Real-world data

April 23, 2026: NIH to Host Workshop on Combining RCT and Real-World Data for Studying Infant Postnatal HIV Prophylaxis

Posted on by Damon Seils

The National Institute of Allergy and Infectious Diseases (NIAID) Division of AIDS (DAIDS), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and the National Institute of Mental Health (NIMH) Division of AIDS Research (DAR) will host a workshop on methods for combining data from randomized controlled trials with real-world data for studying prevention of HIV vertical transmission.

NIH logoThe virtual workshop is scheduled for May 11-12, 2026.

The workshop will convene methodologists, clinical trialists, epidemiologists, data scientists, and regulators to evaluate innovative methods for combining these types of data, with the eventual goal of designing an efficacy-effectiveness trial of long-acting agents as infant postnatal prophylaxis against HIV. The organizers also have an interest in extending this methodology into pragmatic designs.

WORKSHOP INFORMATION AND REGISTRATION

Combining Randomized Controlled Trial and Real-World Data for Long-Acting Agents as Infant Postnatal Prophylaxis Against HIV

  • Dates and Times: May 11–12, 2026, 10:00 am – 2:30 pm EDT
  • Location and Format: Virtual

Objective: To evaluate novel study designs, analytical approaches, and data methods for combining RCT and RWD for an efficacy-effectiveness trial of long-acting agents as infant HIV postnatal prophylaxis against HIV.

Agenda Overview

May 11, 2026, 10:00 am – 2:30 pm EDT (Virtual)

  • Session I. Welcome and Opening Session
  • Session II. Study Design Considerations for Combining RCT and RWD

May 12, 2026, 10:00 am – 2:30 pm EDT (Virtual)

  • Session III. Novel Methods and Analytical Approaches for Combining RCT and RWD
  • Session IV. Data Standards, Management, and Harmonization

Register at https://cvent.me/Yl2RYq. All registered participants will receive the link to the virtual workshop in a reminder email approximately 1 week prior to the event.

October 11, 2022: AHRQ Announces Healthcare Systems Data Challenge

Posted on by Damon Seils

AHRQ logoThe Agency for Healthcare Research and Quality (AHRQ) announced a new competition to explore the feasibility, resources, and infrastructure needed to integrate real-world healthcare system data into the agency’s systematic review findings to improve healthcare practice. The top award winner could earn up to $200,000.

Learn more about AHRQ’s Challenge on Integrating Healthcare System Data With Systematic Review Findings.

The two-phase challenge aims to incorporate unpublished healthcare system data into AHRQ’s systematic review findings to inform local healthcare decisions better. This additional information may be particularly valuable when results from systematic reviews are limited by the number of research studies or only include studies with narrow patient populations.

For details about AHRQ’s challenge, view the Federal Register Notice.

August 31, 2022: New Section of Living Textbook Addresses Evaluating Fitness for Use of Real-World Data Sources

Posted on by Karen Staman

A new section of the NIH Pragmatic Trials Collaboratory’s Living Textbook of Pragmatic Clinical Trials discusses challenges associated with Evaluating Fitness for Use of real-world data for trials. The section uses a case study from the Harmony Outcomes EHR Ancillary Study (eHARMONY) to describe lessons learned and to provide recommendations for studies considering incorporating real-word data as a data source.

Among the lessons learned were:

  • Standalone clinical research sites had very little extractable EHR data about patients.
  • Most lab results and medications were either not extractable or not mapped to a useful terminology.
  • Many sites did not have the ability to transform their data into a common format and had to send rudimentary data extracts to the ancillary study coordinating center. Sites participating in other research networks, such as PCORnet, had no difficulty with this task.

For more, read the chapter or watch the Grand Rounds presentation Leveraging RWD in a Multinational Trial: Results from the other eHARMONY 

July 14, 2022: Grand Rounds Podcast Now Available, Featuring Dr. John Concato on FDA Draft Guidance on Real-World Evidence

Posted on by terren.green@duke.edu

Headshot of Dr. John ConcatoIn the latest episode of the NIH Collaboratory Grand Rounds podcast, Dr. John Concato and Dr. Richard Platt continue their discussion about the FDA draft guidance on real-world evidence.

The full June 24 Grand Rounds webinar with Dr. Concato is also available.

Podcast July 8, 2022: FDA Draft Guidance on Real-World Evidence (John Concato, MD, MS, MPH)

Posted on by terren.green@duke.edu

This podcast continues the discussion with Dr. John Concato as he discusses the FDA draft guidance on real-word evidence. Click on the recording below to listen to the podcast.

Want to hear more? View the full Grand Rounds presentation.

For alerts about new episodes, subscribe free on Apple Podcasts or SoundCloud. Read the transcript.

June 24, 2022: FDA Draft Guidance on Real-World Evidence (John Concato, MD, MS, MPH)

Posted on by terren.green@duke.edu

Speaker

John Concato, MD, MS, MPH
Associate Director for Real-World Evidence Analytics
Office of Medical Policy (OMP)
Center for Drug Evaluation and Research (CDER)
Food and Drug Administration (FDA)

 

 

Keywords

Big data; Real-word evidence; Real-world data; 21st Century Cures Act; FDA Draft Guidance

 

Key Points

  • Big Data, a term first used in the 1990s, leverages modern technology to increase the quantity, forms, speed, and capability to manipulate large-scale data. Real-world data (RWD) is a term with specific regulatory implications referring to health care data routinely collected from a variety of sources. Real-world evidence (RWE) is clinical evidence derived from analysis of RWD regardless of study design.
  • Terminology is important in research work, and we should strive to be as precise as possible with the terminology we use.
  • With the 21st Century Cures Act of 2016, the FDA established a program to evaluate the potential use of real-world evidence to support new indications for drugs and satisfy post-approval study requirements.
  • In 2021, the FDA issued 4 draft guidance documents for Real-world data and Real-world evidence intended to guide the selection and management of data sources to appropriately address the study question and support decision-making for drug and biological products.

Discussion Themes

– Could real-world data sources be certified and preclude the need for submission of source data on a study specific basis? From the FDA point-of-view, while reliability can be more readily evaluated and would tend to be more stable, the relevance to a particular study could not be determined as easily.

– While there can be a reflex that says we can never be sure about major confounding, it should not be the miasma of the 21st century. A thoughtful approach that considers the characteristics that matter is the best approach.

 

Read Dr. Concato’s publication Randomized, observational, interventional, and real-world—What’s in a name? and the FDA Draft Guidance for RWD/RWE.

Tags

#pctGR, @Collaboratory1

May 9, 2022: FDA Leaders Offer Perspective on Real-World Data and Real-World Evidence

Posted on by Damon Seils

FDA leaders Dr. John Concato and Dr. Jacqueline Corrigan-Curay published a perspective piece in the New England Journal of Medicine encouraging researchers to be more explicit and specific about the types of real-world data (RWD) they are using to generate real-world evidence (RWE).

The article, “Real-World Evidence — Where Are We Now?,” cautions that the terms RWD and RWE are too often used interchangeably and inconsistently in describing research and that this imprecision has led to confusion and an inability to assess the impact of RWD and RWE. The authors described 2 misconceptions about RWD and RWE. The first is that RWD is “new” when in fact real world sources of information have always existed; what is new is how researchers are able to access the information. The second misconception is that “a simple dichotomy between randomized, controlled trials (RCTs) and observational studies delineates the entire landscape of study design.” In fact, the authors write, the important distinction in study design is often interventional vs non-interventional.

The authors recommend precision in describing how RWD is incorporated into a variety of study designs to generate regulatory-grade RWE and cited recent examples of FDA approvals that were made possible by RWD. They also describe how the COVID-19 pandemic is changing perceptions about the potential of RWD and RWD.

Read the full article.

July 19, 2021: COVID-19 Grand Rounds Will Feature Survivor Corps and the Use of Real-World Evidence From Citizen-Scientists

Posted on by Damon Seils

Survivor Corp logo

In this Friday’s COVID-19 Grand Rounds session, Diana Berrent, Natalie Lambert, and Nick Guthe of Survivor Corps will present “Long Term COVID Patient Engagement: Best Practices Informed By Patients’ Experiences Seeking Medical Care.” The Grand Rounds session will be held on Friday, July 23, at 1:00 pm eastern. Join the online meeting.

Survivor Corps is an online community of 170,000 patients affected by COVID-19 and their families and friends. The advocacy group is using its members’ collective experience to build a repository of data sets and research tools to support COVID-19 research, including studies of post-COVID conditions, or “long COVID.” The group seeks to fill the gap between real-world evidence and scientific research to advance understanding of the disease and patients’ healthcare options.

“We have been sort of the canary in the COVID coal mine from the beginning,” said Berrent, who founded Survivor Corps in March 2020 after becoming one of the first people in the United States to be diagnosed with COVID-19.

Berrent will be joined during Grand Rounds by Survivor Corps research director Dr. Natalie Lambert and by Nick Guthe, a Survivor Corps member and adviser.

Survivor Corps’s website, which has been selected by the US Library of Congress for inclusion in the nation’s Coronavirus Web Archive, serves as a hub to provide support, information, and education about COVID-19, connect patients to researchers, and facilitate the nation’s COVID-19 response.

The NIH Collaboratory Coordinating Center is using its popular Grand Rounds platform to share late-breaking research and promote resources in support of clinical researchers affected by the COVID-19 public health emergency. For previous COVID-19 Grand Rounds, and more news and resources related to the COVID-19 public health emergency, see the COVID-19 Resources page.

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.