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Center of Excellence in Regulatory Science and Innovation

Clinical Trials: Assessing Safety and Efficacy for a Diverse Population

8:30 a.m. – 4:30 p.m.
December 2, 2015
FDA White Oak Campus, Bldg. 31, The Great Room

Hosted by Johns Hopkins Center of Excellence in Regulatory Science and Innovation (JH-CERSI) and the Food and Drug Administration (Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, Center for Devices and Radiological Health, Office of Chief Scientist’s Office of Minority Health, Office of Regulatory Science and Innovation, and Office of Scientific and Professional Development)

Agenda & Speakers
Attendee Information & Directions



Problem statement: Some medical interventions do not have uniform effects across diverse patient populations represented in the United States, and it can be challenging to identify differences in effects across groups.   This uncertainty leads to regulatory challenges for approval and monitoring of interventions.  The aim of this workshop is to address the scope of potential ethnic/racial subgroup issues based on current evidence and to discuss methodologic approaches to addressing these issues.  The workshop will focus on evidence from clinical trials and other data sources to address potential heterogeneity across large and diverse populations.

The Food and Drug Administration (FDA) is responsible for protecting the public health by ensuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products, and medical devices.  Under the 2012 FDA Safety and Innovation Act, Section 907, FDA was mandated to develop a report (issued August 2013) addressing the extent to which demographic subgroups were included in applications for medical products submitted to FDA, including analyses for safety and efficacy.  The Act also required FDA to develop an Action Plan (issued August 2014) based on the findings of the report as well as stakeholder consultation. The Action Plan notes that “advances in science are playing an increasingly important role in deepening our understanding of how patients within various subgroups respond to medical products….ultimately, this is steering us towards the goal of tailoring treatments to individuals or subgroups of patients through personalized medicine—including patients in underserved and underrepresented populations."

The primary educational mission of the JHU CERSI is to expand the knowledge base and to engage in development of state-of-the-art tools and methods to support regulatory science needs, including advancing understanding of diverse populations.

Objective: This educational workshop aims to describe the significance of population diversity in evaluating medical products and to advance discussion on methodological challenges in subgroup analyses in the evaluation of medical products for diverse populations.

Target audience: This workshop is an educational event and is open to the public.  It is intended for government, academic, and industry scientists with an interest in clinical protocol development, diverse patient participation, and statistical considerations in trial design for statistical analyses.  It may be of particular interest to drug and device developers, clinical trialists, biostatisticians, statistical reviewers, medical reviewers, and students.

Agenda & Speakers

Welcoming remarks                                                                                                   

Jona Bull, MD
Assistant Commissioner for Minority Health, FDA

Luciana Borio, MD
Acting Chief Scientist, FDA

Caleb Alexander, MD, MS
Co-Director, Center for Drug Safety and Effectiveness
Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health

Janet Holbrook, PhD
Associate Professor, Department of Epidemiology
Johns Hopkins Bloomberg School of Public Health

Introduction: Clinical Trials -                                                   
Assessing Safety and Efficacy for a Diverse Population
Keith Ferdinand, MD, FACC, FAHA, FNLA, FASH
Professor of Clinical Medicine,
Tulane University School of Medicine

Keynote: The Importance of Diversity in Medical Research          
Robert M. Califf, MD, MACC
Deputy Commissioner, FDA Office of Medical Products and Tobacco

Panel 1: Identifying interventions that might have different                 
effects across a population (Video)                   

Mark Levenson, PhD
Deputy Director, FDA CDER, Division of Biometrics VII

Case examples: Lessons learned from past trials
Robert Temple, MD
Deputy Center Director for Clinical Science, FDA CDER

Using clinical pharmacology and biology to anticipate and
to account for differences in efficacy and safety across a population

Issam Zineh, PharmD, MPH, FCP, FCCP
Director, FDA CDER Office of Clinical Pharmacology
Office of Translational Sciences

Use of epidemiologic studies to examine safety in diverse populations
Judy Staffa, PhD, RPh
Director, FDA CDER Division of Epidemiology II
Office of Pharmacovigilance & Epidemiology
Office of Surveillance & Epidemiology

Precision medicine and ethnic labeling of genetic variants
Charles Rotimi, PhD
Chief and Senior Investigator, Metabolic, Cardiovascular and Inflammatory
Disease Genomics Branch
Director, Center for Research on Genomics and Global Health National
Human Genome Research Institute, National Institutes of Health

Panel 2: Participant diversity in clinical trials participants (Video)                      

Janet Holbrook, PhD
Associate Professor, Department of Epidemiology
Johns Hopkins University Bloomberg School of Public Health

Diversity in trials of medical devices: Experiences from the field
Paul Underwood, MD
Medical Director, Boston Scientific Corporation

Multi-regional clinical studies and diverse populations: Where they
Overlap, and where they don't

Bruce Binkowitz, PhD
Executive Director, Merck Research Laboratories

Enhancing Minority Participation in Clinical Trials: Consortium
Initiatives, Resources, and Tools

Jennifer Wenzel, PhD, RN, CCM, FAAN
Associate Professor, Johns Hopkins School of Nursing;
Johns Hopkins School of Medicine Sidney Kimmel Comprehensive
Cancer Center, Cancer Prevention and Control Program

Drug Trials Snapshots and Transparency: Opportunities and Challenges
Naomi Lowy, MD
Lead Medical Officer, Professional Affairs and Stakeholder
Engagement (PASE), FDA CDER

Panel 3: Strategies to identify effect moderators (Video)    

Elizabeth Stuart, PHD
Professor, Department of Mental Health,
Johns Hopkins University Bloomberg School of Public Health

Calibrated Risk Adjusted Modeling (CRAM): A bridge for design for extending
the applicability of randomized controlled trials

Ravi Varadhan, PhD
Associate Professor, Johns Hopkins School of Medicine

Strategies to identify effect moderators: opportunities and limitations of
big data to address diversity

Shawn Murphy, MD, PhD
Corporate Director, Research IS and Computing, Parnters Healthcare

The role of meta-analysis in identifying diversity in efficacy and safety
Daniel Caños, MPH, PhD
Associate Director, FDA CDRH, Division of Epidemiology
Robbert Zusterzeel, MD, PhD
Research Fellow, FDA CDRH

Panel 4: Identifying meaningful outcomes for diverse populations (Video)                  

Laura Lee Johnson, PhD

Multiple morbidity: Including people with representative comorbidities
Cynthia Boyd, MD
Associate Professor, Department of Health Policy and Management, JHSPH
Johns Hopkins School of Medicine

Diverse Considerations in the Use of Mobile and Sensor Technologies for Clinical Research
William Riley, PhD
Director, NIH Office of Behavioral and Social Sciences Research

Choosing outcomes that are appropriate across a population: Deliberative approaches
Evan Mayo-Wilson, MPA, DPhil
Assistant Scientist, Department of Epidemiology
Johns Hopkins Bloomberg School of Public Health

Choosing outcomes that are appropriate across a population: Quantitative approaches
John Bridges, PhD
Associate Professor, Department of Health, Policy and Management
Johns Hopkins Bloomberg School of Public Health


Designing for diversity: Inclusivity in trial design                           
Estelle Russek-Cohen, PhD
Director, FDA CBER Division of Biostatistics

Jonca Bull, MD
Assistant Commissioner for Minority Health, FDA

Caleb Alexander, MD, MS
Co-Director, Center for Drug Safety and Effectiveness
Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health

Attendee Information & Directions

The public workshop will be held at FDA White Oak Campus, Building 31 Great Room.  Entrance for the public workshop participants (non-FDA employees) is through Building 1 where routine security check procedures will be performed. For parking and security information, please refer to the FDA information page


For questions and concerns, please email Evan Mayo-Wilson at


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