March 15, 2019: Leveraging RWE to Support Regulatory Decisions–An Update on Efforts to Inform Policy (Gregory W. Daniel, PhD, MPH, RPh)

Speaker

Gregory W. Daniel, PhD, MPH, RPh
Deputy Center Director, Duke-Margolis Center for Health Policy
Clinical Professor, Fuqua School of Business
Duke University

Topic

Leveraging RWE to Support Regulatory Decisions–An Update on Efforts to Inform Policy

Keywords

Real world evidence; Health care system; Regulatory; Data curation; Policy making; FDA

Key Points

  • Real world data and real world evidence are not the same thing. Real world evidence includes observational and randomized designs while real world data does not.
  • While real world data and real world evidence can support important health care system questions, it does not yet inform regulatory decisions.
  • The U.S. Food and Drug Administration (FDA) real world evidence program aims to evaluate potential uses of real world evidence to support drug label changes and product effectiveness.
  • Several planned guidances on real world data and real world evidence released by the FDA are used to support regulatory decision making.

Discussion Themes

Data curation is often complex and hard to explain.

Two key components of dependable data are adequacy and control. Adequacy of data can easily be determined, but within a real world evidence system, determining if data is well controlled can be more challenging. A better understanding of what study designs will help in arriving at the most trustworthy data is paramount in effectively leveraging real world evidence.

Immediate next steps for applying FDA RWE guidance includes determining what the best practices for commonalities in data curation are. It’s also important to explore what has been done in the past and determine where problems lie and what can be done to solve them.  

Research design and methods play a key role in determining the best way of determining the reliability of data and how it can be leveraged in a clinical practice setting.

Tags

#realworldevidence, #pctGR, #healthcaresystem, #regulatory, @gregwdaniel, @dukemargolis, @collaboratory1

March 8, 2019: Dietary Trials in Heart Failure: SODIUM-HF (Justin Ezekowitz, MBBCh, MSc)

Video coming soon

Speaker

Justin A. Ezekowitz, MBBCh, MSc
Professor, University of Alberta
Co-Director, Canadian VIGOUR Centre
Cardiologist, Mazankowski Alberta Heart Institute

Topic

Dietary Trials in Heart Failure: SODIUM-HF

Keywords

Multicenter clinical trial; Heart failure; Dietary sodium; Cardiovascular health; Clinical equipoise; Clinical guidelines; SODIUM-HF

Key Points

  • The SODIUM-HF trial is a multicenter, multinational dietary study evaluating the long-term effects of a low-sodium diet in patients with heart failure on a composite clinical outcome of all-cause mortality, cardiovascular (CV) hospitalizations, and CV emergency department visits.
  • The recommended amount of dietary sodium for patients with heart failure varies among the guidelines, ranging from no restriction to <2300mg per day.
  • Among the successes of the study are the important research question, a simple electronic case report form, engaged clinicians and staff, 100% remote monitoring, and a low administrative burden.

Discussion Themes

Sodium restriction is one of the most challenging things for patients to undertake as it affects their lifestyle significantly.

What is your perspective on the importance of a low-sodium diet for patients with heart failure, given that low sodium was shown to be harmful in some studies?

How do you balance wanting a pragmatic/practical intervention versus wanting a rigorous test of your hypothesis?

For more information on this study, visit the SODIUM-HF trial website and ClinicalTrials.gov.

Tags

#HeartFailure, #pctGR, @SodiumHF, @Collaboratory1, @JustinEzekowitz, @CVC_UAlberta

March 1, 2019: Approaches to Patient Follow-Up for Clinical Trials: What’s the Right Choice for Your Study? (Keith Marsolo, PhD)

Speaker

Keith Marsolo, PhD
Department of Population Health Sciences
Duke Clinical Research Institute
Duke University School of Medicine

Topic

Approaches to Patient Follow-Up for Clinical Trials: What’s the Right Choice for Your Study?

Keywords

Pragmatic clinical trial; Real-world data; Distributed research network; Electronic health records; EHR; Health data sources; Data standardization; Common data model; Fast Healthcare Interoperability Resources (FHIR); Application programming interface (API)

Key Points

  • Different sites have different capabilities and levels of sophistication around data. Clinical trial investigators should think from the beginning about the questions they want to answer and how much data is needed.
  • From different sources, such as the EHR, claims, or participant, data can be procured and provided in different ways, either by the patient, staff or clinician, or through IT and data experts.
  • PCTs with many sites may require a “patchwork quilt” of approaches for patient follow-up depending on the needs of the trial. Clinician-generated reports, direct from patients, and solutions involving application programming interfaces (APIs) are all good options for data exchange.

Discussion Themes

How do we think through the options for getting patient data where some sites may not be in the distributed research network or use a common data model?

Fast Healthcare Interoperability Resources (FHIR) is a draft standard describing data formats and elements and an application programming interface (API) for exchanging electronic health records. The FHIR interface requests data as an object, and for each defined domain it specifies allowable values and variables and predefines the information that you get out of the system.

Until data are collected/generated using the same standards/formats as the API, there will still be a need to understand the EHR-to-interface mapping.

For more information on using health data in embedded pragmatic clinical trials, visit the NIH Collaboratory’s EHR Core webpage.

Tags

#CommonDataModel, #RealWorldData, #FHIR, #pctGR, @Collaboratory1

February 22, 2019: Going from a Learning Health System to Learning Health Units: Opportunities and Challenges (Adrian Hernandez, MD, MHS)

Speaker

Adrian F. Hernandez, MD, MHS
Vice Dean for Clinical Research
Duke University School of Medicine

Topic

Going from a Learning Health System to Learning Health Units: Opportunities and Challenges

Keywords

Pragmatic clinical trial; Learning health system; Health data; Electronic health records; Health risk scores; Health system research

Key Points

  • In a learning health system, it is important to seek feedback and insight from patients, clinicians, and data scientists.
  • Key attributes for an effective learning health system are culture, investment, data, research, and results.
  • The goal of transitioning to learning health units from a comprehensive learning health system are not yet being reached, but there is promise for the future.

Discussion Themes

It’s important to determine what standard of evidence is going to be good enough to drive clinical decision making, and we need to be prepared to understand when lower quality data is good enough.

Finding opportunities for curating patient health records, while also establishing a concordance about what goes into a record and what does not, should be a focus of learning health systems.

In many cases, health risk scores are not adequately shared with patients. Conversations surrounding the ethics of communicating health risk scores should take place. However, there is often a level of uncertainty, so it’s important to communicate in a way that establishes full transparency and understanding.

To make a big difference, we need to rapidly move from learning health systems to learning health communities.

Tags

#pctGR, #clinicaltrial, #learninghealthsystem, #data, #analytics, #healthsystem, @texhern, @DCRInews, @DukeMedSchool

February 15, 2019: Improving Qualification of Investigators: Recommendations from the Clinical Trials Transformation Initiative (Christine M. Hildebrand, PA-C, Janette Panhuis)

Speakers

Christine M. Hildebrand, PA-C
Physician Assistant
Sub-Investigator
Clinical Operations Lead
Amici Clinical Research

Janette Panhuis
Chief Operating Officer
Population Health Research Institute
David Braley Research Institute

Topic

Improving Qualification of Investigators: Recommendations from the Clinical Trials Transformation Initiative

Keywords

CTTI; Investigator qualifications; Investigator training; Site team training; Good clinical practice; Clinical trials

Key Points

  • There is little evidence that good clinical practice (GCP) training alone sufficiently qualifies investigators in the conduct of clinical trials. What is needed is a targeted and risk-based approach to educating clinical trial investigators and their delegates in GCP principles.
  • A culture shift is needed that eliminates the distinction between “qualification” and “preparation” and moves toward investigators and their delegates assuming greater ownership of training and documenting evidence of their qualification.
  • CTTI recommendations outline how to confirm that site teams are qualified while also reducing inefficiencies in training and increasing preparation for successful study execution.

Discussion Themes

Investigators and site teams come to the table with different levels of research experience, training, and credentials. How can we effectively address gaps in skills and knowledge of GCP principles?

With respect to pragmatic trials conducted within healthcare systems, are there approaches to site monitoring that address issues related to turnover of PIs and clinical and research staff?

Greater ownership of GCP training and qualification by investigators and delegates can lead to active remediation of deficiencies at the clinical site.

CTTI recommendations are meant to be adaptable to the protocol, even when conducted in a real-world clinical setting using electronic health records.

For recommendations and tools for improving investigator qualification, visit CTTI’s Investigator Qualification website.

Tags

#ClinicalTrials, #GCPtraining, #pctGR, @Collaboratory1 @CTTI_Trials

February 8, 2019: Paramedic Airway Management in Out-of-Hospital Cardiac Arrest – The Pragmatic Airway Resuscitation Trial (PART) (Henry Wang, MD, MS)

Speaker

Henry E. Wang, MD, MS
Professor and Vice Chair for Research
Department of Emergency Medicine
University of Texas Health Science Center at Houston

Topic

Paramedic Airway Management in Out-of-Hospital Cardiac Arrest – The Pragmatic Airway Resuscitation Trial (PART)

Keywords

Pragmatic clinical trial; Airway resuscitation; Prehospital intubation; Endotracheal intubation; CPR; Informed consent

Key Points

  • The primary purpose of The Pragmatic Airway Resuscitation Trial (PART) was to determine if 72-hour survival after out-of-hospital cardiac arrest is improved with initial endotracheal intubation (ETI) over initial laryngeal tube (LT) airway management strategies.
  • PART was a randomized, interventional trail that included 3,004 enrolled participants and 3,000 participants in the primary analysis.
  • There were no significant differences in oropharyngeal or hypopharyngeal injury, airway swelling, or pneumonia for participants that received ETI over LT.

Discussion Themes

When conducting a clinical trial, seeking informed consent from trial participants is of paramount importance. In the case of PART, written informed consent was not possible because the trial included unconscious participants in an emergency situation. Therefore for PART, Exception from Informed Consent Requirements for Emergency Research (EFIC), as mandated by the U.S. Food and Drug Administration (FDA), was applied. Learn more about EFIC.

Understanding the importance of randomized trial data and having it available for out-of-hospital emergency medicine services is an idea that will continue to be discussed in the field of pragmatic clinical  research.

In a trial like PART, it is difficult to blind the interventional group. Randomization was used to gain reliable data and to compensate for the lack of blinding. In an airway trial, this is one of the accepted limitations of the trial design.

Tags

#pctGR, #clinicaltrials, #informedconsent, #resuscitation, #CPR, @Collaboratory1. @UTHealth, @henrywangmd

February 1, 2019: Promoting Effective Advance Care Planning Communication in the Elderly: The ACP-PEACE Trial (James Tulsky, MD, Angelo Volandes, MD, MPH)

Speakers

James Tulsky, MD
Chair, Department of Psychosocial Oncology and Palliative Care
Dana-Farber Cancer Institute
Professor of Medicine, Harvard Medical School
Chief, Division of Palliative Medicine
Brigham and Women’s Hospital

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

Topic

Promoting Effective Advance Care Planning Communication in the Elderly: The ACP-PEACE Trial

Keywords

Pragmatic clinical trial; Advance care planning; ACP PEACE; Dana-Farber Cancer Institute; National Institute on Aging; Palliative care; Video declarations; Goal-concordant care; Patient preferences

Key Points

  • Many people with serious illness die without receiving goal-concordant care, and patients over the age of 65 with cancer experience this disproportionately. Helping patients engage in advance care planning (ACP) can empower them to express and record their goals so that their care can be aligned with their preferences.
  • The ACP PEACE Demonstration Project is a pragmatic, stepped-wedge, randomized trial of a comprehensive ACP program in oncology clinics at 3 health systems. It will involve a combination of 2 evidence-based programs:
    • VitalTalk teaches clinicians important communication skills in having empathic conversations with seriously ill patients.
    • ACP Decisions uses videos to promote planning and decision-making by patients and families.
  • The ACP PEACE study will monitor long-term outcomes to evaluate whether patients received the care they planned for and wanted.

Discussion Themes

The last element of the ACP PEACE trial is a video declaration (ViDec), recorded by a subset of patients. In recording the ViDec, patients are prompted by questions assessing their confidence with their decision, satisfaction, decisional regret, and patient-provider experience.

The ACP PEACE study team has a scaling strategy in place if the intervention proves effective. Implementing the intervention as standard of care will involve a culture shift from what is currently expected in health systems.

Read more about the ACP PEACE Demonstration Project in the Living Textbook.

Tags

#AdvanceCarePlanning, #pctGR, @Collaboratory1 @VitalTalk, @ACPDecisions

January 25, 2019: A Learning Health System Story: Perinatal Outcomes Associated with a Major Change in Gestational Diabetes Screening (Sascha Dublin, MD, PhD, Gaia Pocobelli, PhD)

Speakers

Sascha Dublin, MD, PhD
Senior Investigator
Kaiser Permanente Washington Health Research Institute

Gaia Pocobelli, PhD
Research Associate
Kaiser Permanente Washington Health Research Institute

Topic

A Learning Health System Story: Perinatal Outcomes Associated with a Major Change in Gestational Diabetes Screening

Keywords

Health system; Gestational diabetes; Perinatal outcomes; Electronic health records; Learning health system

Key Points

  • In a learning health system, research influences practice, and practice influences research.
  • Gestational diabetes (GDM) is common, with a prevalence of 4% to 18% in pregnant women, depending upon population and testing strategy. This is why it’s important that the GDM screening process be demystified to determine which testing strategy approach is best.
  • While screening for and treating GDM to avoid adverse outcomes for women and babies is widely accepted, it is not clear which screening approach is best. Different organizations and professional societies have endorsed different approaches.
  • The presenters proposed a 1-step approach for GDM screening, including a 2-hour screening and lowered threshold to qualify for GDM. Electronic health records and linked state birth certificates served as the primary data sources in determining perinatal outcomes associated with changes to GDM screening.
  • Overall, there was no evidence of a benefit to the 1-step approach to GDM screening and diagnosis. Study findings were disseminated and led to practice change.

Discussion Themes

There is a plan to study de-implementation of the 1-step approach for GDM screening. This will help to show which changes had an impact. It will be interesting to compare the complexity of implementation versus de-implementation.

Also important will be the cost analysis of the study, which has not yet been completed.

Most healthcare systems have not adopted the 1-step strategy.

Tags

@kpwashington, @SaschaDublin, #healthsystem, #gestationaldiabetes, #perinatal, #electronichealthrecords, #pctGR

January 18, 2019: Pragmatic Trials in End-stage Renal Disease (ESRD): HiLo (Myles Wolf, MD, MMSc)

Speaker

Myles Wolf, MD, MMSc
Charles Johnson, MD, Professor of Medicine
Chief, Duke Nephrology
Duke University School of Medicine

Topic

Pragmatic Trials in End-stage Renal Disease (ESRD): HiLo

Keywords

Pragmatic clinical trial; HiLo; End-stage renal disease; ESRD; Kidney disease; Hypophosphatemia; Serum phosphate; Hemodialysis; A vs B trials; Clinical equipoise; National Institute of Diabetes and Digestive and Kidney Diseases; NIDDK

Key Points

  • With high event rates and few proven therapies, patients with end-stage renal disease (ESRD) are in desperate need of clinical innovation.
  • The NIH Collaboratory’s HiLo Demonstration Project is a pragmatic, multicenter, cluster-randomized, open-label, noninferiority outcomes trial that will compare effects of two different phosphate management strategies in patients with ESRD.
  • The study hypothesizes that, compared with strict phosphate control, less stringent control will yield noninferior rates of all-cause hospitalization among patients with ESRD undergoing hemodialysis, as well as reduce the risk of all-cause mortality, enhance markers of diet and nutrition, and improve quality of life.

Discussion Themes

Dialysis clinic dieticians will have a pivotal role in implementing HiLo. They have established a rapport with patients and are among the most motivated caregivers on dialysis teams.

Individual patient-level informed consent for the HiLo trial will be via internet-linked tablets, paper forms, and educational materials including a video. Benefits of obtaining consent include promoting adherence, direct study updates and newsletters to participants, and ability to collect additional data without involving onsite study staff.

HiLo will be the first definitive clinical trial-grade evidence for opinion-based guidelines for phosphate management. Thus, results of HiLo have the potential to rapidly influence ESRD clinical practice.

Read more about the HiLo Demonstration Project in the Living Textbook.

Tags

#ESRD, #pctGR, @Collaboratory1, @DCRINews, @DukeKidney

January 11, 2019: Nudge: Text Messaging at Scale in Diverse Health Systems to Support Adherence to Cardiac Medication (Sheana Bull, PhD, MPH, Michael Ho, MD, PhD)

Speakers

Sheana Bull, PhD, MPH
Professor, Colorado School of Public Health
Director, mHealth Impact Lab

Michael Ho, MD, PhD
Professor of Medicine, University of Colorado School of Medicine
Staff Cardiologist, Eastern Colorado Health Care System (Denver Veterans Affairs)

Topic

Nudge: Text Messaging at Scale in Diverse Health Systems to Support Adherence to Cardiac Medication

Keywords

Nudge; Cardiovascular disease; Mobile technology; Digital technology; Health promotion; Artificial intelligence; mHealth; Behavioral economics

Key Points

  • Medication non-adherence causes one-third of medicine-related hospitalizations. Mobile and digital technologies for health promotion and disease self-management offer an opportunity to adapt behavioral “nudges.”
  • The Nudge Demonstration Project will use population-level pharmacy data to deliver “nudges” via mobile phone text messaging and artificially intelligent chat bots. The nudges provide the patient with a reminder that their medicine refill is due.
  • The Nudge study team plans to engage patients, providers, and health system stakeholders in designing, refining, and implementing the pilot intervention within 3 diverse health systems.

Discussion Themes

There are overlapping notions between “nudge” theory and other socioeconomic theories. The study team relied on supporting literature as well as input from patient stakeholders to design the content of the nudge messages.

“Text message fatigue” is a potential concern that could affect study participation. Investigators relied on guidance from stakeholders to gauge the most appropriate frequency of messages.

Details about the use of a personal cell phone as the primary device messaging system, as well as privacy parameters, are explained in the opt-out letter presented to study participants.


For more information, visit the Nudge Demonstration Project on the Living Textbook.

Tags

#cardiovasculardisease, #pctGR, #mobile, #digitaltechnologies, #healthpromotion, #artificialintelligent, @Collaboratory1, @texhern, @lmhcurtis