About the event:
This public workshop is hosted by the FDA/CDER Office of New Drugs in collaboration with the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI). The purpose is to solicit input from stakeholders on: 1) Increasing the enrollment of historically underrepresented population in pediatric clinical studies, and 2) Encouraging pediatric clinical study participation that reflects the prevalence of the other disease or condition among demographic subgroups, where appropriate and other topics.
FDA Webpage
Background
— Clinical trial populations should, in general, align with the population affected by the disease.
— Some populations are frequently underrepresented in clinical trials despite having a disproportionate disease burden for certain diseases relative to their proportional representation in the general population. Studies that do not include individuals from underrepresented populations can perpetuate and exacerbate gaps in access, gaps in knowledge, and disparities in health outcomes.
— Adequate representation of these populations in clinical trials helps ensure that the data generated in the development program reflect the diversity of the population expected to use the drug or biological product, if approved, and may potentially identify effects on safety or efficacy outcomes that may be associated with, or occur more frequently within these populations.
FDA-University of Maryland CERSI Public Workshop:
ADEPT 9: Enhancing Diversity in Therapeutics Development for Pediatric Patients
Friday, September 6, 2024 (9:00 AM-5:00 PM)
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Session 1: Opening and Overview and Current Status of Pediatric Trial Participation and Lessons Learned (Video Recordings)
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| 9:00 a.m. - 9:05 a.m. |
Welcome and Overview
Lily Mulugeta
Associate Director, Division of Pediatrics and Maternal Health (DPMH) US Food and Drug Administration (FDA)
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| 9:05 a.m. - 9:10 a.m. |
Introductory Remarks
Hilary Marston
Chief Medical Officer US FDA
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| 9:10 a.m. - 9:25 a.m. |
Opening Presentation
Mathilda Fienkeng
Director of Division of Medical Policy Development, Office of Medical Policy US FDA
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Presentation
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| 9:25 a.m. - 9:40 a.m. |
Keynote Talk
Michelle and Michael Burgess
International Children's Advisory Network (iCAN) Patient/Parent
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Presentation |
| 9:40 a.m. - 10:00 a.m. |
FDA Perspective
Christine Lee
Acting Associate Commissioner and Director Office of Minority Health and Health Equity (OMHHE), US FDA
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Presentation |
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| 10:00 a.m. - 10:20 a.m. |
Diversity in Pediatric Research: Academic Perspective
Sue Rahman
Chief Scientific Officer Health Data Synthesis Institute
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Presentation
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| 10:20 a.m. - 10:30 a.m. |
Break
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Break
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| 10:30 a.m. - 10:50 a.m. |
Landscape of Industry Sponsored Pediatric Trials
Pam Simpkins
Managing Partner, Mezzopointe, LLC
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Presentation |
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10:50 a.m. - 11:30 a.m.
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Panel Discussion Moderators:
Dionna Green
Director, Office of Pediatric Therapeutics, US FDA
Lois K Lee
Senior Associate in Pediatrics, Division of Emergency Medicine Boston Children’s Hospital
Panelists:
Sneha Dave
Executive Director, Generation Patient Florence Bourgeois Associate Professor, Pediatrics, Harvard Medical School
Ann McMahon
Regulatory Scientist, Office of Pediatric Therapeutics, US FDA Pam Simpkins Managing Partner, Mezzopointe, LLC
Sue Rahman
Chief Scientific Officer, Health Data Synthesis Institute
Christine Lee
Acting Associate Commissioner and Director, OMHHE, US FDA
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| 11:30 a.m. - 12:30 p.m. |
Lunch
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Lunch
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Session 2: Inclusion Strategies (Video Recordings)
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| 12:30 p.m. - 1:05 p.m. |
Inclusive Trial Designs and Methodological Considerations (Case Examples)
Recruitment and Retention/Decentralized Trials
Rachel Randell
Assistant Professor of Pediatrics Duke University and Duke Clinical Research Institute (DCRI)
Addressing Diversity in Clinical Trials and Diversity Plans
Ted Love
Chair of Board of Directors, Biotechnology Innovation Organization
Bella Oguno
Vice President, Development Operations, Nuvig Therapeutics
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Presentation
Presentation
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| 1:05 p.m. - 1:20 p.m. |
Diversity in Pediatric Type 2 Diabetes Trials
Lauren Wood Heickman
Clinical Reviewer Session 2: Inclusion Strategies 3 Division of Diabetes, Lipid Disorders, and Obesity (DDLO), US FDA
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Presentation
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| 1:20 p.m. - 2:00 p.m. |
Panelists:
Anvita Ambardekar
High School Student, Pediatric Perspective iCAN
Lauren Wood Heickman
Clinical Reviewer, DDLO US FDA
Martha Donoghue
Acting Associate
Director, Pediatric Oncology, Office of Oncologic Diseases, US FDA
LaShell Robinson
Head of Diversity, Equity & Inclusion, Clinical Research Department,Takeda
Ki Lee Milligan
Executive Director,Pediatric Center for Excellence, Global Drug Development, Novartis
Ted Love (Virtual)
Chair of Board of Directors, Biotechnology Innovation Organization
Stephen Balevic (Virtual)
Associate Professor of Medicine and Pediatrics, Duke University and DCRI
Rachel Randell
Assistant Professor of Pediatrics, Duke University and DCRI
Christina Edwards
Director of Clinical Trials, National Minority Quality Forum
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| 2:00 p.m. - 2:15 p.m. |
Break
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Break
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| 2:15 p.m. - 3:00 p.m. |
Panel Discussion: Community Engagement and Trust Building
Moderator:
Carla Epps Senior
Physician, DPMH US FDA
Panelists:
Billie Jo Kipp
Clinical Psychologist, Indigenous Innovators Collaborative 4 Nasrin Sari Patient/Community Representative
Sneha Dave
Executive Director, Generation Patient
LaToya Williams
Community Clinical Director, Inside Edge Consulting Group
Anvita Ambardekar
High School Student, Pediatric Perspective iCAN
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| 3:00 p.m. - 3:45 p.m. |
Best Practices That Help Children and Families to Stay in Clinical Trials
Tamorah Lewis Sellers
Chair, Pharmacology and Pharmacogenetics Division Head, Clinical Pharmacology & Toxicology Staff Neonatologist, The Hospital for SickKids
Christina Edwards
Director of Clinical Trials, National Minority Quality Forum
Puja Umaretiya
Assistant Professor, Division of Pediatric Hematology/Oncology UT Southwestern, Children’s Medical Center
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Presentation
Presentation
Presentation
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| 3:45 p.m. - 4:55 p.m. |
Panel Discussion
Moderators:
Carla Epps
Senior Physician, DPMH US FDA
Billie Jo Kipp
Clinical Psychologist, Indigenous Innovators
Collaborative Panelists:
Lynne Yao
Director, DPMH US FDA
Tamorah Lewis
The Hospital for SickKids
LaToya Williams
Community Clinical Director, Inside Edge Consulting Group
Florence Bourgeois
Associate Professor, Pediatrics, Harvard Medical School
Bella Oguno Vice President, Development Operations, Nuvig Therapeutics 5
Melissa Penn
Director of Patient Engagement R&D, Bayer Pharmaceuticals
Michelle/Michael Burgess
iCAN Patient/Family Representative
Nasrin Sari
Patient/Community Representative
Puja Umaretiya
Assistant Professor, UT Southwestern, Children’s Medical Center
Christina Edwards
Director of Clinical Trials, National Minority Quality Forum
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| 4:55 p.m. - 5:00 p.m. |
Closing Remarks
Lynne Yao
Director, DPMH US FDA
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Funding Statement
This workshop is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award to the Center of Excellence in Regulatory Science & Innovation, U01FD005946 totaling $25,000 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.