Patient-reported outcome (PRO) measures are often used in pragmatic clinical trials to assess endpoints that are meaningful to stakeholders. These measures may also support patient care, although there is mixed evidence about effects of PROs on (1) improved patient-provider communication, clinical decision-making, and patient satisfaction; (2) enhanced patient outcomes; and (3) helped ensure better quality of care from a healthcare systems perspective. In a new white paper from the Collaboratory Patient-Reported Outcomes (PRO) Core, the available evidence in the literature is examined to determine when PROs have the potential to provide added value to patient care.
The full text of the white paper can be found here: Impact of Patient-Reported Outcomes on Clinical Practice_V1.0
On September 17, the Precision Medicine Initiative Working Group presented to NIH Director Dr. Francis Collins a detailed design framework to advance the creation of a national, large-scale research cohort for developing more effective treatments tailored to individuals. The framework makes recommendations on cohort assembly, participant engagement, data, biological specimens, policy, and governance. The recommendations are based on a set of high-value scientific opportunities that were identified by the working group following extensive stakeholder engagement.
The NIH plans to move quickly to build the infrastructure so that participants can begin enrolling in the cohort in 2016, with a goal of enrolling at least 1 million participants in 3 to 4 years. Visit the NIH News & Events website for more details.
The National Patient-Centered Clinical Research Network (PCORnet) has recently made a draft protocol for its first randomized clinical trial available for stakeholder review. Researchers, clinicians, patients and the public are all invited to read the current draft of the study protocol and provide comments and feedback.
The ADAPTABLE Study (PDF), which will investigate whether lower- or higher-dose aspirin is better for preventing heart attack and stroke in patients at risk for heart disease, is PCORnet’s first randomized pragmatic clinical trial. Designed to leverage PCORnet’s Clinical Data Research Networks (CDRNs) and Patient-Powered Research Networks (PPRNs), the trial will serve as twofold purpose: answering a clinical question of direct importance for patients, families, and healthcare providers, and serving as a demonstration of PCORnet’s capabilities in conducting clinical research on a national scale.
Links to the proposed study protocol, a survey tool for capturing feedback, and other information about ADAPTABLE Study, including press releases, fact sheets, and infographics, are available at the link below:
ADAPTABLE: The Aspirin Study
Follow PCORnet on Twitter @PCORnetwork for updates on the ADAPTABLE #ClinicalTrial
An explosion in the collection of personal data is fostering concerns about the extent to which health information is accessed—and about the privacy and confidentiality of this information. Two recent National Public Radio stories highlight a few of the burgeoning uses of these abundant data.
In the first, an insurer uses personal data to predict who will get sick so it can identify patients at highest risk for hospital admission, or readmission, and then provide them with personal health coaches. The coordinated care given to patients by the coaches (for example, arranging a visiting nurse or streamlining appointments) has been shown to improve hospitalization rates. The insurer says it follows federal health privacy guidelines for anonymity and uses the information to better serve its members.
The second story explains that results of online health searches aren’t always confidential, and data brokers are tracking information and selling it to interested parties. The author notes that data gathered on the Web are, for the most part, unregulated. Both stories raise questions about privacy and confidentiality of health information and how to best protect it.
Pragmatic clinical trials also seek to use personal health data to answer important questions on the risks, benefits, and burdens of therapeutic interventions. In a blog post in Health Affairs, Joe Selby, executive director of the Patient-Centered Outcomes Research Institute (PCORI), underscores the need for trust, support, and active engagement of patients when involving them in health data research, even with privacy protections in place. PCORI has launched the National Patient-Centered Clinical Research Network (PCORnet) as a means of harnessing large clinical data sets to study the comparative effectiveness of treatments, and a central tenet of the network is that patients, clinicians, and healthcare systems should be actively involved in the governance of the use of health information.
Read the full articles
From NPR: Insurer Uses Personal Data To Predict Who Will Get Sick
From NPR: Online Health Searches Aren't Always Confidential
From Health Affairs: Advancing the Use of Health Data in Research With PCORnet
The Patient-Centered Outcomes Research Institute (PCORI) has approved the first pragmatic clinical trial to be performed through the National Patient-Centered Clinical Research Network (PCORnet)—the ADAPTABLE study (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-term Effectiveness).
Over the course of the trial, 20,000 study participants with cardiovascular disease will be randomly assigned to receive one of two commonly used doses of aspirin—a low dose of 81 mg per day versus a higher dose of 325 mg per day—in order to determine which provides the optimal balance between protecting patients with cardiovascular disease from heart attack and stroke, and minimizing bleeding events associated with aspirin therapy. The trial will also employ a number of innovative methods, including electronic health record (EHR)-based data collection and a patient-centered, web-based enrollment model in partnership with the Health eHeart Alliance Patient-Powered Research Network (PPRN).
The ADAPTABLE trial, which includes six of PCORnet’s Clinical Data Research Networks (CDRNs), will be led and coordinated through the Duke Clinical Research Institute (DCRI).
Read more about the ADAPTABLE Aspirin Trial here:
Fact Sheet (PDF)
DCRI Coordinating Center Announcement
This month’s issue of the Journal of General Internal Medicine features a systematic review of stakeholder engagement in comparative effectiveness research and patient-centered outcomes research. Thomas W. Concannon, PhD, and coauthors identified 70 peer-reviewed articles since 2003 that reported on this topic.
Key results included:
- Patients were the most commonly engaged group, followed by modest engagement of clinicians, and infrequent engagement of other stakeholders across the healthcare system.
- Stakeholders were more often engaged in earlier stages of research (evidence prioritization and generation) than in later activities such as evidence interpretation and application.
Overall, reporting of stakeholder activities and the effects of engagement were highly variable in the literature. To address this, the authors developed a 7-item questionnaire for the reporting of stakeholder engagement in research. A suggested plan for future research on stakeholder engagement is also outlined.
Recently the journal Health Affairs and the Patient-Centered Outcomes Research Institute (PCORI) conducted a Twitter chat on the topic of patient engagement in research. The chat was an hour-long moderated question-and-answer session with Susan Sheridan, the director of patient engagement at PCORI. Participants joined the conversation via #PatientHC and talked about what patient engagement looks like and concerns about the privacy of patient health data.
The hosts referenced the journal’s 2013 theme issue on patient engagement as well as three videos produced in partnership with PCORI that illustrate ways patients and providers are incorporating patient engagement in healthcare decisions. An archived version of the Twitter chat is here.
“PCORnet: Turning a Dream Into Reality,” an editorial published online this week in the Journal of the American Medical Informatics Association, details the promises of the National Patient-Centered Clinical Research Network (PCORnet):
“Providing accurate answers based on the highest levels of scientific evidence for the majority of unresolved clinical questions is a revolutionary dream shared by patients, providers, payers, health plans, researchers, and policy makers alike. PCORnet, the National Patient-Centered Clinical Research Network, promises a transformative platform that will turn this revolutionary dream into reality.”
The authors describe PCORnet’s patient-centered vision and how its transformative clinical research will be carried out through a national research network involving both Clinical Data Research Networks and Patient-Powered Research Networks. This “network of networks” is designed to include a large, highly representative population. PCORnet will first work to establish data architecture and standards and address key policy questions, followed by the conduct of its first pragmatic clinical trial slated to begin in September 2015.
Editorial authors include NIH Director Dr. Francis Collins and PCORnet Steering Committee members Kathy L. Hudson, PhD, and Josephine P. Briggs, MD.
“Ethics and Regulatory Complexities for Pragmatic Clinical Trials,” a Viewpoint article by Jeremy Sugarman, MD, MPH, MA, and Robert Califf, MD, was published online in JAMA today. In the article, the authors draw on early experiences from two large networks conducting pragmatic clinical trials, the NIH Collaboratory and the National Patient-Centered Clinical Research Network (PCORnet), to describe 10 ethical and regulatory complexities facing this new field of research. Topics covered include informed consent, risk determination, the role of gatekeepers, and institutional review board review and oversight, among others, as well as the ongoing need for further discussion and research as a key part of efforts aimed at creating a learning healthcare system.
Dr. Sugarman is chair of the Regulatory/Ethics Core of the NIH Collaboratory and deputy director for medicine of the Johns Hopkins Berman Institute of Bioethics. Dr. Califf is the principal investigator of the NIH Collaboratory Coordinating Center and director of the Duke Translational Medicine Institute.
Slides and a workshop summary are now available from the Institute of Medicine website.
On April 23-24, 2014, the Institute of Medicine held a Roundtable on Value & Science-Driven Health Care workshop titled “Health System Leaders Working Towards High Value Care Through Integration of Care and Research” in Washington, DC. The workshop, sponsored by the Patient-Centered Outcomes Research Institute (PCORI), brought together stakeholders from across research networks, health systems, industry, and the patient community to engage on barriers and opportunities in building a continuously learning health system, where routinely collected health data informs improvements in evidence-based care. Eric Larson, MD, MPH, of the Group Health Research Institute chaired the workshop Planning Committee.
Workshop goals included:
- Fostering the development of a shared commitment, vision, and strategy among health system leaders building a national clinical research network
- Broadening and deepening health systems’ leadership awareness of the prospects for and from a continuously learning health system
- Learning from models and examples of productive integration of research with care delivery programs
- Identifying common issues compelling to health systems leaders related to science, technology, ethics, regulatory oversight, business, and governance
- Exploring strategic opportunities for executive, clinical, and research leaders to forge working partnerships for progress
- Considering the approach and desirable outcomes of a meeting of CEO leaders vital to building and sustaining a functional, real-time national clinical research network
One particular focus of the meeting was the recently launched National Patient-Centered Clinical Research Network (PCORnet). Workshop participants included representatives from PCORnet’s Clinical Data Research Networks and Patient-Powered Research Networks. Speakers provided real-world examples of learning health systems and urged leaders to make a business case for the learning health system model. Speed and dissemination of research were discussed as important considerations for patients.
The meeting was open to the public via webcast. Archived meeting presentations will be made available; a link will be provided in an update to this post. Workshop-related tweets can be found with the hashtag #IOMPCORI.
View the workshop agenda and briefing materials.