FDA

January 31, 2024: Improving the Public’s Understanding of the FDA, in This Week’s PCT Grand Rounds

Posted on by Damon Seils

In this Friday's PCT Grand Rounds, Susan Winckler of the Reagan-Udall Foundation for the FDA will present "Strategies for Improving Public Understanding of FDA and the Products It Regulates: Why Should We Care, and What Might We Do?"

The Grand Rounds session will be held on Friday, February 2, 2024, at 1:00 pm eastern.

Winckler is the chief executive officer of the Reagan-Udall Foundation for the FDA, a nonprofit organization created by Congress “to advance the mission of the FDA to modernize medical, veterinary, food, food ingredient, and cosmetic product development, accelerate innovation, and enhance product safety.”

Join the online meeting.

June 15, 2023: Regulatory Considerations for Clinical Decision Support Software in Pragmatic Trials Explored at Annual Steering Committee Meeting

Posted on by Gina Uhlenbrauck

This year’s annual Steering Committee meeting for the NIH Pragmatic Trials Collaboratory featured 2 special guests from the US Food and Drug Administration’s Digital Health Center of Excellence—Matthew Diamond, MD, PhD, Chief Medical Officer, and Sonja Fulmer, PhD, Acting Deputy Director. They joined to share information on the FDA’s approach to clinical decision support (CDS) software, including the agency’s recently released guidance, which helped stimulate a discussion on the associated considerations for embedded pragmatic clinical trials. Here we highlight some key takeaways from that discussion.

CDS is a broad term that encompasses a range of different functions, and there are several NIH Collaboratory Trials that are testing CDS software. Diamond explained that some digital health technologies, including some CDS software, meet the regulatory definition of a medical device while others do not. The FDA focuses its oversight on a subset of digital health technologies.

The new FDA guidance outlines criteria for determining which CDS are considered non-devices due to the CDS criteria in the 21st Century Cures Act. In addition to the CDS guidance, there are other policies that may apply to a digital health technology, so the FDA created a Digital Health Policy Navigator to help people find the relevant guidance for their situation. Further, the FDA encourages engagement early and often to understand how the regulations and policies apply to a particular product or technology.

If a software is the focus of FDA’s regulatory oversight, it does not mean the research cannot be done. In fact, research is essential to contribute to the body of evidence on the functioning of a device. The research just needs to go through the right processes. The goals of using the right processes for product review and research are to ensure that patients are protected during research and that the products that are ultimately marketed are safe and effective. The determination of whether an investigational device exemption (or IDE) is required is based on risk.

The details behind the implementation of digital health products matters a lot in these determinations. Subtle differences in the way that a product functions and the claims that are made about a product can make a big difference. Diamond stressed the importance of clearly defining the intended use of a product, including who it is intended for and what the user is supposed to do with information provided by the software. Fulmer highlighted the importance of transparency with stakeholders, including healthcare professionals, patients, and regulators.

For specific questions, the presenters encouraged researchers to reach out to the FDA’s digital health experts at DigitalHealth@fda.hhs.gov.

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.

August 30, 2022: FDA Announces Webinar on Patient-Focused Drug Development Draft Guidance

Posted on by Damon Seils

FDA logoThe US Food and Drug Administration (FDA) will host a webinar on September 9 for industry, patient groups, and other interested stakeholders to discuss and answer questions about the draft guidance, Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments.

The draft guidance, known as “Guidance 3,” is the third of 4 methodological guidance documents for patient-focused drug development that describe how patients, caregivers, researchers, medical product developers, and others can collect and submit patient experience data and other relevant information to be used for medical product development and regulatory decision making. Guidance 3 discusses approaches to selecting, modifying, developing, and validating clinical outcome assessments to measure outcomes of importance to patients in clinical trials.

Register for the webinar at https://www.eventbrite.com/e/public-webinar-patient-focused-drug-development-pfdd-draft-guidance-3-tickets-397246183027.

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

June 22, 2022: FDA’s Draft Guidance on Real-World Evidence to Be Featured in Grand Rounds

Posted on by Damon Seils

Headshot of Dr. John ConcatoIn this Friday’s PCT Grand Rounds, Dr. John Concato of the US Food and Drug Administration will present “FDA Draft Guidance on Real-World Evidence.” The Grand Rounds session will be held on Friday, June 24, 2022, at 1:00 pm eastern.

Dr. Concato is the associate director for real-world evidence analytics in the Office of Medical Policy in the FDA’s Center for Drug Evaluation and Research.

Join the online meeting.

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

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)

Topic: FDA Draft Guidance on Real-World Evidence
Date: Friday, June 24, 2022, 1:00-2:00 pm ET

Meeting Info: To check whether you have the appropriate players installed for UCF (Universal Communications Format) rich media files, go to https://dukemed.webex.com/dukemed/systemdiagnosis.php.

To join the online meeting:
Go to https://dukemed.webex.com/dukemed/j.php?MTID=m4c6e3e4827442a92338a5649cee613e6

Click ‘Connect to Audio”
Choose ‘Computer Audio’ or ‘I will call in’.
If using the ‘call in option’, follow the information from the dialog box that appears.
Be certain to use the Access Code AND the Attendee ID.

Troubleshooting:
If the URL above does not work, go to dukemed.webex.com and enter:
Meeting Number: 2622 682 0537
Meeting Password: 12345

For Audio ONLY:
Call-in toll number (US/Canada): 1-650-479-3207
Access code: 2622 682 0537

NOTE: For Toll-free users, the call-back (call me) services are also available.

April 14, 2022: FDA Announces New Draft Guidance for Increasing Enrollment of Diverse Populations in Clinical Trials

Posted on by terren.green@duke.edu

FDA logoThe US Food and Drug Administration (FDA) issued draft guidance yesterday recommending clinical trial sponsors develop a “Race and Ethnicity Diversity Plan” to ensure representative enrollment of racially and ethnically diverse participants in clinical trials developing medical products.

The draft guidance, Diversity Plans to Improve Enrollment of Participants From Underrepresented Racial and Ethnic Populations in Clinical Trials Guidance for Industry, updates previous FDA guidance issued in October 2016. The “Race and Ethnicity Diversity Plan” is recommended for studies submitting IDE or IND applications to the FDA for approval of an investigational drug or device. The updated guidance provides information about 5 elements that should be included in the plan:

  • Overview of the disease/condition
  • Scope of medical product development program
  • Goals for enrollment of underrepresented racial and ethnic participants
  • Specific plan of action to enroll and retain diverse participants
  • Status of meeting enrollment goals

Achieving heath equity for underrepresented racial and ethnic populations starts with appropriate representation in clinical trials. Disease burden is often higher for underrepresented populations, yet barriers to participation in clinical trials may prevent adequate enrollment. Improving racial and ethnic diversity in clinical trials ensures that results are generalizable and medical discoveries are safe and effective for all patients.

The draft guidance was a collaborative effort between the Oncology Center of Excellence’s Project Equity, the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health.

Read the FDA news release.