRN), who detailed her unique experience as both a provider and a patient with MS. Stephanie stressed the importance of bridging the gap between patients, researchers and caregivers to accelerate real world outcomes.

“Having sat on both sides of the table as practitioner and patient, I can tell you with confidence that patients and caregivers have just as much valuable disease expertise, if not more, than doctors,” said Stephanie. “Via PCORnet’s MS PPRN, I am taking an active role in research and putting power in the hands of the people who should have had it all along—those who are living with the condition.”

You can read more about Stephanie’s experience in this blog, which she posted in anticipation of the conference.

PCORnet also took center stage on day two of the Annual Meeting during an afternoon session that spotlighted its progress and functionality.  Participants heard from PCORnet’s Engagement, Data, and Research Committees about advancements in data infrastructure (including the most recent iteration of the Common Data Model), the research resources currently available to start conducting high-impact research, and models of engagement for participants and clinicians. Enthusiasm was high from the panelists as they celebrated PCORnet’s progress to date and urged continued and frequent early engagement from all stakeholders in the clinical research process. See the slides from these presentations here.

Many were excited that PCORnet is now open for business with a robust data model and encouraged everyone in the room to participate and help inform better ways to use data.

Day three included an all-day session on “PCORnet in Action: The Journey Toward People-Driven Research Transformation.”

Joe Selby, Executive Director PCORI and Heather Holderness, Research Associate Oregon Health and Science University, Department of Family Medicine (ADVANCE CDRN)
Joe Selby, Executive Director PCORI and Heather Holderness, Research Associate
Oregon Health and Science University, Department of Family Medicine (ADVANCE CDRN)

Joe Selby kicked off the day: “PCORnet is the most ambitious visualization of a long-held dream that we can tap the power of computerized clinical data and our capacity to involve stakeholders in the research process to drive better outcomes,” he said. “What was once a visionary ‘holy grail’ has now become a core component of a national evidence generation infrastructure that is embedding itself in delivery systems and communities of patients.”

The first half of the day highlighted PCORnet research underway, including study updates on INVESTED, ADAPTABLE, the PCORnet Bariatric Study, Healthy Mind, Healthy You, and Identifying and Predicting Patients with Preventable High Utilization.

The second part of the day included a poster session that included 16 PCORnet study posters.  Search and see all posters here.

Finally, the day wrapped up with a look at how to access PCORnet resources, including sessions about data characterization, SMART IRB, engagement best practices from demonstration studies and plans for the upcoming PCORnet Commons. You can access all PCORnet slides for day three here.

Many thanks to all who participated in this exciting event on behalf of PCORnet and contributed to its success. If you were unable to attend the PCORI Annual Meeting in person, you can still watch archived webcasts of plenary sessions, and blog posts from meeting presenters, including Stephanie Buxhoeveden and Megan O’Boyle from the Phelan-McDermid Syndrome Foundation here.

 

About PCORnet

PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s capacity to conduct clinical research by creating a large, highly representative network that directly involves patients in the development and execution of research. More information is available at www.pcornet.org.

About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.

Guest Blogs Generate Buzz in Advance of PCORI Annual Meeting

The Patient-Centered Outcomes Research Institute (PCORI) Annual Meeting on November 17-19, 2016 is approaching quickly, and two of its scheduled speakers are building momentum for the event with the publication of guest blogs detailing their clinical research experience. Although both blogs offer a unique perspective, both describe their authors’ personal passion to further PCORI’s mission to build and enhance patient-centered research. 

The first blog is authored by Megan O’Boyle, whose 16-year-old daughter suffers from rare disease called Phelan-McDermid Syndrome (PMS). An active member of PCORnet’s Phelan-McDermid Syndrome Data PPRN, Megan knows all too well how lack of real-world data access can stall progress toward rare disease therapies – and how PCORnet is positioned to reverse this trend. Read her recent blog in anticipation of the event here.

The second blog was written by Stephanie Buxhoeveden, a Multiple Sclerosis (MS) patient and a neurology nurse practitioner specializing in MS. Stephanie will be speaking at the PCORI Annual Meeting about her experience furthering patient-centered research alongside PCORnet’s MS PPRN, iConquerMS. Click here to read her PCORI Annual Meeting pre-event blog in full.

Click here to see the PCORI Annual Meeting’s agenda in full. Can’t make the event in person? Follow us on Twitter for live tweets during the meeting that will keep you up-to-date on highlights and available webcasts.

 

About PCORnet

PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s capacity to conduct clinical research by creating a large, highly representative network that directly involves patients in the development and execution of research. More information is available at www.pcornet.org.

About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.

PCORnet to Take Center Stage at PCORI Annual Meeting

The Patient-Centered Outcomes Research Institute (PCORI) Annual Meeting is planned for November 17-19, 2016, and enthusiasm is high for this prime opportunity to showcase the National Patient-Centered Clinical Research Network, PCORnet, as one of PCORI’s most innovative, data-driven initiatives to transform clinical research. Held at the Gaylord National Resort and Convention Center in National Harbor, Maryland, the meeting is a must-attend event for thought leaders, researchers, patients, family caregivers, clinicians, and key stakeholders who are eager to learn about important trends in patient-centered research and promising progress in PCORI-funded studies.

PCORnet will be in the spotlight during several sessions at the Annual Meeting. The first will be during the meeting’s opening plenary session on Thursday, November 17, which is focused on how to make patient needs and values central to health research and decision-making. Neely Williams, MDiv, patient co-investigator for PCORnet Mid-South Clinical Data Research Network (CDRN) and the patient co-principal investigator for the PCORnet Bariatric Study will participate in a panel discussion about the power of PCORnet to change the research conversation and improve outcomes for underrepresented populations. Watch Neely share her powerful story in the video below and read her recent opinion editorial published in The Tennessean.

On Friday, November 18, PCORnet Engagement, Data, and Research Committee leadership and members will participate in a panel discussion that highlights advancements in enhancing public trust, models of engagement for participants and clinicians, current research underway utilizing the network, and advancements in the data infrastructure.

Finally, attendees will have an opportunity to see “PCORnet in Action: The Journey Toward People-Driven Research Transformation” at a workshop session on Saturday, November 19 from 10:30 a.m. to 4 p.m. ET. The morning portion of the session will highlight current research underway within the Network, while the afternoon portion of the session will focus on innovative tools and resources available within PCORnet to external investigators. It will also feature an interactive poster session where attendees can learn more about PCORnet’s demonstration studies, including the research objectives for these studies and what they are illustrating about PCORnet’s power to conduct efficient, trustworthy clinical research.

Click here to see the agenda in full. Can’t make the event in person? Follow us on Twitter for live tweets during the meeting that will keep you up-to-date on highlights and available webcasts.

 

About PCORnet

PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s capacity to conduct clinical research by creating a large, highly representative network that directly involves patients in the development and execution of research. More information is available at www.pcornet.org.

About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.

PCORnet Offers a First Glimpse of the Nation’s Largest Patient-Centered Clinical Research Network

* Article update:  As of May 2, 2017, PCORnet released a refreshed view of its data that sheds light on more recent patient encounters than was previously available and includes an expanded list of conditions of interest.


The National Patient-Centered Clinical Research Network, PCORnet, today released early data on its patient demographics and conditions of interest, offering the public a first glimpse of the much-anticipated research network. Founded in 2014 as an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI), PCORnet aims to speed the pace and quality of clinical research in the United States.

“We are thrilled to publicly share a first look at the research scope of PCORnet, a network that promises to transform the landscape of clinical research through large amounts of health data and patient partnerships,” said Rachael Fleurence, PhD, Chair, PCORnet Executive Committee, and PCORI, Program Director. “The achievement of this major milestone is the result of countless individuals who have all shared in our dedication to unite people, clinicians, and health systems in a way that drives faster, real-world insights to help people make informed healthcare decisions.”

PCORnet represents millions of Americans across more than 100 healthcare organizations. The demographic summary released today includes data on up to 41 million individuals who received care at a PCORnet network partner in 2014, stratified by their sex, race, and age[i]. It also details the number of individuals who experienced a medical encounter in 2014 in which they were diagnosed with one of nine conditions of interest based on diagnosis codes[ii] for respiratory conditions, selected malignancies, myocardial infarction, stroke, rheumatoid arthritis, ulcerative colitis, hypertension, renal disease, and influenza/pneumonia, also stratified by sex, race, and age.

As early as spring 2017, researchers will be able to partner with PCORnet to query health records and other data from millions of individuals represented in PCORnet’s Common Data Model (CDM). Momentum and interest continue to build as PCORnet shares this first round of data in preparation for its public launch.

“The enthusiasm for PCORnet underscores the pressing need for a national evidence generation system that puts the interests of people and healthcare stakeholders at the forefront,” said Adrian Hernandez, MD, MHS, Co-Principal Investigator, PCORnet Coordinating Center. “Today’s public announcement of our initial network demographics and conditions is an important milestone that puts us one step closer to our goal of facilitating timely, meaningful answers to people’s everyday health questions.”

Trailblazing a new, people-centered approach to clinical research has been a massive undertaking. The PCORnet team began by successfully creating a Distributed Research Network that is comprised of Patient-Powered Research Networks (PPRNs) that are motivated to play an active role in clinical research and Clinical Data Research Networks (CDRNs) representing health organizations partnering to conduct research as a network. With a robust governance structure in place, PCORnet’s available health data is currently being standardized via the CDM for research use. PCORnet allows network partners to respond to research queries and return aggregate data to the PCORnet Coordinating Center for analysis to protect privacy first.

Now that the network has released its initial demographics and conditions, PCORnet is moving forward with its next major effort: the use of a PCORnet-wide single Institutional Review Board (IRB) approach to significantly reduce the time and expense of conducting multisite studies within the network. This effort will enable PCORnet institutions to use the NCATS Streamlined, Multisite, Accelerated Resources for Trials (SMART) IRB Reliance Platform from the National Institutes of Health instead of the traditional approach of using local IRBs for each research site. PCORnet is pilot-testing the SMART IRB approach in partnership with INVESTED, a PCORnet-designated study of high-dose influenza vaccine. This pilot and related evaluation will inform the adoption of a single IRB approach within PCORnet in 2017.

 

About PCORnet

PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s capacity to conduct clinical research by creating a large, highly representative network that directly involves patients in the development and execution of research. More information is available at www.pcornet.org.

About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.


[i] Some individuals may have visited more than one PCORnet network partner and would be counted more than once.

[ii] Diagnosis codes used by PCORnet to define the nine conditions of interest for its demographic summary include ICD-9-CM (International Classification of Diseases, Ninth Revision, Clinical Modification) and SNOMED CT (Systematized Nomenclature of Medicine – Clinical Terms).

PCORnet Partner Networks Share in Mission to Stamp Out Breast Cancer

October is National Breast Cancer Awareness Month, an annual campaign to raise awareness of breast cancer risks, the value of screening and early detection, and treatment options available. Although symbolic loops of pink ribbon put a spotlight on the disease every October, stamping out breast cancer is a year-round effort for the National Patient-Centered Clinical Research Network, PCORnet, which was founded in 2014 as an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). Here, we are highlighting two PCORnet partner networks—the American BRCA Outcomes and Utilization of Testing (ABOUT) network and the Greater Plains Collaborative (GPC)—that are not only committed to their respective organizational mission to combat this devastating disease, but also joining forces to do so via PCORnet’s new Cancer Collaborative Research Group (CRG).

ABOUT Co-PI urges Empowerment Through Genetic Counseling and Testing

ABOUT is a PCORnet Patient Powered Research Network (PPRN), or group of patients motivated to play an active role in research to further health outcomes. ABOUT’s members are specifically focused on genetic factors that cause an increased risk for breast, ovarian, prostate, pancreatic, and other cancers.

Sue Friedman, co-principal investigator for ABOUT and founder of Facing Our Risk of Cancer Empowered, Inc. (FORCE), recently published a moving blog on AstraZeneca’s website sharing her own journey as a breast cancer survivor that highlighted the genetic link between breast cancer and other hereditary cancers, such as ovarian cancer.

Sue encourages higher-risk women to “connect the hereditary dots” and empower themselves by speaking with experts knows as genetic counselors and considering genetic testing, even when the process may make us uneasy. Her message: “this information is too important to be left to chance.” 

GPC’s Breast Cancer Cohort Survey Puts Patient Focus Front and Center

GPC is a PCORnet Clinical Data Research Network (CDRN) of 12 leading medical centers in eight states committed to a shared vision of improving healthcare delivery through ongoing learning, adoption of evidence-based practices, and active research dissemination.

Dr. Betsy Chrischilles, director of the University of Iowa’s Health Effectiveness Research Center is currently leading breast cancer outcomes research studies within the GPC in close collaboration with Cheryl Jernigan, GPC’s lead patient advocate. Using the network’s tumor registries on stage 0-3 breast cancer patients, GPC has developed and administered a survey designed to learn from the experiences of GPC’s breast cancer patients.

“This was a true example of a research effort in which patients were engaged early on and had a key voice in determining questions of interest,” said Cheryl. “For example, while questions related to post-diagnosis quality of life were not a high priority for investigators, patients were eager to learn more, and so these questions were included based on that feedback. This is just one way PCORnet’s unique infrastructure puts the needs of people front and center to drive improved health outcomes.”

GPC’s survey yielded interesting insights that could inform patient care decisions: 10 research papers are currently in development documenting its results, and four of those abstracts have been accepted at the upcoming San Antonio Breast Cancer Symposium.

Cancer Research Partners in the White House Cancer Moonshot Program

In addition to their individual efforts, GPC and ABOUT are also partnering in the creation of PCORnet’s Cancer CRG currently in development as part of a commitment that PCORnet made to support the White House Cancer Moonshot. PCORnet’s Cancer CRG will aim to bring together teams of patients, clinicians, and investigators to prioritize our nation’s top cancer research questions, develop approaches for using real-world electronic data, and reduce disparities in cancer care and outcomes.

“United by PCORnet’s mission to drive efficient, people-centered research, I am proud that networks like GPC and ABOUT are changing the way we learn about and tackle breast cancer, an all-too-prevalent condition,” said Betsy.

For more information visit the GPC and ABOUT network websites. To get involved, join the ABOUT Network.

 

About PCORnet

PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s capacity to conduct clinical research by creating a large, highly representative network that directly involves patients in the development and execution of research. More information is available at www.pcornet.org.

About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.

PCORI Marks Milestones & Highlights PCORnet’s Progress

On PCORI’s sixth anniversary, Executive Director Joe V. Selby, MD, MPH, reflects on its record of achievement and growing influence in a PCORI Guest Blog. PCORI has now approved more than $1.5 billion to fund 551 comparative clinical effectiveness research and related projects. The past year has seen PCORI continue its stakeholder-guided shift to funding larger studies of specific conditions that impose a substantial health burden. And we’ve seen more and more examples of how PCORI’s commitment to engaging patients and other stakeholders in research is influencing others to take a similar approach.  PCORI is especially excited to see the progress being made by PCORnet, the National Patient-Centered Clinical Research Network, in harnessing large-scale health data and stakeholder partnerships to boost the pace and power of health research at lower cost than was previously possible. Read more about PCORnet’s progress in the PCORI Guest Blog.

PCORnet Colleagues Share Insights to Enhance Drug Review at FDA’s PDUFA Public Meeting

Dr. Adrian Hernandez and Sharon Terry, co-principal investigators, PCORnet Coordinating Center, attended the U.S. Food and Drug Administration (FDA) Prescription Drug User Fee Act (PDUFA) Reauthorization Public Meeting on August 15, 2016. In alignment with the meeting’s goal to solicit public feedback on the agreement forged between FDA and stakeholders to evolve the PDUFA program to make the drug review process more efficient, Hernandez and Terry voiced their support of the agreement’s efforts toward patient inclusion, real-world evidence use, and rare disease drug development. The latest reauthorization, which is the sixth iteration of PDUFA, will determine the FDA’s user fee policies from 2018 to 2022.

“With the current legislative authority for PDUFA expiring in September 2017, FDA is seizing the opportunity to revamp the PDUFA program to align with the future of drug development,” said Hernandez. “In my professional capacity at the National Patient-Centered Clinical Research Network (PCORnet) and Duke Clinical Research Institute (DCRI), and in my personal life as a father, brother, and son of family members who have conditions that could be improved with better real-world evidence use and the inclusion of patient voices in research, I was pleased to see that the evolved PDUFA agreement emphasized many of the areas we at PCORnet and the DCRI are passionate about furthering.”

PCORnet as a Tool for Transformation

Indeed, in her introductory address, Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research (CDER), highlighted PDUFA VI’s intent to enhance patient input and integrate it into regulatory decision making. Woodcock also noted that fit for purpose tools to collect meaningful patient input must to be addressed. With access to more than 145 million people’s health data, PCORnet is well positioned to serve as a key tool in this transformation toward a clinical research culture driven by the needs of patients. By tapping into electronic health records, insurance claims data, data reported directly by people, and other data sources, PCORnet offers researchers an innovative network underpinned with the infrastructure, tools, and policies to support rapid, efficient clinical research. In fact, it is the first network of its kind to involve patients as well as those who care for them in a substantive way. Patients are central to the governance structure of PCORnet and are also key in determining what research questions will be studied.

Facilitating the Patient Perspective in Drug Development

The meeting explored the FDA’s continued interest in patient-focused drug development, an aspect of PDUFA V that created a successful, albeit limited, program to ascertain the preferences and lived experiences of individuals affected by diseases and conditions. PDUFA VI will facilitate the systematic integration of the patient perspective into the drug development and regulatory review of innovative medicines.

“It is critical that we continue to adopt interventions based on community involvement, patient reported outcomes (PROs), and the preferences of those who know best—the very patients who will use the medications and products,” said Terry. “PCORnet is uniquely poised to help with this because of our focus on integrating clinicians, people, and their communities into all aspects of research.”

The PDUFA reauthorization also explores the need and ability to enhance the use of real-world evidence in regulatory decision-making, a focus area for which both Hernandez and Terry voiced strong support. For more information on their views, read PCORnet Leaders Participate in Policy Roundtable to Advance the Use of Real-World Evidence.

“Every moment, there are patient experiences occurring that are not being included in a formal way for evidence,” said Terry. “Patients expect that we will learn from what they go through to improve the experience for those who will walk in their shoes the next time, which unfortunately often does not happen.  We applaud that PDUFA VI is driving change toward a better approach through its emphasis on real-world evidence such as that being generated every day in PCORnet.”

Finally, the meeting addressed the need to promote rare disease drug development by continuing with PDUFA V’s Rare Disease Program (RDP) activities, as well as conducting outreach to sponsors and patient groups to promote best practices. This is a continued focus area for PCORnet, with one or more rare diseases as the focus of half of PCORnet’s Patient-Powered Research Networks. In addition, each of PCORnet’s Clinical Data Research Networks has selected a specific rare disease and is developing methods for identifying and characterizing it, as well as recruiting patients.

To learn more about the PDUFA Public Meeting, view the full webcast.

PCORI Announces Progress Toward PCORnet’s White House Cancer Moonshot Commitments

Moonshote coverThe Patient-Centered Outcomes Research Institute (PCORI) this week issued an update to the White House outlining progress made by its National Patient-Centered Clinical Research Network (PCORnet) toward its commitments to the Administration’s “Moonshot” initiative to accelerate cancer research. As the nation’s most innovative and robust patient-centered clinical research network, PCORnet offers cancer researchers a wealth of real-world insights to address unmet research needs and reduce disparities in cancer care and outcomes. PCORnet has experienced great momentum over the past several weeks, which is reflected in notable progress made toward its commitments pledged to White House Cancer Moonshot.

PCORnet’s first commitment is to enhance its highly representative national clinical research network to allow large-scale research, including cancer research, to be conducted with greater quality and efficiency.

In the 12 weeks since first pledging its Moonshot commitments, PCORnet has already grown its number of patients with a demographic record from 110 million to 145 million, substantially enriching its data pool to support observational studies as well as clinical trials. To ready the network for research, PCORnet’s developers are continuing to characterize its available data and to enhance quality. The team is also working with its partners to examine the data, explore questionable patterns, flag missing data, and determine its utility to answer specific research questions.

145 M Canserve

PCORnet’s second commitment is to create data tables that include descriptive characteristics of patient cohorts treated for the most common types of cancer (including breast, colorectal, prostate, lung, and melanoma) within PCORnet’s 130 healthcare systems.

This is a massive undertaking that requires a stepwise process, with the first step being to organize all of PCORnet’s available data into a standard structure called the Common Data Model (CDM) version 3.0. This activity is well underway, and the “DataMarts,” or repositories of structured data from PCORnet’s healthcare systems, have made meaningful progress toward transforming their electronic health records into the CDM to ready them for research use. In fact, 70 percent of PCORnet’s DataMarts have now met the technical criteria for structure in the CDM and have signed PCORnet’s data-sharing agreement, with the remaining DataMarts expected to follow suit within the next six months. With these data in a standard structure, PCORnet is actively building a “Table 1” to summarize the demographics of its population – including sex, race, age, and 10 conditions of interest – and to answer research questions.

Progress toward this commitment has also contributed to the arrival at an exciting milestone: PCORnet’s very first research query, known as the “weight cohort research query,” was sent on July 11, 2016 and results are expected back in August, 2016. The results of this query will provide detailed weight information across PCORnet’s network and inform its obesity demonstration projects. With PCORnet’s data nearly characterized and its first research queries already in motion to test the strength and readiness of the network, the team will soon be in a position to produce descriptive tables of patient cohorts with the common cancers mentioned above.

PCORnet’s final commitment is to build a Cancer Collaborative Research Group (CRG) affiliated with PCORnet that brings together teams of patients, clinicians, and investigators to 1) identify and prioritize our nation’s top cancer research questions; 2) develop approaches for using real-world electronic data to address unmet research needs in prevention, diagnosis, and treatment of cancer; and 3) reduce disparities in cancer care and outcomes.

PCORnet’s efforts to this commitment are well underway, with the details of its Cancer CRG concept submitted to PCORI’s executive committee on July 15, 2016 and the notice of interest process initiated. A PCORnet Cancer CRG Planning Committee has also been established. This committee is led by patients, clinicians and investigators from our Greater Plains Collaborative (GPC) Clinical Data Research Network (CDRN) focused on breast cancer, Kaiser Permanente & Strategic Partners Patient Outcomes Research to Advance Learning (PORTAL) CDRN focused on colorectal cancer, and the American BRCA Outcomes and Utilization of Testing (ABOUT) Patient-Powered Research Network (PPRN) focused on hereditary breast and ovarian cancer.

One of the noted primary objectives of the proposed Cancer CRG will be to rapidly enhance the current PCORnet CDM by including key cancer-specific data tables and structures supportive of the Administration’s Cancer Moonshot, including, but not limited to, tumor registry, cancer systemic therapy, germline mutations, and molecular marker tables. In addition, breast and colorectal cancer patients who were identified from electronic health records and merged registry data have already been surveyed to collect patient-reported data. This will be valuable when considering what cancer-specific data, including patient-reported data, should be added to the CDM.

To further solidify the Cancer CRG and its remit, the networks are currently in collaboration to finalize their proposal in preparation for PCORnet’s October 31, 2016 CRG application deadline. The final CRG recommendations will be presented to PCORI’s executive committee in November 2016 and in early December 2016, it is expected that the PCORnet Council to vote to approve and fund the Cancer CRG.

PCORnet will continue to pursue its three Moonshot commitments enthusiastically in the months ahead, with progress reports planned on a quarterly basis in 2017. Stay tuned for more updates on these goals as PCORI and PCORnet partner to advance cancer research and care.

PCORnet Advocate Calls for Patients to Have a Louder Voice in Medical Research

Neely Williams, patient co-investigator for PCORnet’s Mid-South Clinical Data Research Network, was featured in The Tennessean on Sunday, August 7, 2016 sharing her perspective on why patients need a louder voice in medical research. Read the full story here.

As an African American woman living in the Deep South who has battled obesity for most of her adult life, Williams knows all too well what it means to be part of an underrepresented community seeking answers to an under-researched disease. In the opinion editorial, Williams shares her story of living with obesity and seeking a bariatric surgery option that would deliver the best health outcomes for her, personally. What followed was a surgery that was not only minimally effective in the long-term, but also resulted in multiple subsequent surgeries. Williams shares her frustrating and unsettling experience of receiving medical advice based on research from a pool of individuals whose experience with obesity was vastly different from her own.

To help others have a better experience, Williams is a driving force behind PCORnet, the National Patient-Centered Clinical Research Network, an initiative that was launched with PCORI funding in 2013.  PCORnet is trailblazing a new approach to clinical research – one that invites all the voices in every community to join the chorus. Williams is currently the patient co-investigator for PCORnet’s bariatric study to glean insights on which procedure has the best results for specific patient communities. Williams believes that the work she is doing can build stronger collaborations among patients and doctors and clinicians of all types, and bring greater awareness into the community.

By shedding light on real-life experiences and bridging important knowledge gaps, studies like these are reducing the likelihood that tomorrow’s bariatric patients will have experiences like Neely’s. To learn more about Williams, her experience and her role in PCORnet’s bariatric study, watch her video.

Bariatric Surgery Study A Patient Advocacy Perspective