All posts by Karen Staman

New White Paper from Collaboratory PRO Core on the Impact of Patient-Reported Outcomes on Clinical Practice

Patient-Reported Outcomes, Uncategorized, ,

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

New Living Textbook Chapter on Acquiring and Using Electronic Health Record Data for Research

Data quality, Data sharing, Data standards, Electronic health records, Pragmatic clinical trials, , , ,

Topic ChaptersMeredith Nahm Zozus and colleagues from the NIH Collaboratory’s Phenotypes, Data Standards, and Data Quality Core have published a new Living Textbook chapter about key considerations for secondary use of electronic health record (EHR) data for clinical research.

In contrast to traditional randomized controlled clinical trials where data are prospectively collected, many pragmatic clinical trials use data that were primarily collected for clinical purposes and are secondarily used for research. The chapter describes the steps a prospective researcher will take to acquire and use EHR data:

  • Gain permission to use the data. When a prospective researcher wishes to use data, a data use agreement (DUA) is usually required that describes the purpose of the research and the proposed use of the data. This section also describes use of de-identified data and limited data sets.
  • Understand fundamental differences in context. Data collected in routine care settings reflect standard procedures at an individual’s healthcare facility, and are not collected in a standard, structured manner.
  • Assess the availability of health record data. Few assumptions can be made about what is available from an organization’s healthcare records; up-front, detailed discussions about data element collection over time at each facility is required.
  • Understand the available data. A secondary data user must understand both the data meaning and the data quality; both can vary greatly across organizations and affect a study’s ability to support research conclusions.
  • Identify populations and outcomes of interest. Because healthcare facilities are obligated to provide only the minimum necessary data to answer a research question, investigators must identify the needed patients and data elements with specificity and sensitivity to answer the research question given the available data.
  • Consider record linkage. Studies using data from multiple records and sources will require matching data to ensure they refer to the correct patient.
  • Manage the data. The investigator is responsible for receiving, managing, and processing data and must demonstrate that the data are reproducible and support research conclusions.
  • Archive and share the data after the study. Data may be archived and shared to ensure reproducibility, enable auditing for quality assurance and regulatory compliance, or to answer other questions about the research.

Study Recommends Shared Decision Making for Research on Medical Practices

Uncategorized

Research on medical practices (ROMP) includes medical record reviews, comparative effectiveness research, quality improvement interventions, and point-of-care randomization, and may improve the efficiency, quality, and cost-effectiveness of medical care.

In a study by Maureen Kelly and colleagues recently published in The American Journal of Bioethics, researchers found that patients may not fully understand the rationale for ROMP or the extent to which this type of research already exists. Patients care most about how risks and consent are managed and communicated within the physician-patient relationship, view research as separate from usual care, and place their trust in their physician, whom they rely on to identify and filter risks.

Because current approaches to oversight, risk assessment, and informed consent are poorly suited to ROMP, the authors suggest a model of Shared Decision Making (SDM) as an approach to disclosure, consent, randomization and data sharing. With SDM, the physician engages the patient in the decision-making process and encourages conversations regarding the uncertainty of treatment options.

In a related commentary, Dr. Jeremy Sugarman urges consideration of the appropriateness of this analytic frame for ROMP due to the important differences between the primary aims of research and clinical care: in research the primary goal to generate information, while for clinical care, the primary goal is to benefit patients.

Reference: Kelley M, James C, Alessi Kraft S, et al. Patient Perspectives on the Learning Health System: The Importance of Trust and Shared Decision Making. 2015;15:4–17. PMID: 26305741. doi: 10.1080/15265161.2015.1062163.
For more information on RoMP, see the Grand Rounds Presentation from December 2014: A RoMP through the Empirical Ethics of Pragmatic Clinical Trials

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

Patient-Centered Outcomes Research, Patient-Reported Outcomes, Pragmatic clinical trials, ,

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).

Groundbreaking Suicide Prevention Trial has Enrolled Initial Patients

Clinical trials, Cluster Randomized Trials, Pragmatic clinical trials, Randomized controlled trials, , , , ,

March 5, 2015

Dr. Greg Simon and the Suicide Prevention Team have enrolled the first participants in the Pragmatic Trial of Population-Based Programs to Prevent Suicide Attempt. This groundbreaking study was developed by researchers at Group Health Cooperative in Seattle, Washington, Health Partners Medical Group in Minnesota, and Kaiser Permanente of Colorado, in collaboration with patients who have experienced suicidal thoughts or survived suicide attempts themselves.

Over 9 million adults in the United States experience suicidal thoughts, and more than 1 million adults attempt suicide each year. However, patients at risk for suicidal behavior are not routinely identified, and successful interventions for depression and suicide are not routinely implemented. New evidence suggests that patients who report frequent thoughts of death or self-harm on a commonly-used depression questionnaire are at higher risk for suicide attempt and death over the following year.

This study aims to address the significant problem of suicide by identifying patients who are at risk for suicidal behavior and testing two suicide prevention strategies. Patients at participating institutions will complete a standard depression severity questionnaire during routine clinical care, and the results will be stored in their electronic health records (EHR). Investigators will use the responses in the EHR to identify at-risk individuals, and once identified, the patients will be randomly assigned to either usual care or to two treatment programs. The first is a collaborative care-management approach; the second is an online skills training program called “Now Matters Now,” which is designed to help people manage painful emotions and stressful situations.

Over the next 5 years, the team plans to enroll 19,500 adult patients. The study is an NIH Collaboratory Demonstration Project being overseen by the National Institute of Mental Health (NIMH).

Congratulations to Dr. Simon and his team for their achievement!

 

Office for Human Research Protections Releases Draft Guidance on Disclosing Risks in Standard-of-Care Research

Comparative Effectiveness Research, Informed Consent, Pragmatic clinical trials, Regulatory issues, , , ,

On October 20, 2014 the Office for Human Research Protections (OHRP) released a draft guidance on how to apply the Department of Health and Human Services (HHS) regulations on protecting human subjects (45 CFR Part 46) who participate in research studies intended to evaluate risks of treatments or procedures commonly used by healthcare professionals and recognized as “standard of care.” In standard-of-care research (or comparative effectiveness research), participants are randomized to receive one of two (or more) treatments that are accepted by medical experts as appropriate treatments for a given disease or condition.

Because treatments assigned to some participants might be different than the treatments they would have been assigned if they were not participating in the study, and the risks associated with one treatment might be different from the risks associated with another treatment, the OHRP recommends that these risks be fully described to potential participants as a part of the informed consent process.

Click here for the full draft guidance: Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care

The Institute of Medicine is planning a two-day public workshop in December to discuss human subjects protections in standard-of-care research. Click here for more information.​

 

Patient-Generated Data and Comparative Effectiveness Research

Comparative Effectiveness Research, Patient-Centered Outcomes Research, Patient-Reported Outcomes

July 9, 2014

Researchers at Duke Medicine published an article in the July 2014 issue of Health Affairs that assesses the need for, uses of, and strengths and weaknesses of patient-generated data and reviews efforts to create new streams of patient-generated data for clinical and research use. The authors discuss patient-facing technologies, such as wearable sensors and other devices that collect patient-reported outcomes (PROs), and explore ways that these technologies could potentially help inform decisions made by patients, providers, and policy makers and ultimately improve the quality of patient care.

Click here for the article: Assessing the Value of Patient-Generated Data to Comparative Effectiveness Research.

Amy Abernethy and Tracie Locklear, two of the paper’s authors, are also members of the NIH Collaboratory’s Patient-Reported Outcomes (PRO) Core Working Group. Click here to see their Living Textbook chapter on PROs.

The Diane Rehm Show Features PCORnet

Comparative Effectiveness Research, Patient-Centered Outcomes Research, Patient-Reported Outcomes, , ,

On Thursday, April 17, The Diane Rehm Show, a nationally syndicated news and call-in program carried on National Public Radio, aired a program titled The Promise and Perils of a New Project to Share Individual Patient Records. Susan Page of USA Today led the discussion on PCORnet: the National Patient-Centered Clinical Research Network, which is supported by the Patient-Centered Outcomes Research Institute (PCORI). Dr. Joe Selby, the executive director of PCORI, describes how PCORnet will take advantage of the incredible quantity of clinical information in electronic health records to conduct much-needed research on the relative effectiveness of different medical devices, drugs, and other medical therapies. Click here for a transcript of this program, or listen to the full episode below:

Listen now: The Promise and Perils of a New Project to Share Patient Records