Regulatory Science Symposium: Diversity in Clinical Trials in the Time of COVID-19 Session 3: FDA Initiatives to Address Diversity in Clinical Trials

1. Disclaimer
a. FDA makes sure medical treatments are safe and effective for people to use
b. FDA does not develop new treatments or conduct CTs
c. FDA provides guidance to academia via NIH and industry
2. FDA Guidance on Diversity
a. Does not dictate what sponsors should do to make their trials more diverse
b. FDA recommends recruiting the population that is being afflicted by the diseases being studied
3. Clinical Trial Participation
a. Racial and ethnic minorities are underrepresented 
b. This is a problem as people of different ages, races, and ethnicities may react differently to medical products
4. Diversity in Sex/Gender
a. Historically, women were excluded from CTs – there were concerns regarding hormonal interference
b. Now, women are in CTs in representative numbers (51%)
i. There is still a need for women from diverse backgrounds to participate
5. Office of Women’s Health (OWH) 
a. Women excluded from CTs due to pregnancy, reproductivity concerns, especially thalidomide tragedy
6. FDA Office of Women’s Health
a. Project HOPE: Making sure that indications that women suffer from are being studied and developed
7. FDA Safety and Innovation Act (FDASIA -2012)
a. Enacted by Congress to ensure there are funds and oversight
b. Section 907: Requires FDA to write and submit an action plan to Congress for attaining good quality data
c. Section 907: Explain why demographic subgroups are not being included
d. Diversity information will appear in the labeling
8. FDA Action Plan (2014)
a. Priority 1| Quality: Associated reporting and analysis of data
b. Priority 2 |Participation: Priority of FDA to identify barriers to CT access and for sponsors to employ strategies to encourage recruitment of certain populations
c. Priority 3 |Transparency: Making demographic subgroup data more available and transparent, leading to accountability
9. Priority 1
a. 1.1: Review and develop a plan to update/finalize relevant demographic subgroup data
b. 1.2: Work with sponsors to enhance information on demographic subgroups in medical product applications
c. 1.3: Add education/training on demographic inclusion, analysis and communication of clinical data for FDA reviewers
d. 1.4: Improve FDA systems for collecting, analyzing, and communicating diverse clinical information
e. 1.5: Conduct research on specific areas of public health concern, relating to demographic subgroups
10. FDA Guidance 
a. Can separate out the broad Asian and Pacific Islander categories
11. Priority 2
a. Participation – need to encourage CT participation by demographic subgroups
b. What are the barriers to subgroup participation? Need to know these barriers in order to overcome them
12. Priority 3 
a. Information that is available on the FDA website or dailymed.org is not written for regular consumers
b. FDA have implemented communication strategies to address this issue
13. Labeling and Literacy – Drug Trials Snapshots
a. Part of the action plan to address transparency and patient literacy
14. Drug Trials Snapshot – ADLYXIN® (lixisenatide)
a. Written in a Q&A manner as seen in OTC drug labels and informed consent forms
b. With an aging geriatric population, grouping everyone above 65+ into one category is not sufficient since there are physiological differences (65 vs 75year-old)
15. FDA Office of Minority Health and Health Equity (OMHHE)
a. Established in 2010, this is the first office at FDA dedicated to protecting and promoting health of diverse populations
16. OMHHE – Diversity in Clinical Trials Initiative  
a. Goal = raise awareness around racial and ethnic minority participation in CTs
b. Utilizes multimedia campaign to highlight the value, risks and benefits of diverse population in CTs
17. Clinical Trials Transformation Initiative (CTTI)
a. This is public and private membership organization, FDA and CTTI members
18. CTTI – Diversity Project
a. Intrinsic and extrinsic factors can lead to a disparity in the practice of medicine for demographic subgroups
19. CTTI Diversity Findings on Investigators/Sites
a. Research teams at sites face barriers due to lack to cultural competency and a lack of resources and staffing to develop strategies to recruit underrepresented populations
20. Case Study #1
a. TODAY (Treatment Options for Type 2 Diabetes in Adolescents and Youth) study
i. Native Americans have highest prevalence of type 2 diabetes of any racial or ethnic group
ii. University of Oklahoma included a representative population and integrated study into healthcare ecosystem
iii. Result: study successfully engaged and retained Native American adolescents
21. Case Study #2
a. Patient Navigator Program
i. University of Alabama at Birmingham’s Comprehensive Cancer Center
ii. Provided community education and conducted needs assessments to support patients, with goal being to increase participation of Blacks in CTs
iii. Result: significant increase in enrollment and retention of the Black population in CTs
22. CTTI Diversity Findings on Sponsors
a. Sponsors can provide resources and incentives, promoting diversity initiatives on the research team
23. Case Study #3
a. Lupus (2 to 3 times more prevalent in women of color, creating a sizeable health disparity)
i. Recent Lupus treatment approved by FDA, did not have robust data on efficacy in a diverse population
ii. In post-marketing research, company found mixed results on the treatment’s efficacy in the Black population
24. NIH Initiatives
a. NIH is mandated by Public Health Service Act to ensure inclusion of women and minority groups in all NIH-funded clinical research
b. Goal: ensure research findings can be generalizable to entire population
25. Diversity in Covid19 Trials
a. In FDA guidance regarding COVID-19 medical product development, states sponsors should place CT sites include locations with a higher concentration of racial and ethnic minorities to recruit a diverse study population
26. Final Thoughts
a. Increasing diversity in CTs is a group effort--- the clinical research community and sponsors 
27. Questions
a. Hollywood and Media Industries: Portrayal of CTs 
b. Does FDA provide guidance for transgender individuals? Currently, No.
i. Is it the biological sex, environmental or social factors affecting their health?
ii. More research is needed
c. In a COVID-19 vaccine CT, the site is proposing only health care workers should be included in the trial. Is this a diversity problem?
d. CTs in Africa: Access issue, making sure sites are adequately trained and can execute, infrastructure difficulties i.e. government policies and relations that can hinder CT development
e. An Interdisciplinary Outreach Model of African American Recruitment for Alzheimer’s Disease Research:  https://academic.oup.com/geron... 
f. EBOLA therapy is now approved; when one needs to study do studies in Africa, then it is possible

Accompanying text created by Annie Ly | Graduate Student, Regulatory Science, USC School of Pharmacy lyannie@usc.edu