May 23, 2018: New Podcast: Dr. Rachael Fleurence on Launching the NESTcc Data Network to Improve the Use of Real-World Evidence in the Medical Device Ecosystem

In a new episode of the Grand Rounds podcast which is now available, Dr. Rachael Fleurence, Executive Director of the National Evaluation System for health Technology Coordinating Center (NESTcc) and the Medical Device Innovation Consortium (MDIC), discusses the mission, challenges, and long term goals of NESTcc with moderator Dr. Adrian Hernandez. Dr. Fleurence describes how NESTcc is working to accelerate the development and translation of new and safe health technologies, and explains where she hopes the program will be in the next three years.

Listen to the episode here:

At least once a month, we will release interviews with Grand Rounds speakers that delve into their topic of interest and give listeners bonus time with these featured experts.

Please let us know what you think by providing your feedback through the podcast page. We also encourage you to listen and share the recordings with your colleagues!

May 18, 2018: The PCORnet Antibiotics and Childhood Growth Study: Using PCORnet Data for Longitudinal Observational Research

Speaker

Christopher B. Forrest, MD, PhD
Professor, Pediatrics & Health Care Management
PI, PEDSnet, A National Pediatric Learning Health System
Director, Center for Applied Clinical Research, Children’s Hospital of Philadelphia and University of Pennsylvania

L. Charles Bailey, MD, PhD
Associate Professor of Clinical Pediatrics
Perelman School of Medicine
Children’s Hospital of Philadelphia and University of Pennsylvania

Jason Block, MD, MPH
Assistant Professor, Department of Population Medicine
Harvard Medical School, Harvard Pilgrim Health Care Institute
PI, PCORnet Antibiotics and Childhood Growth Study

Topic

The PCORnet Antibiotics and Childhood Growth Study: Using PCORnet Data for Longitudinal Observational Research

Keywords

PCORnet; Antibiotics; Childhood obesity; Weight outcomes; Antibiotic association; Growth; Common data model; Demonstration project; Observational research

Key Points

  • PCORnet is a national infrastructure for people-centered clinical research. PCORnet embodies a “network of networks” that harnesses the power of partnerships.
  • The PCORnet Antibiotics and Childhood Growth Study provided an opportunity to “test” the capabilities of PCORnet in determining how the network would aid in providing data related to an important research question.
  • The PCORnet Antibiotics Study aimed to assess the association between antibiotic use before age 2 and childhood weight outcomes.
  • The Antibiotics Study helped a team of investigators better understand parents’ and providers’ beliefs about risks and benefits of antibiotic use, while also understanding how potential risks of future obesity could be integrated into parents’ and providers’ decision making when discussing the use of antibiotics.
  • 28 PCORnet partners and 36 healthcare institutions contributed to the Antibiotics and Childhood Growth Study.

Discussion Themes

The more PCORnet’s capabilities are utilized by other studies, the easier it will be to use. While there are still some challenges, a consistent effort to continue utilizing the network will make the benefits more plentiful and far-reaching.

The PCORnet Antibiotics Study was funded for 2 years and was able to include data from 362,000 children. Because the data are captured systematically, PCORnet is able to dramatically impact the way studies are conducted and the amount of progress that can be done in a short amount of time. While there is still progress to be made around institutional attribution, among other things, incremental improvements are being made and benefits are being seen.

Some areas for potential improvement for PCORnet include an agreement to make contributions to one another’s research through support of and contribution to the network, an extension of the Common Data Model, and solving the multi-institutional contracting dilemma.

For more information on the PCORnet Antibiotics and Childhood Growth Study, visit www.pcornet.org and follow @PCORnetwork.

 

Tags

@jasonpblock, @PCORnetwork, #pctGR, @Collaboratory1

May 11, 2018: Launching the NESTcc Data Network to Improve the use of Real-World Evidence in the Medical Device Ecosystem

Speaker

Rachael Fleurence, PhD
Executive Director, National Evaluation System for health Technology Coordinating Center (NESTcc)
Medical Device Innovation Consortium (MDIC)

Topic

Launching the NESTcc Data Network to Improve the use of Real-World Evidence in the Medical Device Ecosystem

Keywords

NESTcc; Unique device identification; Real-world evidence; Clinical research; Medical device; Clinical data; Technology; Innovative research; Data models

Key Points

  • The mission of NESTcc is to accelerate the development and translation of new and safe technologies to leverage real-world evidence and innovative research.
  • NESTcc is building a sustainable network of collaborators committed to advancing real-world evidence generation.
  • NESTcc is launching 4 subcommittees to aid in establishing its value in the medical device ecosystem: the Charter Committee, the Sustainability Committee, the Data Quality Committee, and the Methods Committee.
  • NESTcc data currently represents more than 400 million records.

Discussion Themes

Unique device identification (UDI) will aid researchers in determining the type of device that is being used. Over the next few years, health systems will likely realize the benefits of using UDI, not only for research but for operations of care. Because device manufacturers have their own registries, it is possible to make linkages with health system data.

NESTcc has ambitious goals. The key is to be able to do proof of concept for a small number of devices that are scalable to others.

The integration of multiple data models provides NESTcc with flexibility, allows for more cohesion in determining the way to optimize, and allows NESTcc to build on expertise.

NESTcc plans to provide proof of concept within the next 2 years, and if successful will be scalable within 5 years.

 

For information on NESTcc, visit https://nestcc.org/ and follow @NESTccMedTech on Twitter.

Tags

@NESTccMedTech, @Fleurence, @MDIConline, @Collaboratory1, #pctGR, #RealWorldEvidence, #MedicalDevices

May 4, 2018: Leveraging Community Engagement and Informatics-Based Tools to Increase Participant Recruitment and Retention

Speakers

Paul A. Harris, PhD
Director, Office of Research Informatics
Professor, Department of Biomedical Informatics & Department of Biomedical Engineering Vanderbilt University

Consuelo H. Wilkins, MD, MSCI
Executive Director
Meharry-Vanderbilt Alliance

Patricia Jones, DrPH, MPH
Program Director
National Center for Advancing Translational Sciences (NCATS)

Topic

Leveraging Community Engagement and Informatics-Based Tools to Increase Participant Recruitment and Retention

Keywords

Clinical Research; National Center for Advancing Translational Sciences; NCATS; Public health; Trial Innovation Network; Recruitment Innovation Center; Clinical and Translational Science Awards; CTSA; Community engagement; Participant retention; Health informatics

Key Points

    • The National Center for Advancing Translational Sciences aims to improve health through smarter science.
    • Moving from scientific discovery to true public health benefit requires multisite collaboration, impactful dissemination, and smarter use of data by clinicians and patients.
    • The mission of the NIH-supported Trial Innovation Network (TIN) is to develop, demonstrate effectiveness of, and disseminate scientific and operational innovations that increase the efficiency of clinical trials.
    • Novel study designs with compelling endpoints and data-driven approaches create innovation in the execution of clinical trials.
    • The Vanderbilt University Medical Center Recruitment Innovation Center (RIC) was developed to improve participant enrollment and retention in multicenter clinical trials, including enrollment and retention of diverse populations.

Discussion Themes

Clinician engagement can be a challenge with informatics-based tools. To increase engagement, researchers should expand their attention to clinical staff so that they feel empowered to share study-related information. Staff-level engagement techniques include sharing flyers and conducting screenings.

The Faster Together project, an RIC supplement, will test innovative approaches within trials by including culturally tailored messaging, plans created with minority communities, and approaches to engage minorities and marginalized communities in clinical trials. To test effectiveness, Faster Together will randomly expose minority participants to tailored materials versus non-tailored materials, with a blind assessment of the impact on participant accrual.

There will potentially be far-reaching implications resulting from the Common Rule changes, which the Clinical and Translational Science Awards (CTSA) Program is well positioned to address.

 

For information on NCATS, follow @ncats_nih_gov and for more information on Vanderbilt RIC, check out the RIC website.

Tags

@Collaboratory1, @ncats_nih_gov, @VUMChealth, #clinicaltrials, #publichealth, #CTSAProgram, #publichealth, #pctGR

April 27, 2018: Expanding Use of Real-World Evidence: A National Academies Workshop Series

Speaker

Greg Simon, MD, MPH
Senior Investigator
Kaiser Permanente Washington Health Research Institute

Topic

Expanding Use of Real-World Evidence: A National Academies Workshop Series

Keywords

Clinical research; Real-world evidence; Real-world data; Pragmatic research

Key Points

  • Real-world evidence (RWE) should be generalizable, relevant, adaptable, and efficient.
  • Are our research traditions vital anchors to our central purpose? Or just anchors that keep us stuck?
  • Real-world data (RWD) does not always make real world evidence (RWE), but RWE usually starts with RWD.
  • The real-world evidence (RWE) mantra is “faster, better, cheaper.”

Discussion Themes

The third workshop will be held in July of 2018 and will be focused on generating useful tools that stakeholders might use regarding when and how to use real-world evidence.

Is the term “real world evidence” starting to have so many meanings that it is losing meaning?  It is a broad term that includes real-world data, real-world treatment, and real-world treatment assignment.

It is essential to make a clear decision to define usual care, assess it during a trial, and report on it. The question is how much do researchers try to control usual care? There are situations when there is an ethical obligation to provide a certain level of care.

Transparency regarding methods and intermediate data steps is necessary for credibility.

 

For information on the real-world evidence workshops, visit http://bit.ly/RWEworkshop1 and http://bit.ly/RWEworkshop2 

Tags

@Collaboratory1, @GregSimonKPWHRI, #realworldevidence, #realworlddata, #pragmatictrial, #pctGR

April 20, 2018: The Trauma Survivors Outcomes and Support (TSOS) Trial as an ePCT Training Case Study

Speaker

Doug Zatzick, MD
TSOS Principal Investigator
Professor, Department of Psychiatry and Behavioral Sciences
University of Washington School of Medicine

Topic

The Trauma Survivors Outcomes and Support (TSOS) Trial as an ePCT Training Case Study

Keywords

Clinical research; TSOS; Trauma Registry; Embedded pragmatic trial; Stepped-wedge design; Cluster-randomized trial; Pragmatic trial; PRECIS-2

Key Points

  • Trauma Survivors Outcomes and Support (TSOS) was designed as a stepped-wedge, cluster-randomized pragmatic trial at 25 trauma centers in the United States, with 935 patients consented so far.
  • Researchers’ hypotheses for the intervention group versus the control group included decreased post-traumatic stress disorder (PTSD), lower rates of depression and suicidal ideation, and less alcohol abuse.
  • The advantage of a stepped-wedge design is the mitigation of site variability as sites contribute patients to both control and intervention conditions.
  • The TSOS trial targets real-world decisions and is generalizable to sites, patients, and providers, but tradeoffs include regulatory intensity and ongoing development of pragmatic follow-up assessments.

Discussion Themes

Everyone in TSOS begins as a control participant, and TSOS training at trauma centers includes assessing these controls and linking them with primary care if needed.

It is important to do empiric research, such as asking participants “Had you known in advance what study participation would be like, would you still participate?”

Researchers hope that after TSOS ends, some of the electronic screening and consent documents will remain at sites. 

In the startup year of TSOS, regulatory issues were key in getting trauma registry data from patients, and researchers hope to have access to this data as the trial progresses.

One challenge of stepped-wedge design is that phased roll-out limits trial flexibility particularly with regard to recruitment pauses.

 

For information on the TSOS trial, visit The Living Textbook https://bit.ly/2vp1hJX

Tags

@Collaboratory1, @DougZatzickMD , #steppedwedge, #depression, #PTSD, #clinicalcare, #pragmatictrial, #ePCT, #pctGR

April 17, 2018: New Podcast: Dr. George Hripcsak on OHDSI and Drawing Reproducible Conclusions From Observational Clinical Data

A new episode of the Grand Rounds podcast is now available, featuring Dr. George Hripcsak, Chair of the Bioinformatics Department at Columbia University. In this episode, Dr. Hripcsak discusses the Observational Health Data Sciences and Informatics (OHDSI) Program with moderator Dr. Adrian Hernandez, and describes the program’s vision to create accessible and reliable clinical evidence by analyzing the clinical data of millions of patients globally. Dr. Hripcsak also talks about how listeners can “Join the Journey” and become part of the OHDSI research network, helping to generate high-quality evidence needed to inform medical decision-making.

Listen to the episode here:

At least once a month, we will release interviews with Grand Rounds speakers that delve into their topic of interest and give listeners bonus time with these featured experts.

Please let us know what you think by providing your feedback through the podcast page. We also encourage you to listen and share the recordings with your colleagues!

April 13, 2018: OHDSI: Drawing Reproducible Conclusions from Observational Clinical Data

Speaker

George Hripcsak, MD, MS
Vivian Beaumont Allen Professor of Biomedical Informatics, Columbia University
Chair, Department of Biomedical Informatics, Columbia University
Director, Medical Informatics Services, NewYork-Presbyterian Hospital/Columbia

Topic

OHDSI: Drawing Reproducible Conclusions from Observational Clinical Data

Keywords

Clinical research; Observational research; Precision medicine; Electronic health records; OHDSI; Clinical data

Key Points

  • Observational Health Data Sciences and Informatics (OHDSI) was developed to improve health by empowering communities to collaboratively generate the evidence that promotes better health decisions and patient care.
  • Patient-level predictions for personalized evidence requires big data. OHDSI provides a global network of at least a billion open-source records on more than 400 million patients in 80 databases.
  • It is feasible to encode the world population in a single data model if results are shared through a multi-stakeholder, interdisciplinary approach.
  • Through access to observational data, researchers can look at multiple outcomes or treatments at once to produce large numbers of comparisons.

Discussion Themes

There are five steps in reproducible research:

  1. Address measured confounding variables
  2. Identify residual confounding variables
  3. Use multiple databases, locations, and practice types
  4. Publish hypotheses, parameters, and runs
  5. Generate evidence

Data dredging is not about what researchers do, but about what they throw out, so it should not be done in the literature. Every last parameter should be published so a study can be reproduced.

There is an “upside down” approach to the problem of multiple comparisons and selective publication, by telling researchers that all comparisons are required.

People get their care from multiple institutions, so OHDSI is looking for ways to ensure that outcomes are not left out. Administrative databases tend to be more complete in terms of longitudinal information.

NCDR cardiovascular registry data, certain labs, radiology/path notes, and sociodemographic information are all key elements that are being considered as sources to enrich observational research.

 

To learn more about OHDSI, visit http://ohdsi.org or follow @OHDSI on Twitter.

For information on electronic health record (EHR) phenotyping, visit The Living Textbook https://bit.ly/2Hk5pQ8.

Tags

@PCORI, @Columbia, #clinicaldata, #observationaldata, #PrecisionMedicine, #openscience, #jointhejourney, #pctGR

April 11, 2018: New Podcast: Dr. Robert Califf on Data Science in the Era of Data Ubiquity

The NIH Collaboratory is pleased to announce that the new episode of the Grand Rounds podcast is now available, featuring Dr. Robert Califf, former FDA Commissioner and current Director of Duke Forge. In this episode, Dr. Califf discusses with moderator Dr. Adrian Hernandez how the vast amount of information now available to people through cell phones and other devices is both an opportunity and obstacle for the field of data science.  While improving technology provides the chance to equalize access to health resources and knowledge for people across the country, it is also creates the challenge of ensuring that this information is complete and accurate.

Listen to the episode here:

At least once a month, we will release interviews with Grand Rounds speakers that delve into their topic of interest and give listeners bonus time with these featured experts.

Please let us know what you think by providing your feedback through the podcast page. We also encourage you to listen and share the recordings with your colleagues!

April 6, 2018: Legal and Ethical Architecture for PCOR Data

Speakers

Jane Hyatt Thorpe, JD
Associate Professor
Department of Health Policy and Management
Milken Institute School of Public Health
George Washington University

Lara Cartwright-Smith, JD, MPH
Associate Research Professor
Department of Health Policy and Management
Milken Institute School of Public Health
George Washington University

Elizabeth Gray, JD, MHA
Research Scientist
Department of Health Policy and Management
Milken Institute School of Public Health
George Washington University

Topic

Legal and Ethical Architecture for PCOR Data

Keywords

Patient-centered outcomes research; PCOR; Data privacy and security; Research ethics

Key Points

  • The patient-centered outcomes research (PCOR) Privacy and Security Research Scenario Initiative supports the development of a legal, ethical architecture to enable robust and secure PCOR data.
  • The legal and ethical framework was created to foster stakeholder trust through compliance with federal and state privacy laws.
  • The framework is a visual decision tool highlighting key characteristics and considerations associated with the spectrum of data used for PCOR and the nature of the relationships between researchers and other stakeholders.
  • The framework directs stakeholders through considerations organized into relevant legal provisions according to 6 key data characteristics:
    • Identifiability and Content
    • Subject
    • Source
    • Access and Use/Purpose
    • Consent/Authorization
    • Security

Discussion Themes

While the PCOR framework is not intended to replace a conversation with individual counsel, it should provide guidance to researchers on the types of questions to ask about their data.

The framework’s appendix was created to organize issues that may have ambiguous or overlapping legal authority.

Ethical considerations were embedded in the document, rather than being broken out separately apart from legal considerations, as they often go hand in hand (e.g., informed consent considerations).

 

To see the full framework documents, visitHealthIT.gov https://bit.ly/2H2RLke 

For more about regulatory frameworks, visit The Living Textbook  https://bit.ly/2GG3sOr

Tags

@PCORI, @ @ONC_HealthIT, @HHSONC, @GWtweets, #PCOR, #stakeholderengagment, #pctGR