Tag Archives: NIH Collaboratory

Collaboratory Phenotypes, Data Standards, and Data Quality Core Releases Data Quality Assessment White Paper


The NIH Collaboratory’s Phenotypes, Data Standards, and Data Quality Core has released a new white paper on data quality assessment in the setting of pragmatic research. The white paper, titled Assessing Data Quality for Healthcare Systems Data Used in Clinical Research (V1.0) provides guidance, based on the best available evidence and practice, for assessing data quality in pragmatic clinical trials (PCTs) conducted through the Collaboratory. Topics covered include an overview of data quality issues in clinical research settings, data quality assessment dimensions (completeness, accuracy, and consistency), and a series of recommendations for assessing data quality. Also included as appendices are a set of data quality definitions and review criteria, as well as a data quality assessment plan inventory.

The full text of the document can be accessed through the “Tools for Research” tab on the Living Textbook or can be downloaded directly here (PDF).


Collaboratory Biostatistics and Study Design Core Releases Guidance Documents


The NIH Collaboratory’s Biostatistics and Study Design Core has released the first in a series of guidance documents focusing on statistical design issues for pragmatic clinical trials. Each of the four guidance documents are intended to help researchers by providing a synthesis of current developments in the field, discuss possible future directions, and, where appropriate, make recommendations for application to pragmatic clinical research.

The guidance documents are available through the Living Textbook and can be accessed on the “Tools for Research” tab or directly here.


New Living Textbook Chapter – Electronic Health Records-Based Phenotyping


A new Living Textbook topic chapter, “Electronic Health Records-Based Phenotyping,” has just been published. The chapter defines computable phenotypes and describes their role in data queries of electronic health records as part of pragmatic clinical trials. A main focus of the chapter is outlining mechanisms for identifying and evaluating phenotype definitions, with particular emphasis on standardization efforts of the NIH Collaboratory, including the Table 1 Project. Also included are links to recommended phenotype definitions from the Collaboratory Phenotypes, Data Standards, and Data Quality Core.


Dr. Califf to Speak Today at NIH Common Fund 10-Year Commemoration Symposium


On June 19, 2014, the NIH Common Fund is celebrating 10 years of achievement with a symposium, A Decade of Discovery, featuring its far-reaching research. Dr. Robert Califf, principal investigator of the NIH Collaboratory Coordinating Center, will speak during the 2:15-3:15 pm session “Reengineering the Clinical Research Enterprise.” Proceedings will be streamed live throughout the day.

View the agenda, live videocast, and commemorative book

Winners from the video and song contest will also be recognized.


Collaboratory Investigators Publish Article on Ethical and Regulatory Complexities for Pragmatic Clinical Trials in JAMA


“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.


NIH Common Fund Video Competition Goes Live


As part of its 10-year commemoration activities, the NIH Common Fund is holding a video and song competition to showcase Common Fund–supported activities in a fun and creative way. Grantees and program staff were encouraged to submit brief, original works that would be accessible and engaging to the public.

The video entries have been posted, and voting will be open until May 9. View the videos on YouTube and “like” your favorites to vote. Videos from NIH Collaboratory investigators and committee members include:

Song entries are still being screened.

Winners will receive recognition at the Common Fund 10-Year Commemoration Symposium in June 2014, where NIH Director Dr. Francis Collins will perform the winning song.


Grand Rounds (4-11-2014): A PCORnet Update

Update:

Archived video and slides from the April 11 Grand Rounds are now available on the NIH Collaboratory Grand Rounds webpage.


Today’s NIH Collaboratory and PCORnet Grand Rounds (“A PCORnet Update”) will be presented by Richard Platt, MD, MSc, professor and chair of the Department of Population Medicine at Harvard Pilgrim Health Care Institute. Dr. Platt is co-principal investigator of the Coordinating Center for the NIH Collaboratory, principal investigator of the Coordinating Center for PCORnet, and principal investigator of the FDA’s Mini-Sentinel program.

The Grand Rounds presentation will take place from 1:00-2:00 PM Eastern time on Friday, April 11. Details are available here. Archived video and slides from the presentation will be available early the following week; links to archived material will be provided in an update to this post.


Grand Rounds (3-26-2014): Why Is the FDA Interested in the Collaboratory and PCORnet?

Update:

Archived video and slides  from the March 26 Grand Rounds are now available on the NIH Collaboratory Grand Rounds webpage.

Photograph of FDA CDER Director Janet Woodcock, MD
CDER Director Janet Woodcock, MD. Photo credit: FDA

The guest speaker for this Friday’s Collaboratory/PCORnet Grand Rounds presentation will be the FDA’s Janet Woodcock, MD. Dr. Woodcock is the current director of the Center for Drug Evaluation and Research (CDER), the division of the FDA primarily responsible for evaluating the safety and effectiveness of both prescription and over-the-counter drugs and biologic therapies marketed in the United States.

The Grand Rounds presentation will take place from 1:00-2:00 PM Eastern time on Friday, March 26. Details are available here. Archived video and slide sets from the presentation will be available early the following week; links to archived material will be provided in an update to this post.


Grand Rounds (3-21-2014): The Use of Social Media in Clinical Research

Update:

Archived video and slides  from the March 21 Grand Rounds are now available on the NIH Collaboratory Grand Rounds webpage.


On Friday, March 21, the NIH Collaboratory and PCORnet Grand Rounds featured a presentation on “The Use of Social Media in Clinical Research” by Stephanie Startz, Associate Director of Digital Strategy for the Michael J. Fox Foundation. The Fox Foundation is a patient-focused nonprofit organization dedicated to finding a cure for Parkinson disease.

In December 2013, the Fox Foundation hosted a Twitter chat (#FoxChat) about challenges in clinical trial recruitment. The foundation’s blog notes that the discussion involved more than 90 participants and more than 600 tweets, making it one of Twitter’s hottest topics during that time. The Twitter chat, a full transcript of which is available online, engaged a diverse audience that included researchers, nonprofit organizations, patients, and patient advocacy groups, demonstrating that the Twitter chat format has the potential to elicit dialogue among these groups.

Ms Startz described the Fox Foundation’s other social media efforts on Facebook, Instagram, YouTube, etc, which have been successful in engaging the patient community and encouraging participation in clinical research. For example, a 3-day campaign on Facebook led to more than 1800 visitors to the Fox Trial Finder website. Advice for engaging patients via social media included being social by sharing stories and videos (not just requests for action), avoiding jargon, and responding to users. The Fox Foundation shares firsthand patient experiences, gives updates on trials, and thanks participants using social media. The Grand Rounds discussion covered issues of privacy and the extent to which social media could potentially play a role in involving patients in research design, not just research participation.


Changes to Informed Consent in the Era of Learning Healthcare Systems


In 2007, a seminal report from the Institute of Medicine (IOM) threw a sharp spotlight on a series of problems facing the broader U.S. healthcare system:

Evidence on what is effective, and under what Cover page of IOM report - The Learning Healthcare Systemcircumstances, is often lacking, poorly communicated to decision makers, or inadequately applied, and despite significant expenditures on health care for Americans, these investments have not translated to better health.

—The Learning Healthcare System (IOM Workshop Summary), 2007

The IOM report called for a new approach to closing the existing gaps in patient care, one that would create a system in which patient care, quality improvement efforts, and clinical research would exist as integrated components within a virtuous cycle of feedback—the “learning healthcare system.” (The original report, plus a series of related reports [PDF], can be read for free online at the National Academies Press website.)

In the years since The Learning Healthcare System was first published, researchers, healthcare providers, health systems, and governmental and regulatory agencies have all struggled with the monumental task of building such a system. One aspect that has presented a particularly complex set of challenges centers on the issue of informed consent. The principle of informed consent—the idea that all patients have the right to make a fully informed decision, free from coercion or other undue pressure, about whether or not to participate in research—is a foundational tenet of clinical research ethics. However, some of the tools that are widely considered to be critical to the success of achieving a workable learning healthcare system, such comparative effectiveness studies and cluster-randomized trials, are difficult or even impossible to conduct under existing models of individual informed consent.

Recent efforts from the Ottawa Consensus Statement Group and a series of articles published in an issue of the Hastings Center Report have explored explored informed consent in such circumstances. These are now joined by a pair of articles published in the February 20, 2014 issue of the New England Journal of Medicine. In the first, Faden and colleagues outline a case for streamlining or even dispensing with individual informed consent in certain kinds of randomized comparative-effectiveness or quality improvement studies that present a minimal risk of harm to patients [1]. The authors also describe the larger framework that would provide transparent and accountable oversight of such studies, as well as overseeing the integration of findings from such research into the patient-care process.

The second article, by Kim and Miller, presents a different vision for informed consent,  the “Integrated Consent Model” [2]. Unlike Faden and colleagues, the authors argue for preserving the element of informed consent for all randomized pragmatic research, even in circumstances considered to pose minimal risk to patients. Kim and Miller suggest that such a model, in which the prospect of participating in research is offered as part of the general clinical discussion about treatment options and documented by the physician, will satisfy ethical imperatives for informing patients while remaining sufficiently streamlined to meet the demands of pragmatic clinical research. They also argue that an “integrated consent” approach could be accommodated under existing regulations.

Regardless of whether either or both of these perspectives can be incorporated into the emerging learning healthcare system, the larger questions surrounding informed consent are  garnering significant interest, as evidenced by the response to the Department of Health and Human Service’s 2011 call for public comment on a proposal to modify rules governing clinical research. And with the proliferation of new technologies and trial designs that use data extracted directly from patient’s electronic health records (such as the demonstration projects coordinated through the NIH Collaboratory) and the emergence of innovative networks devoted to pragmatic patient-centered research, the need for a solution both protects patients and enables vitally needed research will only continue to grow.

For additional perspective on the recent publications in the New England Journal of Medicine, see "Research Permissions–Angels on the Head of a Pin, or the Key Issue to Decipher?" by NIH Collaboratory PI Dr. Rob Califf.

References


1. Faden RR, Beauchamp TL, Kass NE. Informed consent, comparative effectiveness, and learning health care. N Engl J Med 2014;340:766-768. 10.1056/NEJMhle1313674.

2. Kim SYH, Miller FG. Informed consent for pragmatic trials — The integrated consent model. N Engl J Med 2014; 370:769-772. doi: 10.1056/NEJMhle1312508.