All posts by meagan.daly@duke.edu

Attending AMIA 2017? Here’s What You Need to Know

The American Medical Informatics Association (AMIA) is set to hold its biggest conference to date on November 4-8, 2017, in Washington, DC. Numerous PCORI and PCORnet colleagues will be there sharing leading-edge scientific research. AMIA’s 2017 Annual Symposium will host over 100 scientific sessions, presenting work spanning translational bioinformatics, clinical research informatics, public health informatics, and more. This year, the symposium revolves around the theme “Precision Informatics for Health: The Right Informatics for the Right Person at the Right Time,” which speaks to the heart of PCORnet’s people-centered mission.

Are you attending the meeting? Please support your PCORI and PCORnet colleagues by joining the sessions listed below. You can also download the schedule, follow and continue the conversations online @AMIAInformatics #AMIA2017.

Presenter What When
Hossein Estiri Applying unsupervised learning to characterize rare observations in clinical data: the DQe-p tool Monday, November 6: 10:30-10:48am
Gunston
Jeffrey Klann Web-Service-Enabled Apps for Research: SMART-on-FHIR for OMOP and PCORNet   Monday, November 6: 11:00-11:30am
Lincoln West
Keith Marsolo Laura Qualls
Bradley Hammill Jeffrey Brown
The PCORnet Learning Cycle Tuesday, November 7: 8:30-10:00am
International Ballroom West
Jon Puro Social and Behavioral Determinants of Health: Fundamental Informatics Challenges for Enriching Health IT Systems Tuesday, November 7: 1:45-3:15pm
Georgetown
Jeffrey Klan Reuse of PCORnet Data to Support the Precision Medicine Initiative: Data Model Harmonization Tuesday, November 7: 2:21 PM–2:39 PM
International Ballroom Center
Jiang Bian Implementing a Hash-based Privacy-Preserving Entity Resolution Tool in the OneFlorida Clinical Data Research Network Tuesday, November 7: 5:00-6:30pm  Columbia Hall
Casie Horgan Standardization of Prescribing Data in PCORnet: RxNorm Concept Unique Identifiers in Multi-Site Research Tuesday, November 7: 5:00-6:30pm  Columbia Hall
Levon Utidjian Developing Computable Phenotypes of Pediatric Chronic Conditions in PEDSnet Wednesday, November 8: 11:42-12:00pm
Jefferson East


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 Blog

Matching Patients to Clinical Trials for People Affected by Hereditary Cancers

Author: Sue Friedman, DVM, is Executive Director for Facing Our Risk of Cancer Empowered (FORCE) which is a lead organization in PCORnet’s ABOUT Patient Powered Research Network (PPRN).  Friedman is a Co-Principal Investigator for ABOUT.

It’s an exciting time for hereditary cancer research as the pace of new personalized medicine treatments and targeted therapies has increased. But for these new therapies to translate into better health, they must be tested on specific populations through clinical trials. Unfortunately, less than 5% of adults with cancer enroll in clinical trials and about a fifth of adult cancer clinical trials fail to recruit enough participants to complete the research.

Matching patients to clinical trials for prevention, detection and treatment is even more challenging for groups that are a subset of a larger disease population, such as people with, or at high risk for hereditary breast, ovarian, pancreatic, melanoma and prostate cancers (HBOC) such as those enrolled in the ABOUT Network.

ABOUT is a PPRN within PCORnet, the National Patient-Centered Clinical Research Network. PCORnet is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). ABOUT grew out of the work of a national nonprofit advocacy organization for individuals and families impacted by HBOC — Facing Our Risk of Cancer Empowered, Inc. (FORCE) — and a multiyear collaboration between FORCE and hereditary cancer researchers at the University of South Florida Morsani College of Medicine.

In 2015, FORCE, received a Celgene Innovative Impact Award to develop more HBOC patient-friendly resources to help our community learn about, and enroll in specific clinical trials. The project used the resources of ABOUT to engage in a pilot program that sought to improve clinical trial participation for hereditary breast and ovarian cancer by identifying patients’ information needs around clinical trials and developing patient-friendly education and clinical trial matching materials. The project had three key components: a survey, a custom research matching database and educational tools.

Community voice on research participation
The community engagement survey we designed for this project included over 1,100 cancer survivors and “previvors” (high-risk individuals who are survivors of a predisposition to cancer but who haven’t had the disease.) Not surprisingly, the results indicated that the technical language of clinical trials and the challenge for patients to determine whether they are eligible present substantial barriers. Many participants also expressed frustration that they did not get to learn the results of the research study in which they participated. Dissemination of research results is an active area of focus for FORCE and ABOUT and when we collaborate with researchers, we help them share their results back to the community.

Research study search tool
The project’s second component is our new and improved research study search tool. It searches two databases for studies enrolling people with HBOC: clinicaltrials.gov and FORCE’s own custom study database which is enriched with studies enrolling people with or at high risk for hereditary cancer. Users can look up studies by type:

  • Treatment
  • Prevention and detection
  • Risk assessment
  • Quality of life and wellbeing
  • Registries, surveys and interviews

Searches may also be sorted by the type of cancer, specific genetic mutations, tumor markers and keywords.

Click to Enlarge Image: Screenshot of research study search portal.

The tool provides an easy-to-read, user-friendly description of the study, which defines inclusion and exclusion criteria, an overview of what is expected of participants, information about enrolling sites and the study contact information. The dynamic glossary feature provides definitions of research terms without leaving the webpage. All of these easy-to-read and user friendly features help address the challenges that survey respondents expressed regarding technical language and complicated eligibility criteria. Making the tool easily searchable by different categories helps reduce the difficulty of matching patients to clinical trials.

Click to Enlarge Image: Example of user-friendly study detail page

Educational resources
The third component, which also focuses on eliminating the barriers mentioned in the survey,  is the development of new educational resources about:

  • Why you should consider enrolling in research
  • How to find research studies for which you are eligible
  • Legal protections for participants
  • Glossary
  • Frequently asked questions about clinical trials.

We are now developing training modules so that our 200 volunteers can assist patients in using the tool and direct them to additional resources.

We are already integrating the research study search tool we developed through this project into ABOUT efforts, for example our most recent ABOUT participant newsletter included a “featured research” link to the study portal we created. ABOUT is also using what we learned through this project to inform people about the Athena WISDOM Study, a PCORI- funded study that aims to determine whether we can improve breast cancer screening by incorporating risk assessment into the process.

Athena WISDOM Study

The Athena WISDOM Study is a PCORI-funded study that aims to determine whether we can improve breast cancer screening by incorporating risk assessment into the process.

  • Read PCORI’s Blog Post on the Athena WISDOM Study. 

A patient advocate who was trained by FORCE has been actively engaged in the WISDOM study team, from writing its grant, planning recruitment efforts and participating in the future dissemination of study results. We are also excited about using our matching tool to enroll participants in the PCORI funded “Healthy Mind, Healthy You: A Study of Mindfulness” Cross-PPRN study.

We are well on our way to achieving our goal to better target study promotion to the most appropriate audiences and track use and outcomes. Ultimately, we hope that this work will further the mission of PCORI and PCORnet and bring patients and researchers together. Stay tuned for updates over the next year!


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 Executive Committee Welcomes Three New Members, Names New Chair

The PCORnet Council, the main governing body of PCORnet, has approved the slates for PCORnet’s Executive Committee, welcoming three new committee members and appointing a new committee chair.

Kiely Law, research associate in the Department of Pediatrics at the Johns Hopkins School of Medicine and co-founder of the Interactive Autism Network (IAN), has joined the Executive Committee. Advancing autism spectrum disorder (ASD) research is both a professional and personal mission for Dr. Law. In 1996, her son was diagnosed with autism at the age of three, giving her personal insight into the unique value of information collected directly from those living with the struggles and triumphs of autism every day.

The Executive Committee also welcomed Kathleen McTigue as a new member. She is an associate professor of medicine and epidemiology at the University of Pittsburgh and a general internist whose research has focused on the epidemiology of obesity and on using technology to provide evidence-based and patient-centered care. Dr. McTigue is also the lead principal investigator of the PaTH Clinical Data Research Network and a co-principal investigator of the PCORnet Bariatric Study, one of the first PCORnet observational studies.

Elizabeth Shenkman, chairperson for the Department of Health Outcomes and Policy and director of the OneFlorida Clinical Research Consortium, is another new Executive Committee member. Dr. Shenkman’s research focuses on determining which combinations of health care delivery, community, and patient factors influence quality and outcomes of care, as well as developing corresponding evidence-based strategies to improve health outcomes.

In addition to welcoming these new members, the Executive Committee also congratulates Russell Rothman on his appointment to the committee’s chair position. Dr. Rothman is a professor of Internal Medicine, Pediatrics and Health Policy, director of the Vanderbilt Center for Health Services Research and chief of the Internal Medicine/Pediatrics Section. He is also the principal investigator for the Mid-South Clinical Data Research Network and co-chair of the Steering Committee of the ADAPTABLE study. His research focuses on improving care for patients with diabetes, obesity and other chronic diseases.

Congratulations to all on these new appointments!


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 atwww.pcori.org.

PCORnet Leaders Speak Out at Public Workshop to Further Use of Real-World Evidence

The Duke Margolis Center for Health Policy and U.S. Food and Drug Administration (FDA) recently hosted a public workshop to discuss how to effectively use real-world evidence (RWE) – the insights gleaned from information collected outside of a clinical trial, such as electronic medical records—in drug development and regulatory decision-making. PCORI and its innovative initiative, PCORnet, the National Patient-Centered Clinical Research Network, were front and center at the event, with several of our renowned experts sharing how PCORnet is ready to put RWE practices into action and propel healthcare forward.

The opportunity: a living textbook of medicinal products
With RWE, there is a massive opportunity to achieve a totality of information that a single study or trial will never allow, said Dr. Adrian Hernandez, director, Health Services and Outcomes Research, Duke Clinical Research Institute (DCRI) and panel participant at the workshop. He added that by partnering with the FDA and other stakeholders to develop the right framework for using real-world data (RWD) such as electronic medical records, we can more effectively use RWE to complement other kinds of research and create a living textbook about medicinal products and the experiences of patients who use them.

Dr. Hernandez was a panelist at the workshop’s session that spotlighted matching RWE to regulatory use cases. To illustrate how RWE can fill crucial knowledge gaps, he used a personal example: his own mother, a premature coronary disease patient who wants to help other patients like her but finds clinical trials inconvenient for her lifestyle. Dr. Hernandez explained how pragmatic clinical trials like PCORnet’s ADAPTABLE study are engaging participants electronically to better suit patients’ lifestyles and needs.

According to Dr. Hernandez, another key element to effectively leverage RWE lies in understanding the benefit to risk ratio. By creating a parallel system that addresses safety and benefits that matter to patients (e.g. will this improve my quality of life, not just length of life?), we can create a learning healthcare system where patients, clinicians, healthcare systems, and regulators use RWE to generate outcomes that matter most to patients.

 

 

 

 

 

 

 

 

 

 

Joe Selby, executive director, PCORI, also spoke at the workshop, discussing how to chart a collective path as we move forward with the use of RWD and RWE. He shared key elements he believes are essential to the future success of RWE.

 First, he touched on the importance of data linkage and bringing stakeholders and partners together to understand data. He also discussed the need for a societal change. Patients must become aware of barriers to improving their care and have platforms where they can use their own voices to solve these issues.

Dr. Selby also stressed that we aren’t going to learn anything unless we actually dive in and start doing research. Take, for example, PCORnet demonstration studies. Aimed to answer critical research questions, demonstration studies give researchers a chance to compile a list of learnings and trail blaze a new path for collecting data. PCORI is currently funding several of these demonstration studies.

Dr. Selby also touched on standardization and the need to bring stakeholders together to discuss measures that haven’t yet been incorporated into any common data model. He closed with a mention of the need to develop an infrastructure to conduct pragmatic trials in the real world, something that PCORnet and its teams have in their sights for the coming years.

By closing gaps in data, we will close gaps in care
Dr. Kevin Haynes, clinical epidemiologist in translational research for HealthCore, a PCORnet Health Plan Research Network, was a panelist at the workshop highlighting the development of fit-for-purpose RWD. Throughout his discussion, he stuck to a common theme: “We need to close the gaps in data necessary to close the gaps in evidence and ultimately gaps in care.”

We need to close the gaps in
data necessary to close the gaps
in evidence and ultimately gaps in care.

Dr. Kevin Haynes – Clinical Epidemiologist, HealthCore

Dr. Haynes stressed the need to integrate data, and he proposed two forms of data that must be linked: longitudinal data, such as Medicare data, and the data that is held deep inside a clinical system over defined periods of time that is unavailable to today’s administrative claims. HealthCore is one of two PCORI-funded health plan networks partnering with PCORnet’s CDRNs and PPRNs to take the lead in developing a stepwise approach toward successful linkages of claims and EHR data.

You can find additional information about the regulatory use of real-world evidence in this white paper, which both Adrian Hernandez and Richard Platt, co-Principal Investigator of PCORnet’s Coordinating Center, helped compose as contributing authors.

Were you unable to catch the event? Watch the webinar recording and see what you missed!

 

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 Blog

PARTNERS PPRN Uses Health Literacy Tools to Empower Patients

Authors: Vincent Del Gaizo is the father of a systemic onset juvenile idiopathic arthritis patient diagnosed in 2001 at 15 months of age. For the past 12 years, he has been actively involved in the field of pediatric rheumatology research, directly contributing the patient voice and perspective to research initiatives in these rare diseases. He was a founding member and past Chair of Friends of CARRA, a nonprofit organization established to promote needed research and awareness of pediatric rheumatic conditions. Vincent is Co-PI of PARTNERS, a PCORnet patient powered research network in pediatric rheumatic diseases, and is currently the Co-Chair of PCORI’s Rare Disease Advisory Panel.

Sarah Mabus, MLA is the Project Manager for the Center for Advancing Pediatric Excellence (CAPE) in the Pediatric Specialties Care Division at Carolinas HealthCare System’s (CHS) Levine Children’s Hospital. She is also involved in numerous health literacy initiatives, including membership on the CHS Patient/Family Health Education Governance Council’s Infrastructure Subcommittee and Evaluation Subcommittee, and participation on the Outreach and Communications Subcommittee the PARTNERS.

Laura K. Noonan, MD is a founding organizer and current Director of CAPE in Levine Children’s Hospital at Carolinas Medical Center. She has extensive experience and leadership roles in both Quality Improvement (QI) and health literacy. She is a QI consultant for the PARTNERS Patient Powered Research Network and a QI consultant and Parent Advisor for PR-COIN (Pediatric Rheumatology Care and Outcomes Improvement Network). 


 Worried.  Scared.  Confused.

These are some of the feelings people have after learning they or a loved one have a disease with no cure. “What do I do now?” The lack of answers deepens these feelings. Patients and families must choose a medicine they know nothing about to treat an illness they didn’t know existed. When they find information about the disease, it is often hard to understand. Patients and their families might feel left out of the research meant for them.

Thanks in large part to PCORnet, the National Patient-Centered Clinical Research Network funded by the Patient-Centered Outcomes Research Institute (PCORI), this is changing. PCORnet brings patients, caregivers, researchers and doctors together to partner on research so that everyone is included.

In pediatric rheumatology, patients, families, doctors, and advocacy groups teamed up to form PARTNERS (Patients, Advocates and Rheumatology Teams Network for Research and Service). PARTNERS is a Patient-Powered Research Network (PPRN) within PCORnet and its mission is to do the kind of research that will help answer the question “what do I do now?” – so families can be a little less worried, scared, and confused.

One way to include patients and caregivers is with the use of health literacy tools. Health literacy means the ability to work through the health care system, such as finding your way around the building, knowing what test results mean, knowing how to take your medicines, or knowing what your bill or insurance rules mean.

That’s why PARTNERS wrote a health literacy policy. Using this, all members can connect with each other and feel more at ease. Within PARTNERS, everyone from teenagers to doctors has an equal voice on every topic. Bringing together such a diverse group of people is no easy task.  Just as the United Nations uses a translator to connect people from different countries, this health literacy policy is PARTNERS’ translator.

As parent member Marcela Riano says, “When I heard the word research before, I thought we were only talking about new medicines and finding a cure. I now see that health care providers also need to know how to communicate better with patients and find ways to answer our questions and fears. I felt intimidated at first, but now I know, I have a view from my own experience and cultural background that has value.”

One of the project leaders for PARTNERS has been involved in health literacy work across the U.S. She is a doctor and the parent of a child with Juvenile Idiopathic Arthritis. Her experience, passion, and knowledge helped inspire PARTNERS to write the policy in 2016. Many people—including parents, doctors and advocates – wrote pieces of the policy, adding ideas and drawing from many sources. After several drafts, all PARTNERS members agreed to the current policy.

Based on advice from national health literacy experts, the policy includes points on target grade level, word choice, structure, tone, numbers, layout, and graphics. Below are some highlights from the policy:

  • All content must be shared in a way that families can understand, and all contact must encourage patients and families to share their ideas openly.
  • The policy guides all PARTNERS teams and applies to any form of communication including pamphlets, research results, consent forms, invitations, news, etc. (Even this blog post!)

We aim for: 

  • A 6th grade reading level, using tools such as Health Literacy Advisor or the Flesch-Kincaid score found under “Readability Statistics” in Microsoft Word
  • Clear, brief messages with only “need to know” points, that offer ways to learn more, such as non-written options (hotlines, support groups)
  • “Living room language,” choosing short common words, and avoiding jargon
  • Simple and direct sentences, keeping each sentence to 10 – 15 words, using bulleted lists, and avoiding padding sentences with extra words (“very,” “actually”)
  • A positive, active tone spoken directly to the reader (“you” and “your”)
  • Simplified numbers when talking about amount, time, and risk and avoiding very big numbers (over 100) or very small (less than 1), using absolute terms (“6 out of 12”) instead of relative (“twice as many”) and using pictures
  • Plain language definitions for medical terms: “Rheumatologic disease (pain in your joints)”
  • Clean pages with fonts, spacing, and design that help readers process better. We start and end with key points, group similar items, keep white space, and use at least 12-point type.

Health Literacy Tools

If you want to learn more about health literacy, go to the Agency for Healthcare Research and Quality’s (AHRQ’s) Health Literacy Toolkit. It has free health literacy tools for spoken contact, written contact, self-care, and support systems, and tips to get started. The CDC’s “Plain Language Thesaurus” is also an excellent resource to help replace medical words with more health literate choices.

This is harder than it sounds. We don’t always succeed, but we will continue to try. We see it as one of the best ways to improve the health care experience for everyone.


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.

CreakyJoints Recruits Over 11,000 Patients into ArthritisPower

CreakyJoints® recently announced that ArthritisPower™, the first ever patient-led, patient-centered research registry for joint, bone, and inflammatory skin conditions successfully completed enrolling 11,000 arthritis patients, fulfilling one of its primary contract requirements with the Patient-Centered Outcomes Research Institute (PCORI) more than 12 months ahead of schedule.  ArthritisPower is a free science-based, privacy-protected web and mobile app for iPhone and Android devices that allows patients to track their symptoms and treatments while simultaneously participating in arthritis research. Since launching a completely refreshed and advanced version of ArthritisPower in March 2017, over 8,500 patients joined the community and have already logged more than 80,000 patient reported outcome measures. ArthritisPower (AR-PoWER) is a patient powered research network (PPRN) that is part of PCORnet, the National Patient-Centered Clinical Research Network, developed with support from the Patient-Centered Outcomes Research Institute (PCORI), a nonprofit, nongovernmental organization authorized by Congress in 2010. Its overall goal is to support clinical research that will enhance informed health care decision making and improve health care delivery.

University Partners Enhance Understanding
Having a diverse, large, and engaged patient population within ArthritisPower enhances researchers’ ability to study and better understand the arthritis community. Researchers from elite universities and research organizations nationwide, such as Johns Hopkins University, Duke University, Yale University, University of Alabama at Birmingham, and others, access the ArthritisPower research registry to inform their studies.

We are absolutely delighted that ArthritisPower is being actively used by thousands of arthritis patients (like me) who personally benefit from tracking and directly sharing their disease experience with their loved ones and their healthcare providers.

Seth Ginsberg – President and Co-Founder of CreakyJoints and Principal Investigator of ArthritisPower

Resulting data (past and forthcoming) is anonymized and focuses on the patient experience of arthritis to better understand patient preferences related to disease management as well as with traditional and complementary therapies. Already, data derived from ArthritisPower has been presented at major medical meetings such as the American College of Rheumatology and the European League Against Rheumatism, and is slated for future peer-reviewed publications.

“We are absolutely delighted that ArthritisPower is being actively used by thousands of arthritis patients (like me) who personally benefit from tracking and directly sharing their disease experience with their loved ones and their healthcare providers,” said Seth Ginsberg, president and co-founder of CreakyJoints and a principal investigator of ArthritisPower. “We’re also proud that we’ve developed a highly useful tool for researchers who expand our understanding of arthritis while protecting patient identity. But we won’t rest on our laurels. In the future, ArthritisPower will be integrated with other research registries, laboratory tests, and electronic health records so that we can build a truly interactive system for studying arthritis and related conditions.”

Easy Access to Research and Tracking
When people with arthritis sign up for ArthritisPower, they provide informed consent, which allows the app to offer information about a variety of clinical trial and other research opportunities. Patients can proactively decide when and how to participate in studies.

In addition to participating in studies, simultaneously, patients are completing verified self-assessments of their arthritis related symptoms as well as others related to sleep, physical function and pain. As they enter their patient reported outcomes, they can track results over varying durations of time and overlay their usage of medications to see when a new treatment impacts their symptoms. Their personal data can be directly emailed to their provider in advance of an appointment to encourage data driven conversations about treatment and management strategies.

“We’re not done enrolling ArthritisPower. There are millions of Americans living with arthritis whom we invite to join us. Every person who enters the registry helps us understand arthritis better and discover novel treatments and management strategies,” added Mr. Ginsberg.

CreakyJoints®, now in its 18th year, is the go-to source for more than 100,000 arthritis patients and their families world-wide who are seeking education, support, advocacy and patient-centered research. Co-founded in 1999 by arthritis patient Seth Ginsberg and social entrepreneur Louis Tharp, CreakyJoints is part of the Global Healthy Living Foundation, whose mission is to improve the quality of life for people with chronic illness. For more information and to become a member (for free), visit CreakyJoints.org. To learn more about ArthritisPower visit ArthritisPower.org.


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 Board of Governors Approves $27 Million for Projects Using PCORnet

As PCORnet, the National Patient-Centered Clinical Research Network, continues to expand and improve, more research projects are incorporating PCORnet resources and infrastructure. The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors recently approved over $27 million to support four patient-centered research studies that are using PCORnet:

  • A Non-Inferiority Comparative Effectiveness Trial of Physician-Led vs. Nurse-Led Home-Based Palliative Care in Older Adults with Advanced Illness and Their Family Caregivers: This project will compare a conventional physician-led home-based palliative care (HBPC) model that includes routine physician home visits to a contemporary more efficient nurse-led HBPC model that leverages experience and training of nurses and remote physician supervision to facilitate greater access to palliative care services. This study will take place in sites across the Partners Patient Outcomes Research to Advance Learning (PORTAL) Clinical Data Research Network (CDRN).

     

  • Population-Based Comparison of Evidence-Based, Patient-Centered Advance Care Planning Interventions on Advance Directive Completion, Goal Concordant Care and Caregiver Outcomes for Patients with Advanced Illness: This project will compare three practical approaches to advance care planning (ACP) for patients with cancer, heart, and lung disease at 27 clinics at three University of California (UC) sites (UCLA, UCSF, and UCI) that are part of the pSCANNER CDRN. Outcomes for ACP and treatment will be collected for all patients with advanced illness in these participating clinics.

     

  • Provider-Targeted Behavioral Interventions to Prevent Unsafe Opioid Prescribing for Acute Non-Cancer Pain in Primary Care: This project, occurring in three health systems that participate in the PaTH CDRN, will look at the comparative effectiveness of different health system strategies to prevent unsafe opioid prescribing while ensuring access to non-opioid methods for pain management. The project team will assess whether behavioral science-based interventions can “nudge” providers toward more evidence-based care for patients with acute non-cancer pain. The multidisciplinary research team will work closely with a stakeholder advisory committee comprised of patients, patient advocates, primary care providers, pain medicine specialists, payers, health system executives, experts in behavioral science, and regional and national organizations.

     

  • Statistical Methods and Designs for Addressing Correlated Errors in Outcomes and Covariates in Studies Using Electronic Health Records Data: This project will develop statistical methods that allow researchers to obtain accurate estimates using data that have been only partially validated and to better understand which patient records should be validated to optimize resources. It will then apply the methods to a real-world study using EHR data from the Mid-South CDRN that is identifying factors that affect risk of early childhood obesity.

     

  • Comparative Effectiveness of an Exercise Intervention Delivered via Telerehabilitation and Conventional Mode of Delivery: This project will aim to address exercise challenges for those with multiple sclerosis (MS). For a variety of reasons, many people with MS do not exercise, which can lead to increased health challenges.  In partnership with PCORnet’s iConquer, the study will compare an individual  exercise program followed at home or at an exercise facility by people with MS. Based on findings, tools will be developed to help educate the MS community so patients for whom it is difficult to go to an exercise facility regularly can have access to the program at home.

     

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

Save the Date: PCORI Annual Meeting Features PCORnet Workshop

The Patient-Centered Outcomes Research Institute’s (PCORI) Annual Meeting, taking place October 31st– November 2nd, will showcase the National Patient-Centered Clinical Research Network (PCORnet) in a special workshop: “PCORnet in Action: Utilizing PCORnet for Research.”  This workshop, occurring Thursday, Nov. 2nd, will focus on how to conduct research using PCORnet resources and infrastructure. It will include an introduction to PCORnet and three panel discussions. The first panel will discuss a range of research studies taking place throughout PCORnet, highlighting the value added by using this national research infrastructure. The second panel will focus on research, data, and engagement resources available through PCORnet. Resources addressed will include Collaborative Research Groups, the PCORnet Front Door, the PCORnet Common Data Model, the PCORnet Engagement Assessment Tool, and the PCORnet Commons.  Finally, the last panel will present multiple use cases to describe the process of initiating projects in PCORnet.

As you make your travel plans to attend the PCORI Annual Meeting, be sure to check out the agenda and make time to attend this important PCORnet workshop. If you 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

PRIDEnet to Support Engagement of LGBTQ Community in NIH’s ‘All of Us’ Research Program

The San Francisco General Hospital Foundation (SFGHF) has been awarded $542,000 by the National Institutes of Health to engage sexual and gender minorities across the country in the All of Us Research Program, in part through outreach to PRIDEnet, a PCORnet Partner Network. Formerly known as the Precision Medicine Initiative Cohort Program, the All of Us Research Program is an ambitious effort to gather data over time from one million or more people living in the United States to ultimately accelerate research and improve health.

“This is a historic time for sexual and gender minorities in which these communities are beginning to be accounted for and empowered as stakeholders in the clinical research process from the very outset,” said Mitchell Lunn, MD, MAS, a principal investigator for the NIH award and of PRIDEnet. “In alignment with the patient-centered vision of PCORnet and the Patient-Centered Outcomes Research Institute (PCORI), the All of Us Research Program’s efforts will cultivate long-term, meaningful relationships, which will help us shed light on relevant health disparities within the entire nation, including these minority groups.”

As part of this effort, Lunn and his entire team, including University of California, San Francisco professor and co-principal investigator Kirsten Bibbins-Domingo, PhD, MD, MAS, will serve as All of Us Engagement Partners. In this capacity, they will engage the LGBTQ (Lesbian, Gay, Bisexual, Transgender, Queer and Questioning) community to ensure all study materials are appropriate for this specific group. The team will also provide input on enrollment materials and research plans, develop customized educational and recruitment/retention programs, and study best practices in the dissemination of research results to support retention.

The All of Us Research Program intends to open for national enrollment in late 2017 or early 2018. SFGHF’s award will last three years, while the entire program will continue indefinitely.

 

*Precision Medicine Initiative, PMI, All of Us, the All of Us logo, and The Future of Health Begins with You are service marks of the U.S. Department of Health and Human Services.

 

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.

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.

Diversity, Communication and Trust: Three PPRNs Share the Recipe for Effective Engagement in Rare Disease Research

Imagine you are a patient who has just been diagnosed with primary Nephrotic Syndrome. Your doctor explains to you that this is a rare kidney disorder and then hits you with the gut punch: there is no known cause, no cure, and no FDA-approved medication for treatment. We often think of rare conditions as something that will never impact us, but the truth is that rare diseases collectively are not so rare. In fact, they impact nearly 25 million Americans according to the National Institutes of Health. Patients and caregivers impacted by rare diseases are frustrated by the lack of knowledge around these conditions and, as a result, are often more motivated than most to participate in efforts to drive research forward. It’s a rich opportunity for the research community; but how do we channel that opportunity to drive long-term engagement in clinical research?

PCORnet, the National Patient-Centered Clinical Research Network, a PCORI initiative, has made answering this question a priority. Rare diseases are well represented in PCORnet’s Partner Networks; in fact, several of these networks have a singular focus on one or more such illnesses. To fast track the sharing of lessons learned across the Network, these networks have joined forces to create the Engagement & Experience Research Group, an innovative collaborative focused on advancing the science of multi-stakeholder engagement and enhancing the patient experience in clinical care and research. The group is comprised of five PCORnet Partner Networks: ADVANCE CDRN, CAPriCORN CDRN, DuchenneConnect PPRN, Interactive Autism Network PPRN, and Phelan-McDermid Syndrome Data Network PPRN. Unsurprisingly, some common themes quickly emerged as success indicators for engaging the rare disease community: trust, diversity and communication.

Building Trust Between Communities

Those involved with Phelan-McDermid Syndrome Data Network (PMS_DN), a network dedicated to advancing the knowledge, care, and treatment of a rare pediatric disease known as Phelan-McDermid Syndrome (PMS), cite trust as the fire that fuels all effective engagement, especially for their specific patient community in which children are the primary participants.

PCORnet and Rare Disease

Learn more about how PCORnet can benefit
rare disease research.

“We have found engagement to be most effective when we leverage communities of fellow patients and caregivers to drive awareness of the research we conduct. Parents encouraging parents to engage is more effective than researchers or clinicians trying to motivate them,” said Megan O’Boyle, principal investigator for PMS_DN. “Trust is already established when you are talking to a mom or dad going through the same thing you are. When caregivers come to us and tell us that they had a positive research experience, we encourage them to share that feeling with others families.” O’Boyle also stressed what she refers to as the golden rule of all research, rare disease or otherwise: return the data.

“Patients and caregivers in rare disease communities are seldom motivated by money,” she said. “They are motivated by the need for answers. The relationship with the research community has to work both ways: if families give us their time and effort, it is incumbent on us to give them the results of the studies they have participated in.”

Putting Diversity Into Action

One of PCORnet’s youngest PPRNs and former member of the Engagement & Experience Research Group, NephCure Kidney Network (NKN), is on a quest to improve insights into the very condition mentioned above: primary Nephrotic Syndrome, a rare disease that causes the body to excrete too much protein into the urine, leading to swelling in the body and potential kidney failure. At NKN, one key to unlocking answers within rare disease communities lies in finding the right balance of diversity—and then putting that mix of diversity into action to make sure all patient participants feel empowered.

“We have found that effective engagement doesn’t just mean collecting any patient feedback—it means collecting the right patient feedback,” said Elizabeth Cope, former principal investigator for NKN and current co-investigator for ADVANCE. “For example, a patient advisory group is a great start to giving patients a voice in the research process, but we have to keep in mind that patient advisory groups only appeal to certain types of people with the interest and ability to offer a deep commitment to projects; the collective experience of this group may not be generalizable to all patients. Finding the right balance of diversity is essential for building a resource that meets the needs of its patient community at-large.”

NKN operationalized this one-size-does-not-fit-all approach to engagement by offering a buffet of study engagement options for patients to choose from. While deeper, time-heavy options were presented, such as participation in network governance, Cope and her colleagues also offered “single-touch” opportunities to increase the diversity of participant input. Additionally, NKN provides education and motivational outreach content to patients, which allows participants to see that their role in advancing rare disease research matters, even at the single-touch level.

Focusing on Effective Communication

Like NKN, DuchenneConnect (DCN), a PCORnet PPRN created to support families and advance research toward a cure to a set of rare neuromuscular disorders known as Duchenne and Becker muscular dystrophy (DBMD), believes patient empowerment and bi-directional communication between the patient community and the research community is essential. DCN uses various communication tools and tactics to open new possibilities within rare disease research and put patients in the driver’s seat.

 “Many roadblocks in clinical research, particularly rare disease research, can be attributed to knowledge gaps. We focus on those gaps, including helping basic researchers and sponsors understand unmet needs; informing sponsors and regulators about meaningful benefits and acceptable risks and burdens; and educating people with Duchenne and their families around what it means to participate in a trial,” said Holly Peay, co-principal investigator for DCN. “The more ways we find to speed the research process, clarify misconceptions, and minimize the intimidation factor often associated with clinical research, the faster we can uncover insights to help the Duchenne community.”

“The more ways we find to speed
the research process, clarify
misconceptions,and minimize
the intimidation factor often
associated with clinical research,
the faster we can uncover insights
to help the Duchenne community.”

Holly Peay– Co-Principal Investigator, DCN

One of the newest communication tactics recently unveiled by DCN is a decision tool that helps people with Duchenne and their families determine whether participating in clinical trials might be the right choice for them. This tool helps patients deliberate potential trial benefits, harms and burdens, and ultimately choose a trial that best suits their needs. The overall objective is to increase the number of people who make an informed choice to participate in clinical research.

PCORnet’s networks study a range of conditions and diseases, and effective engagement is central to every one of them. Learn more about how PCORnet is engaging networks and participants to advance clinical research and drive healthcare outcomes.


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