Real-world evidence

February 26, 2021: Calibrating Real-World Evidence Against RCT Evidence: Early Learnings from RCT-DUPLICATE (Sebastian Schneeweiss, MD, ScD)

Posted on by Gina Uhlenbrauck

Speaker

Sebastian Schneeweiss, MD, ScD
Chief, Division of Pharmacoepidemiology and Pharmacoeconomics
Department of Medicine, Brigham and Women’s Hospital
Professor of Medicine, Harvard Medical School
Professor in Epidemiology, Harvard T.H. Chan School of Public Health

Topic

Calibrating Real-World Evidence Against RCT Evidence: Early Learnings from RCT-DUPLICATE

Keywords

Real-world evidence (RWE); Randomized controlled trials (RCTs); Epidemiology; Emulation; Fit-for-purpose data

Key Points

  • While RCTs are an accepted research study design to establish the efficacy of medical products, RWE studies can complement the evidence generated by RCTs, as well as expand the line of inquiry around population, endpoints, treatment patterns, and comparators.
  • The RCT-DUPLICATE study aimed to understand and improve the validity of RWE studies for regulatory decision making. One objective was to identify factors that predictably increase the validity of such studies.
  • In RCT-DUPLICATE, RWE studies were designed to emulate 20 target RCTs. The regulatory-standard RCTs for replication underwent feasibility checks and quality assessments.
  • With data that are fit-for-purpose and proper design and analysis, nonrandomized RWE studies usually come to the same conclusion as the RCT about a drug’s treatment effect.
  • In any emulation, despite best efforts, there will remain differences in population, measurement, and drug use. Data fit-for-purpose and study design choices are the most important considerations for emulation success.

Discussion Themes

Initial findings of RCT-DUPLICATE identify circumstances when RWE may offer causal insights in situations where RCT data are either not available or cannot be quickly or feasibly generated.

Can this approach be used to predict results for a new entity?

It will be useful to establish a repository of case studies to increase the predictability of future RWE studies; increase the use of common methodological approaches to emulate target trials; and point out areas that are currently difficult to address with RWE.

Read more about RCT-DUPLICATE in Circulation.

Tags

#pctGR, @Collaboratory1

January 5, 2021: New Video Collection Highlights Advances in Electronic Health Records for Pragmatic Research

Posted on by Damon Seils

We are pleased to announce our new collection of short educational videos that highlight advances in the use of electronic health records (EHRs) in pragmatic clinical trials.

The videos are drawn from our 2020 EHR Workshop Grand Rounds Series, “Advances at the Intersection of Digital Health, Electronic Health Records and Pragmatic Clinical Trials.” The series highlighted advances in digital health, new approaches and evolving standards for EHRs, and implications for researchers conducting pragmatic trials. Experts discuss the evolving regulatory context for EHRs, national policy priorities, and innovative uses of EHRs in several NIH Collaboratory Trials.

Video topics include:

Visit the EHR Workshop Video Modules page for access to all of the new videos.

September 2, 2020: Chapter on Assessing Fitness for Use of Real-World Data Sources Added to the Living Textbook

Posted on by Karen Staman

The NIH Collaboratory published a new chapter of its Living Textbook of Pragmatic Clinical Trials. The chapter, “Assessing Fitness-for-Use of Real World Data Sources,” describes several approaches for determining whether real-world data are fit for their intended purpose in pragmatic clinical trials.

“Real-world data” are collected for clinical care, insurance claims, administrative purposes, registries, or are generated directly by the patient. Because these data are collected for a purpose other than a specific research project, an investigator must understand the characteristics and limitations of the data to determine whether they can be used in a pragmatic trial.

The new chapter includes the following sections:

The new chapter updates a previous resource based on work by experts in the NIH Collaboratory’s Electronic Health Records Core Working Group.

August 27, 2020: Chapter on Acquiring Real-World Data Added to the Living Textbook

Posted on by Karen Staman

The NIH Collaboratory this week published a new chapter of its Living Textbook of Pragmatic Clinical Trials. The chapter, “Acquiring Real-World Data,” outlines strategies for obtaining real-world data for use in research.

“Real-world data” include data relating to the health status of a patient or the delivery of healthcare services. Common sources include electronic health records (EHRs), administrative claims, patient-reported outcomes, patient-generated health data, medical product and device registries, and databases relating to environmental factors or social determinants of health. Real-world data can support a number of activities in pragmatic clinical trials, such as patient identification and recruitment, monitoring of outcomes, and ascertainment of endpoints.

The new chapter includes the following sections:

The new chapter updates a previous resource, one of the most popular on the Living Textbook, based on work by experts in the NIH Collaboratory’s Electronic Health Records Core Working Group.

July 31, 2020: Using Real-World Data to Plan Eligibility Criteria and Enhance Recruitment: Actionable Recommendations and Resources from the Clinical Trials Transformation Initiative (Sudha Raman, PhD, MA; John Sheehan, PhD, MBA, RPh)

Posted on by Gina Uhlenbrauck

Speakers

Sudha Raman, PhD, MA
Assistant Professor
Department of Population Health Sciences
Duke University

John Sheehan, PhD, MBA, RPh
Senior Director, Value and Evidence (HEOR) Neuroscience
Janssen Scientific Affairs, LLC

Topic

Using Real-World Data to Plan Eligibility Criteria and Enhance Recruitment: Actionable Recommendations and Resources from the Clinical Trials Transformation Initiative

Keywords

Clinical Trials Transformation Initiative (CTTI); Real-world data (RWD); Recruitment planning; EHR; Eligibility criteria; Fit-for-purpose data

Key Points

  • Real-world data (RWD) are data relating to patient health status and/or the delivery of health care routinely collected by a variety of sources.
  • CTTI provides recent recommendations, resources, and case studies that highlight actionable tools and best practices for evaluating and using real-world data (RWD) in clinical trial recruitment activities:
    • General principles for using RWD
    • Using RWD to plan eligibility criteria
    • Using RWD to support recruitment
    • Enhancing RWD capabilities for the research enterprise
  • Using RWD from data sources such as electronic health records and claims data brings challenges for completeness, accuracy, and generalizability of the data.
  • RWD holds the potential to increase patient eligibility and enrollment as well as reduce recruitment timelines.

Discussion Themes

Insights from RWD should be sought early in the product lifecycle and include context from patients and sites.

One challenge of RWD data sources is finding appropriate databases for the disease area of interest, especially for trials of rare diseases.

Are there lessons learned about when using RWD becomes prohibitive or too expensive?

Read and download CTTI’s recommendations for using RWD. Learn more about FDA’s guidance for real-world data and real-world evidence. A publication is available about the health plan recruitment method used in the ADAPTABLE aspirin study.

Tags

#pctGR, @Collaboratory1

April 20, 2020: Grand Rounds EHR Workshop Series Will Highlight Advances in Digital Health, Electronic Health Records, and Pragmatic Clinical Trials

Posted on by Damon Seils

NIH Collaboratory Grand Rounds will launch a special workshop series next month to explore challenges and opportunities in using electronic health records (EHRs) in pragmatic clinical trials. The series will highlight advances in digital health, new approaches and evolving standards for EHRs, and implications for researchers conducting pragmatic trials.

The webinar series, Advances at the Intersection of Digital Health, Electronic Health Records and Pragmatic Clinical Trials, will kick off on May 1 with a keynote presentation by Dr. Robert Califf, head of strategy and policy for Verily Life Sciences and Google Health.

The new Grand Rounds series will include moderated panel discussions that focus on the use of digital technologies in pragmatic clinical trials, case examples from the NIH Collaboratory Demonstration Projects, and recent initiatives from the Office of the National Coordinator for Health Information Technology to reshape standards and drive the evolution of EHRs in healthcare and research. A podcast discussion with a panel of experts will wrap up the series in July.

Download the EHR workshop series flyer and see the full schedule below:

April 13, 2020: New Living Textbook Chapter on Pragmatic Mobile Health (mHealth) Trials

Posted on by Damon Seils

A new chapter in the Living Textbook provides expert advice for investigators considering using mobile technologies in pragmatic clinical trials. The chapter outlines many of the possibilities, advantages, and challenges associated with mobile health (mHealth) interventions, with a particular focus on design and evaluation of these programs in pragmatic clinical trials.

“We will illustrate many design and evaluation challenges, culminating with a discussion of how these considerations influence the ongoing development of the Personalized Patient Data and Behavioral Nudges to Improve Adherence to Chronic Cardiovascular Medications (Nudge) project.”—Real-World Evidence: Mobile Health (mHealth)

For more on using real-world evidence in pragmatic trials, see the Living Textbook chapter Choosing and Specifying Endpoints and Outcomes.

October 25, 2019: Real-World Evidence for Drug Effectiveness Evaluation: Addressing the Credibility Gap (Richard Willke, PhD)

Posted on by Gina Uhlenbrauck

Speaker

Richard Willke, PhD
Chief Science Officer
ISPOR

Topic

Real-World Evidence for Drug Effectiveness Evaluation: Addressing the Credibility Gap

Keywords

Real-world evidence; Non-interventional studies; Health economics; ISPOR; Transparency; Reproducibility

Key Points

  • ISPOR is an international, multistakeholder nonprofit dedicated to advancing health economics and outcomes research excellence to improve decision making for health globally.
  • Key characteristics of credible and useful real-world evidence include:
    • Careful data collection or curation
    • Appropriate analytic methods
    • Good procedural practices for transparent study process
    • Replicability and reproducibility
    • Informed interpretation
    • Fit-for-purpose application
  • For transparency, it is recommended that researchers declare their study to be an exploratory (hypothesis evaluation) study and post the study protocol and analysis plan on a public study registration site prior to conducting the study analysis.

Discussion Themes

A draft white paper, Improving Transparency in Non-Interventional Research, is available for comment until November 15, 2019.

Sharing all study implementation parameters and definitions provides clarity on what was actually done and enables reproduction with confidence.

Potential registries for non-interventional real-world evidence studies include:

Read more about ISPOR.

Tags
#pctGR, @Collaboratory1, @ISPORorg

January 11, 2019: FDA Releases Framework for Evaluating the Use of Real-World Evidence

Posted on by Karen Staman

To help fulfill the requirements of the 21st Century Cures Act by accelerating medical product development and fostering innovation and advances in medicine, FDA recently created a framework for evaluating the use of real-world evidence. The framework is intended to help evaluate trials that use real-world data for the creation of real-world evidence.

  • Real-world data: routinely collected information about a person’s health status in the electronic health record, claims, registries, and other sources, including patient-generated sources.
  • Real-world evidence: reliable, clinical information derived from real-world data about risks, benefits, and burdens of therapies.

This framework will apply to various pragmatic clinical trials embedded in health care systems and conducted as part of routine care (and will not apply to more traditional clinical trials conducted parallel to care).

Three main considerations are included in the framework:

  1. Will the real-world data be fit for use (do they reliably and adequately represent the concept)?
  2. Will the evidence generated by the trial provide adequate evidence to help answer regulatory questions?
  3. Will the conduct of the study meet FDA regulatory requirements?

October 12, 2018: MDEpiNet RAPID and SPEED Projects: Leveraging Real World Evidence to Get Better, Faster, Cheaper Medical Devices for Physicians and Patients (Renee Mitchell, MT, CLS, Terrie Reed, MSIE, Roseann White, MA)

Posted on by Gina Uhlenbrauck

Speakers

Renee Mitchell, MT(ASCP), CLS(NCA)
Regulatory Affairs
Boston Scientific Corporation, Inc.

Terrie Reed, MSIE
Senior Advisor for UDI Adoption
U.S. Food and Drug Administration (FDA)

Roseann White, MA
Director of Innovative Clinical Trial Statistics
Duke Clinical Research Institute

Topic

MDEpiNet RAPID and SPEED Projects: Leveraging Real World Evidence to Get Better, Faster, Cheaper Medical Devices for Physicians and Patients

Keywords

Medical devices; Real-world evidence; Medical Device Epidemiology Network; MDEpiNet; Unique device identifier; UDI

Key Points

  • In partnership, clinicians, device developers, and FDA can benefit from the use of real-world evidence on medical devices:
    •  Clinicians can contribute to the generation of real-world evidence.
    •  Device manufacturers can use real-world evidence to evaluate and release new devices and expand indications.
    •  Regulatory bodies can increase the use of patient-level data for device approval.
  • Unique device identifiers (UDIs) make it possible to follow medical devices longitudinally, advancing the quality of real-world evidence and allowing more sophisticated analyses.

Discussion Themes

The vision for the future is that registries will transform into big data solutions using multiple sources and will be more robustly integrated with electronic health records (EHRs). Both EHRs and registries will play a role.

More organizations as partners brings greater diversity, advancing better data and results. When stakeholders work together, learning curves can be accelerated toward a transformational approach to real-world evidence.

Tags

#MedicalDevices, #pctGR, @PCTGrandRounds, @Collaboratory1, @MDEpiNet_ppp