Comments Submitted: Expedited Program for Serious Conditions – Accelerated Approval of Drugs and Biologics

Public Comment

Submitted on: February 4, 2025

Submitted to: Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration

The MRCT Center submitted public comments in response to FDA’s draft guidance on the accelerated approval program, suggesting areas needing further clarification such as the identification and validation of surrogate endpoints, the rigor and timeliness of confirmatory trials, expansion of public disclosures for regulatory actions, increased transparency regarding decision-making and withdrawal, and global considerations in the absence of international harmonization of these regulations. 

What’s Up with Long-Term Follow-Up: Ethical, Regulatory, & Operational Challenges

Webinar

Presented on: December 5, 2024

Presented at: Virtual

This webinar was co-hosted by the MRCT Center and NYU’s Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group as a part of their 5th Annual Lunchtime Learning Series. The panel on Long-Term Follow-Up (LTFU) was moderated by Dr. Carolyn Chapman and captured the ethical, regulatory, and operational challenges of LTFU studies through diverse viewpoints and perspectives.

What’s Up with Long-Term Follow-Up: Ethical, Regulatory, & Operational Challenges webinar recording


Public Comments submitted: “Ethical Guidelines for Research Using Pervasive Data”

Public Comments

Comments provided on: January 15, 2025

Comments provided to: Chief Counsel, National Telecommunications and Information Administration

Description: The NTIA’s proposed rule on the “Ethical Guidelines for Research Using Pervasive Data” aims to increase U.S. privacy protections with a heightened focus on responsible use. In response, the MRCT Center has submitted a public comment that recommends enhancing safeguards through expanded legislative protections, increased transparency, continuous ethical oversight, and periodic review of data practices to address data advancements and mitigate risks to individuals and communities. 

Biannual Global Meeting of The Joint Task Force For Clinical Trial Competency, December 2024

Proceedings and Presentations

Proceedings from: Joint Task Force for Clinical Trial Competency Biannual Global Meeting on December 10, 2024

Held at: Virtual Meeting

Key Topics:

  • Availability of updated Powerpoint presentation on JTF history and function for general use
  • Training modules being used in Philippines, Japan and the ARISE network based on JTF Framework
  • Use of JTF Framework for training workforce at clinical sites in Portugal by ROCHE and AICIB
  • Use of JTF Framework to train community health members in New York City enhances diversity of participants in clinical trials
  • Update on revisions to JTF Domain 6 – Data Management and Informatics by the Data Management Task Force
  • Update on newly formed Patients Participants Task Force
  • Discussion of team science competencies as expansion of JTF Domains 7 and 8

Conference Slides

Public Comments submitted: “Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice”

Public Comments

Comments provided on: December 13, 2024

Comments provided to: U.S. Food and Drug Administration

Description: The FDA draft guidance, “Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice,” (FDA-2024-D-2052) highlights the need for maintaining scientific rigor and data reliability when using real world data (RWD) amidst diverse healthcare settings. The MRCT Center comments addressed data quality when using clinical (and variable) data, ethical and practical challenges of randomization in clinical settings, vulnerabilities in data privacy and security, and participant safety while promoting innovative approaches and pilot programs to refine implementation strategies for this purpose. 

Public Comments submitted: “Proposed Rule: Provisions Pertaining to Preventing Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons”

Public Comments

Comments provided on: November 29, 2024

Comments provided to: U.S. Department of Justice

Description: The DOJ Notice of Proposed Rulemaking (NPRM), “Provisions Pertaining to Preventing Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons,” (DOJ-NSD-2024-0004) aims to safeguard sensitive U.S. personal- and government-related data from countries of concern or covered persons, emphasizing national security and data protection. MRCT Center comments highlighted the need to balance these security measures with global health priorities, recommending explicit public health and research exemptions, alignment with international standards, refined definitions, and transparent compliance to support access to health-related data and the advancement of science, medicine, and individual and public health. 

Podcast Episode 2: Trials Beyond Borders

Podcast

Episode 2 of the Trials Beyond Borders podcast is now available on all streaming platforms. In this episode, Racquel Bruton, Associate Director and Clinical Trials Diversity Portfolio Lead at Biogen, and Willyanne DeCormier Plosky, Program Director at the MRCT Center, discuss how sponsor companies are enhancing representation in clinical trials, particularly outside the US. Racquel highlights Biogen’s longstanding commitment to reaching underrepresented populations and explores how the MRCT Center’s roadmap can help organizations develop effective diversity action plans. Tune in to learn practical insights on operationalizing diversity strategies.

Exit Survey Inclusive of the LGBTQIA+ Perspective

Tools

Published on: November 18, 2024

The “Exit Survey Inclusive of the LGBTQIA+ Participant Perspectiveis one tool in the LGBTQIA+ Inclusion by Design in Clinical Research Toolkit, and the third of three tools in the section of the Toolkit directed more toward participants. It is important for participants in clinical research activities to give feedback about their experiences. This feedback helps research teams and organizations better understand the participant’s experience of the research activity and learn where they can improve on efforts to empower research participants and the participants’ supporting families, friends, and communities. One way to gather feedback is through a survey, which may be given to participants periodically (e.g., once a month), and/or (as an exit survey) at the end of research activities. In this tool, we aim to show examples of the topics that participants may be asked about in a survey or interview or, if not asked, that the participant may wish to share with the research team in a patient portal, email, or other format.

Related Resources

LGBTQIA+ Inclusion by Design in Clinical Research Toolkit
LGBTQIA+: Inclusive Imagery Case Study
LGBTQIA+: Inclusive Language Checklist
SOGI Data Collection Checklist
SOGI: Data Privacy Checklist
Site Feasibility Decision Tree from the LGBTQIA+ Perspective
Participant Questionnaire from the LGBTQIA+ Perspective
Exit Survey Inclusive of the LGBTQIA+ Perspective

Participant Questionnaire from the LGBTQIA+ Perspective

Tools

Published on: November 18, 2024

 The “Participant Questionnaire from the LGBTQIA+ Participant Perspective” is one tool in the LGBTQIA+ Inclusion by Design in Clinical Research Toolkit, and the second of three tools in the section of the Toolkit directed more toward participants. There are numerous questions that participants may want to ask as they move from thinking about possible trials and sites to starting the process of enrolling and participating in a trial. Some of these questions may be covered by informational materials given to participants during the informed consent process and study visits, and some may not be. This list is to help participants prepare so that they can get the answers that they need and feel comfortable before continuing with the trial. The Participant Questionnaire tool is divided into sections that include questions to ask the research team, questions you may want to ask family, friends, and others you trust, and questions to ask yourself. It includes questions that anyone might want to ask and adds questions that LGBTQIA+ people (and/or their accompanying friends and family) might also want to ask.

Related Resources

LGBTQIA+ Inclusion by Design in Clinical Research Toolkit
LGBTQIA+: Inclusive Imagery Case Study
LGBTQIA+: Inclusive Language Checklist
SOGI Data Collection Checklist
SOGI: Data Privacy Checklist
Site Feasibility Decision Tree from the LGBTQIA+ Perspective
Participant Questionnaire from the LGBTQIA+ Perspective
Exit Survey Inclusive of the LGBTQIA+ Perspective

Site Feasibility Decision Tree from the LGBTQIA+ Perspective

Tools

Published on: November 18, 2024

The “Site Feasibility Decision Tree from the LGBTQIA+ Participant Perspective” is one tool in the LGBTQIA+ Inclusion by Design in Clinical Research Toolkit, and the first of three tools in the section of the Toolkit directed more toward participants. This tool is meant to empower potential LGBTQIA+ participants to assess whether a site may be trustworthy and welcoming. This tool is structured in tiers, with checkpoints in between. The first tier is potential capacity, where we provide prompts, or “determination factors” based on what IS happening at a site (or in the area nearby) that LGBTQIA+ people can use in considering whether the site is potentially a good place to participate in a study. In the second tier we focus on “historical capacity,” or the things that the site HAS/HAD done to support the well-being of LGBTQIA+ people that may inspire confidence that the site is trustworthy.

Related Resources

LGBTQIA+ Inclusion by Design in Clinical Research Toolkit
LGBTQIA+: Inclusive Imagery Case Study
LGBTQIA+: Inclusive Language Checklist
SOGI Data Collection Checklist
SOGI: Data Privacy Checklist
Site Feasibility Decision Tree from the LGBTQIA+ Perspective
Participant Questionnaire from the LGBTQIA+ Perspective
Exit Survey Inclusive of the LGBTQIA+ Perspective