Real-world data

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

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?