August 28, 2018: ADAPTABLE Patient-Reported Health Data Codes Now Available

The ADAPTABLE pragmatic trial relies on patients to report key information at baseline and throughout follow-up. To capture these data, ADAPTABLE investigators developed a LOINC (Logical Observation Identifiers Names and Codes) patient-reported item set, which is now publicly available.

The development of the item set is part of the ADAPTABLE Supplement, an initiative funded by the Office of the Assistant Secretary for Planning and Evaluation to develop best practices for capturing patient-reported outcome data and optimal analytic approaches for using the data in a pragmatic clinical trial. Additional reference material can be found in the ADAPTABLE Supplement Roundtable Meeting summary, in a report describing the results of a literature review of data standards and metadata standards for variables of interest, and on GitHub. The project is expected to inform future efforts to integrate patient-reported data in the electronic health record and provide opportunities to streamline data for use in pragmatic trials. Information from the project is being added to the Living Textbook as it accumulates; learn more in the chapters on Using Electronic Health Record Data and Choosing and Specifying End Points and Outcomes.

ADAPTABLE (Aspirin Dosing: A Patient-Centric Trial Assessing Benefits and Long-Term Effectiveness) aims to identify the optimal dose of aspirin therapy for secondary prevention in atherosclerotic cardiovascular disease and is the first major randomized comparative effectiveness trial to be conducted by the National Patient-Centered Clinical Research Network (PCORnet).

CTSA-PCORnet Webinar: A Central IRB Approach


The webinar copresented on March 2, 2016, by the Clinical and Translational Science Awards (CTSA) program and PCORnet is available as a video and slideset.

Petra Kaufmann, MD, MSc
Director, Office of Rare Diseases Research and Division of Clinical Innovation
National Center for Advancing Translational Sciences
National Institutes of Health

Rachael Fleurence, PhD
Program Director, CER Methods and Infrastructure Program
Patient-Centered Outcomes Research Institute

Sabune J. Winkler, JD
Director, Regulatory Affairs Operations
Harvard Catalyst

Webinar details:
March 2, 2016
3pm - 4pm ET
To join the WebEx, click here: http://bit.ly/1TGRTFS
Call-in number: 1-855-244-8681
Access code: 737 807 582

Special Issue Published on Ethical & Regulatory Complexities of Pragmatic Clinical Trials


Tools for ResearchA new series of 12 articles published in a special issue of the journal Clinical Trials addresses ethical and regulatory challenges particular to pragmatic clinical research. Pragmatic clinical trials are designed to efficiently provide answers to important clinical questions, yet they present special challenges in conforming to the ethical and regulatory guidelines that were developed for more traditional clinical research. The special issue describes these challenges and begins to outline possible solutions that will protect the rights and welfare of research participants while allowing pragmatic clinical trials to gather much-needed evidence for informing healthcare decisions. An introductory article is followed by 11 articles addressing individual topics, such as alteration of informed consent, privacy, gatekeepers, and defining minimal risk research. The effort was funded by the NIH Health Care Systems Research Collaboratory, with additional support from the Patient-Centered Outcomes Research Institute (PCORI), and involved diverse groups of stakeholders, including researchers, patient advocates, bioethicists, and regulatory experts. Robert M. Califf, MD, and Jeremy Sugarman, MD, MPH, were editors of the special issue.

For more information:


Closer Partnerships Needed Between Researchers and Healthcare Executives


Larson-JohnsonIn a commentary published this week in Modern Healthcare, Eric Larson, MD, MPH, and Karin Johnson, PhD, of Group Health Research Institute, argue that greater collaboration is needed between clinical researchers and healthcare system executives to address a “a gap between research approaches and delivery system needs.” Perspectives gathered through a survey and Institute of Medicine workshop with healthcare executives indicated that research is not conducted fast enough or designed in a way that facilitates translation of evidence into clinical practice. The NIH Collaboratory and National Patient-Centered Clinical Research Network (PCORnet) are cited as examples of effective partnerships between researchers and healthcare leaders; these research programs are addressing high-priority clinical questions and generating actionable knowledge. According to Drs. Larson and Johnson, pragmatic clinical trials and big data offer opportunities to create a learning health system, but this will require combining the perspectives and expertise of researchers and stakeholders from healthcare delivery systems. Drs. Larson and Johnson are part of the NIH Collaboratory’s Health Care Systems Interactions Core, a working group that “aims to support and facilitate productive collaboration between researchers, clinicians, and health system leaders.”

Related links:


PCORnet Posts Aspirin Study Protocol for Public Review and Comment


PCORnetThe 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


Patient-Reported Outcomes Workshop Report Available


Tools for ResearchIn January of 2015, the NIH HCS Collaboratory’s Patient-Reported Outcomes (PRO) Core Group convened a 2-day workshop in Baltimore devoted to identifying barriers and possible solutions to the use of NIH-supported PRO tools in comparative-effectiveness research (CER).

Findings from the meeting, which include case study presentations and reflections from multiple stakeholders representing the research, clinical, and patient communities, were distilled into a summary document available from the NIH Collaboratory Knowledge Repository at the link below:

The workshop summary is also available on the Living Textbook’s “Tools for Research” section, under “Patient-Reported Outcomes White Paper.


In the News: Increase in Use of Personal Health Data


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

 

PCORI Announces First PCORnet Demonstration Project: The ADAPTABLE Aspirin Study


PCORnetThe 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)
Infographic (PDF)
DCRI Coordinating Center Announcement

Study Examines Public Attitudes Toward Data-Sharing Networks


A new study examining public attitudes about the sharing of personal medical data through health information exchanges and  distributed research networks finds a mixture of receptiveness and concerns about privacy and security. The study, conducted by researchers from the University of California, Davis and University of California, San Diego and published online in the Journal of the American Medical Informatics Association (JAMIA), reports results from a telephone survey of 800 California residents. Participants were asked for their opinions about the importance of sharing personal health data for research purposes and their feelings about related issues of security and privacy, as well as the importance of notification and permission for such sharing.

The authors found that a majority of respondents felt that sharing health data would “greatly improve” the quality of medical care and research. Further, many either somewhat or strongly agreed that the potential benefits of sharing data for research and care improvement outweighed privacy considerations (50.8%) or the right to control the use of their personal information (69.8%), although study participants also indicated that transparency regarding the purpose of any data sharing and controlling access to data remained important considerations.

However, the study’s investigators also found evidence of widespread concern over privacy and security issues, with substantial proportions of respondents reporting a belief that data sharing would have negative effects on the security (42.5%) and privacy (40.3%) of their health data. The study also explored attitudes about the need to obtain permission for sharing health data, as well as whether attitudes toward sharing data differed according to the purpose (e.g., for research vs. care) and the groups or individuals among which the data were being shared.

The authors note that while data-sharing networks are increasingly viewed as a crucial tool for enabling research and improving care on a national scale, they ultimately rely upon trust and acceptance from patients. As such, the long-term success of efforts aimed at building effective data-sharing networks may depend on accurately understanding the views of patients and accommodating their concerns.


Read the full article here: 

Kim KK, Joseph JG, Ohno-Machado L. Comparison of consumers' views on electronic data sharing for healthcare and research. J Am Med Inform Assoc. 2015 Mar 30. pii: ocv014. doi: 10.1093/jamia/ocv014. [Epub ahead of print]

The National Patient-Centered Clinical Research Network (PCORnet) Adopts Definitions for Information Provided by Patients

The NaTools for Researchtional Patient-Centered Clinical Research Network (PCORnet) Patient-Reported Outcomes (PRO) Task Force has released a new white paper that delineates definitions for data contributed by patients. They hope to facilitate the incorporation of information provided by patients across the networks and to maximize data sharing and interoperability by operationalizing the terms within PCORnet.

The following three terms were adopted to describe the patient-contributed data collected across PCORnet:

PATIENT-REPORTED OUTCOME (PRO): a report that comes directly from the patient about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.

PATIENT-CENTERED OUTCOMES: outcomes that matter to patients

PATIENT-GENERATED HEALTH DATA: health-related data (such as health history, symptoms, biometric data, treatment history, lifestyle choices, and other information) that are created, recorded, gathered, or inferred by or from patients or their designees (i.e., care partners or those who assist them) to help address a health concern. Patient-generated health data include patient-reported outcomes.

The white paper can be found under Patient-Reported Outcomes White Papers on the Tools for Research page on the living textbook, or accessed directly here (PDF).