21st Century Cures Act

February 7, 2023: FDA Issues Draft Guidance for Use of Real-World Data in Externally Controlled Trials

Posted on by Damon Seils

The US Food and Drug Administration last week issued draft guidance for the use of “externally controlled clinical trials” to provide evidence of the safety and effectiveness of drugs and biologics. An externally controlled trial uses patient-level data from a source outside the clinical trial—such as a registry, electronic health records, or administrative claims data—to provide a historical or concurrent control group for the study.

Reviewers are asked to submit written comments on the draft guidance by May 2, 2023. Read the draft guidance.

Externally controlled trials can be useful when it would not be feasible or ethical to use an internal control in the study, such as in studies of populations with rare diseases. The FDA issued the draft guidance as part of a series of guidance documents under its Real-World Evidence Program to satisfy a mandate under the 21st Century Cures Act.

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 John Concato on FDA Draft Guidance on Real-World Evidence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 Richard Platt continue their discussion about the FDA draft guidance on real-world evidence.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.

August 4, 2020: New Living Textbook Sections Describe Interoperability, Patient Access to Data

Posted on by Karen Staman

Members of the NIH Collaboratory Electronic Health Records Core have authored 2 new sections of the Living Textbook chapter Using Electronic Health Record Data in Pragmatic Clinical Trials:

  • Interoperability
    • This section describes efforts to support interoperability and the sharing of patient data across care teams and organizations, including the creation the United States Core Data for Interoperability (USCDI) standard.
  • Patient Access to Data
    • This section describes provisions of the 21st Century Cures Act intended to support the access, exchange, and use of electronic health information by patients and their caregivers.

Many of the NIH Collaboratory Trials use electronic health record and claims data for pragmatic research, and these data are from a fundamentally different context than data prospectively collected for more traditional, explanatory research. The Living Textbook chapter, Using Electronic Health Record Data in Pragmatic Clinical Trials, describes how data from real-world sources can be used in pragmatic clinical trials to develop and refine research questions, identify the study population and assess baseline prognostic characteristics, implement and monitor the delivery of the intervention, and assess outcomes.

The 2 new sections describe the latest developments and considerations for use of electronic health data in pragmatic clinical trials.

February 22, 2019: Proposed Rule to Implement Provisions of the 21st Century Cures Act

Posted on by Karen Staman

The Office of the National Coordinator for Health Information Technology (ONC) and the Centers for Medicare and Medicaid (CMS) have announced a proposed rule intended to advance interoperability and support the access, exchange, and use of electronic health information. Notably, the rule would require that patients have the ability to electronically access their health information at no cost.

The rule also proposes a United States Core Data for Interoperability (USCDI) standard, which, if adopted, would add data beyond those included in the current common clinical data model to support nationwide interoperability of CMS data. Specifically, clinical notes, data provenance, pediatric vital signs, patient address and phone number (to support data matching) will be added if the measures are adopted.

“Today’s announcement builds on CMS’ efforts to create a more interoperable healthcare system, which improves patient access, seamless data exchange, and enhanced care coordination,” — CMS Administrator Seema Verma, from the NPRM Press Release

There are nine fact sheets on other important aspects of the rule, including sheets on interoperability, the Cures Act, and electronic health information export for patient and provider access.

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?