July 13, 2018: Clinical Trial Data Sharing: Perspectives from Participants and PCORI (Michelle M. Mello, JD, PhD, Steven Goodman, MD, MHS, PhD)

Speakers

Michelle M. Mello, JD, PhD
Professor of Law, Stanford Law School
Professor of Health Research and Policy, Stanford University School of Medicine

Steven Goodman, MD, MHS, PhD
Associate Dean, Clinical and Translational Research
Professor of Medicine and Health Research and Policy
Stanford University School of Medicine
Vice-chair, PCORI Methodology Committee

Topic

Clinical Trial Data Sharing: Perspectives from Participants and PCORI

Keywords

Clinical research; Data sharing; Data metrics; Patient-Centered Outcomes Research Institute; PCORI; Patient participation; Patient perspective

Key Points

  • The presenters conducted a study that explored patients’ perspectives on being included in clinical trials. The study aimed to understand how patients feel about being part of trials and potential risks associated with the use of their clinical data.
  • Results from the study are published in “Clinical Trial Participants’ Views of the Risks and Benefits of Data Sharing” in the New England Journal of Medicine.
  • The majority of participants indicated that potential health benefits for their children or themselves is their primary reason for their clinical trial participation.
  • The majority of participants view the benefits of data sharing as greatly outweighing the potential risks.

Discussion Themes

The distinction between anonymous and de-identified data is an important one. Many people do not know what de-identified means and lack understanding that it is different from data being anonymous.

Data linkage is key in achieving data longevity. It would be interesting to extend some of the findings to better understand how the average American understands how their data could potentially be reused.

While the results of this study are not generalizable to those not actively participating in clinical trials, it is important to note that historical data have shown that most people prefer being asked to use their data for research purposes.

 

Tags

@Collaboratory1, @MichelleM_Mello, @goodmanmetrics, @NEJM, #clinicaltrial, #datasharing, #pctGR

June 29, 2018: Policy & Priorities: Rethinking University Research with State Data (Aaron McKethan, PhD)

Speaker

Aaron McKethan, PhD
Assistant Professor of Population Health Sciences, Duke School of Medicine
Senior Policy Fellow, Duke-Margolis Center for Health Policy
Chief Data Officer, NC DHHS

Topic

Policy & Priorities: Rethinking University Research with State Data

Keywords

State data; Policy implications; Research; Analytic priorities; Front-end technology

Key Points

  • In order to effectively incorporate state data in university research, policy implications must be considered at the beginning of the planning process.
  • There is a need for analytic priorities when determining how to rethink university research with state data.
  • What is known? What is not known? What are the highest-priority questions? These are the types of questions that must be asked when determining analytic priorities for the inclusion of state data in university research.
  • Using state data to help support university research provides better front-end technology, collaboration with community-based organizations, and more.

Discussion Themes

Being explicit about the type of analytics and the research questions we are trying to answer, will improve the process of better understanding how policy and priorities of the state can be used to impact university-level research.

To impact policy, we must be open to arrangements between the state and universities that will potentially generate research results may be hard to publish but could be widely impactful.

The notion of incorporating state data into university-level research is, in a way, related to the work of NIH Collaboratory and PCORnet. All entities are working towards making research more accessible and practical.

 

Tags

@Collaboratory1, @A_McKethan, @dukemargolis, #statedata, #opiod, #pctGR

June 22, 2018: Pragmatic Trial Design to Study Health Policy Interventions: Lessons Learned from ARTEMIS (Tracy Wang, MD, MHS, MSc)

Speaker

Tracy Y. Wang, MD, MHS, MSc
Director, DCRI Health Service Research
Fellowship Associate Program Director
Associate Professor of Medicine, Cardiology
Duke University Medical Center

Topic

Pragmatic Trial Design to Study Health Policy Interventions: Lessons Learned from ARTEMIS

Keywords

Clinical research; Pragmatic clinical trial; Pragmatic trial design; Health policy; ARTEMIS; Health policy; Health system; Cost-sharing models

Key Points

  • The ARTEMIS trial aims to improve patient outcomes by simulating health system and payer consideration of novel cost-sharing models.
  • Health policy and implementation studies require pragmatic trial design.
  • The ARTEMIS trial consists of 301 sites across the United States, with 23% classified as teaching hospitals.
  • Design and execution of the ARTEMIS trial prompted many questions, such as “Can we innovate the design of pragmatic health policy trials?”

Discussion Themes

As with most pragmatic clinical trials, the ARTEMIS trial is aimed at decision makers. However, in ARTEMIS, the “decision makers” are the healthcare systems, as well as the payers which is unusual as compared to most pragmatic clinical trials.

Variability in payment coverage contributed to the design of the ARTEMIS trial.

While the patient population in the ARTEMIS trial is analogous, the randomization of hospitals and the way in which the work of the hospitals is conducted is highly variable. Linking and data collection helped contribute to ARTEMIS findings.

 

Tags

@Collaboratory1, @TYWangMD, #pragmatictrial, #clinicaltrials, #pctGR, #EHR, @DCRnews

June 15, 2018: Research at Scale – Exploring What is Possible with High-Quality Real-World Data. Examples from Flatiron Health. (Amy P. Abernethy MD, MPH)

Speaker

Amy P. Abernethy MD, MPH
Chief Medical Officer, Chief Scientific Officer, SVP Oncology
Flatiron Health

Topic

Research at Scale – Exploring What is Possible with High-Quality Real-World Data. Examples from Flatiron Health.

Keywords

Clinical research; Clinical trials; Health data; Cancer research; Flatiron health; Real-world evidence; High-quality data; Real-world data

Key Points

  • Flatiron Health is evaluating the emerging role of data and technology to reimagine the clinical trials process from design through execution. Their technology is standardizing electronic health record data to a common data model, enabling millions of electronic health records in a single common dataset.
  • The 21st Century Cures Act presents an opportunity to use regulatory-grade real-world evidence. Characteristics of real-world data and real-world evidence have the potential to improve the clinical depth and completeness of health data as well as scalability and generalizability.

Discussion Themes

The addition of other linked datasets will provide a broader set of data and contribute to the expansion of use for other systems. The more datasets, the more “triangular” the sets will become. The more that high-quality real-world datasets are used in concert, the better.

Linkage to data claims is an integral piece of the high-quality real-world data puzzle. With large portions of oncological data, necessary records can be abstracted, providing simplicity in linking.

 

Tags

@Collaboratory1, @dramyabernethy, @flatironhealth, #clinicaltrials, #pctGR, #realworldevidence, #cancerresearch, #oncologist, #healthdata

June 8, 2018: Implementation in the PROVEN Trial: Challenges of Conducting PCTs in Nursing Home Health Care Systems (Susan Mitchell, MD, MPH; Angelo Volandes, MD, MPH)

Speakers

Susan Mitchell, MD, MPH
Senior Scientist, Institute for Aging Research, Hebrew Senior Life
Professor of Medicine, Harvard Medical School

Angelo Volandes, MD, MPH
Associate Professor of Medicine, Harvard Medical School
Division of General Internal Medicine, Massachusetts General Hospital

Topic

Implementation in the PROVEN Trial: Challenges of Conducting PCTs in Nursing Home Health Care Systems

Keywords

Clinical research; Clinical decision making; Healthcare; Medical systems; Patient experience; Clinical data; Pragmatic cluster trial; Randomized clinical trial; Advance care planning; Nursing homes; Quality assurance

Key Points

  • The objective of the PROVEN trial is to conduct a pragmatic cluster-randomized clinical trial of an advance care planning video shown to nursing home patients with advanced conditions in 2 nursing home healthcare systems.
  • Distribution of the nursing home sites for the PROVEN trial involves intervention and control centers and is far-reaching across the United States.
  • A primary challenge of the PROVEN trial is defining compliance and triaging long-stay patients.
  • Implementing interventions into nursing home healthcare systems in pragmatic clinical trials requires a mandate from senior management and endorsement in standard operating procedures.

Discussion Themes

The PROVEN trial is a first of its kind. Much has been discovered and additional lessons will be learned over time.

Reducing hospitalization served as an incentive for many of the PROVEN partnering organizations. However, the same incentive is used elsewhere in the market, so the potential of including some sort of financial incentive is something that has been discussed.

The advance care planning videos are intended to serve as a catalyst for better care. Patient and caretaker engagement is the ultimate goal. The videos help provide a more engaged and collaborative environment for patients and can better inform the medical team on why decisions are made by patients and their families.

Currently, there is a lack of the sites in the Midwest region due to the nature of the market.

 

Tags

@AngeloVolandes, #randomizedclinicaltrial, #nursinghome, #healthcaresystems, #pragmaticclinicaltrial, #pctGR, @Collaboratory1

June 1, 2018: How Would You Build an Academic Medical System to Bridge Research and Care if You Could Start from Scratch?

Speaker

S. Claiborne “Clay” Johnston, MD, PhD, MPH
Dean and Vice President for Medical Affairs
Dell Medical School, The University of Texas at Austin

Topic

How Would You Build an Academic Medical System to Bridge Research and Care if You Could Start from Scratch?

Keywords

Real-world evidence; Clinical research; Clinical decisions; Healthcare; Medical systems; Patient experience; Physicians; Clinical data

Key Points

  • About 20% of major clinical decisions are based on real-world evidence. This low percentage perpetuates an inconsistency between the research that is conducted and the care that is provided to patients.
  • The recently established Dell Medical School, at The University of Texas at Austin, aims to start from scratch by building a bridge between research and care. This effort involves a cycle of outcomes that includes payment for value, design, clinical expertise, team care, and technology.
  • To change the relationship between research and care for individuals, we must ask tough questions like, “Can we reduce physician distraction and improve the patient experience?”

Discussion Themes

Consent is often an underutilized area of routine care. Consent, through the work being done at Dell Medical School, is provided on a case-by-case basis, in addition to “blanket consent.”

New schools, like Dell Medical School, have the advantage to take bigger leaps in exploring the connection between research and care. Demonstrating what works and what doesn’t helps to create a business case for other schools and research entities.

Setting up a medical school in a nontraditional way has a large impact on medical students and residents; for example, the novel curriculum and increased interest in medical school applicants who exemplify leadership and creativity skills.

Key success factor: Prime everyone to truly care about improving health.

For more information on Dell Medical School, visit dellmed.utexas.edu  and follow @DellMedSchool.

 

Tags

@DellMedSchool, @ClayDellMed, #pctGR, @Collaboratory1

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