Strategies for Ensuring Diversity, Inclusion, and Meaningful Participation in Clinical Trials: A Workshop

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FDA Office of Minority Health The Institute of Medicine, Roundtable on

1 Strategies for Ensuring Diversity, Inclusion, and Meaningful Participation in Clinical Trials: A Workshop Jonca Bull, MD Director, FDA Office of Minority Health The Institute of Medicine, Roundtable on the Promotion of Health Equity and the Elimination of Health Disparities April 9, 2015

30th Anniversary US Health and Human Services (HHS) 1985 2003 IOM Report Landmark effort in analyzing and synthesizing the present state of knowledge of

It represents the first time the Department of Health and Human Services (DHHS) has consolidated minority health issues into one report.

2 30th Anniversary US Health and Human Services (HHS) IOM Report Landmark effort in analyzing and synthesizing the present state of knowledge of the major factors that contribute to the health status of Blacks, Hispanics, Asian/Pacific Islanders, and Native Americans. It represents the first time the Department of Health and Human Services (DHHS) has consolidated minority health issues into one report. documented the existence of health disparities among racial and ethnic minorities in the United States and called such disparities "an affront both to our ideals and to the ongoing genius of American medicine."

3 July 2012: FDASIA Section 907 History American Heart Association, WomenHeart, and Society for Women s Health Research Lobbied Congress for legislation requiring FDA to publicly report data on the inclusion and analysis of women in FDA applications Resulting Legislative Proposal: Heart for Women s Act (HEART) Sen. Debbie Stabenow (D-MI) and Rep. Lois Capps (D-CA) Provision added to include reporting of a race and ethnicity Sen. Benjamin Cardin (D-MD) Final legislation reauthorizing FDA user fees (essential for Agency operations) - Section 907 incorporated HEART

Section 907 Requirements: A Report Within one year of enactment: Provide to Congress Post on FDA website Report On: Extent of clinical trial participation Quality of analyses to determine safety and

4 Section 907 Requirements: A Report Within one year of enactment: Provide to Congress Post on FDA website Report On: Extent of clinical trial participation Quality of analyses to determine safety and effectiveness for demographic subgroups included in applications submitted to FDA Take into account FDA regulations and requirements for protection of sponsor confidential commercial information One year after the report post an Action Plan addressing deficiencies

5 Congressional Concerns for Action Plan April 30, 2014 Letter to FDA led by Senator Stabenow Require representative proportions of women and minorities in industry-sponsored clinical trials comparable to NIH Specific actions FDA will take, in cooperation with industry: To achieve meaningful subgroup analyses for safety and efficacy Clear timelines for enforcement that do not unnecessarily disrupt trials Making results transparent and publicly available Publicly reporting progress implementing the Action Plan: Regular Further action needed

6 mendmentstothefdcact/fdasia/ucm htm

7 August 2014: Section 907 Action Plan Three overarching priorities: Priority One: Improve the completeness and quality of demographic subgroup data collection, reporting and analysis (Quality) Priority Two: Identify barriers to subgroup enrollment in clinical trials and employ strategies to encourage greater participation (Participation) Priority Three: Make demographic subgroup data more available and transparent (Transparency)

8 907 Action Plan Status Update- Highlights Steering Committee chaired by Barbara Buch, MD, Associate Director For Medicine, CBER Strengthened collaboration with NIH 907 Steering Committee NIH Inclusion Governance Group NIH Office of Research in Women s Health and FDA OWH Medwatch Form: Update at Office of Management & Budget Closing on 4/27/15: FDA OMH Federal Register Notice public input on FDA research priorities in minority health and health disparities Transparency-Drug Trials Snapshot provides information about the sex, age, race and ethnicity of clinical participants for recently approved drugs. Today s IOM Meeting- Ensuring Diversity, Inclusion and Meaningful Participation

FDA Drug Snapshots http://www.fda.gov/drugs/informationondrugs/ucm412998.

9 FDA Drug Snapshots Zontivity: To reduce the risk of heart attack, stroke, cardiovascular death, and need for procedures to restore the blood flow to the heart in patients with a previous heart attack or blockages in the arteries to the legs. Figure 1: Zontivity Baseline Demographics by Sex Figure 2: Zontivity Baseline Demographics by Race

FDA Drug Snapshot: FARYDAK FARYDAK Table 1: FARYDAK Baseline Demographics (n = 758) Prescribed in combination with bortezomib and dexamethasone Indicated for the treatment of

10 FDA Drug Snapshot: FARYDAK FARYDAK Table 1: FARYDAK Baseline Demographics (n = 758) Prescribed in combination with bortezomib and dexamethasone Indicated for the treatment of patients with multiple myeloma who have received at least 2 prior regimens, including bortezomib and an immunomodulatory agent Table 2: Baseline Demographics by Race (Safety)

11 FDA Policy for Clinical Database and Inclusion for New Drugs The database submitted in a marketing application should reflect usage in a diverse racial population, one reflective of the likely patient mix postmarketing, for potential differences in response to become apparent (Good Review Practice for INDs Dec 2013) Inclusion and exclusion criteria Co-morbidities, BMI, concomitant meds Global : Who are the subjects in the trials? Geographic/Ancestry/Living conditions/culture/differences in standards of medical care outside the U.S. which may impact outcomes Valid? Representative

FDA Commissioner Margaret Hamburg (August 2014): One of the core tenets of rigorous biomedical research, as well as a guiding principle of the FDA s goal to meet the health needs of patients across

12 FDA Commissioner Margaret Hamburg (August 2014): One of the core tenets of rigorous biomedical research, as well as a guiding principle of the FDA s goal to meet the health needs of patients across the demographic spectrum, is the importance of encouraging diversity in clinical trials. When a more diverse population participates in clinical trials, we increase the potential to know more about the extent to which different subgroups males and females, young and old, people of various racial and ethnic backgrounds, and patients with differing comorbid diseases and conditions might respond to a medical product. And when subgroup data are analyzed, we have available more information about the product that can be communicated to the public. The result is greater assurance in the safety and effectiveness of the medical products used by a diverse population.

13 As We Begin Some Points to Consider: Health equity perspective (social justice) and improving participation Health disparities-- What to measure? How to measure? Classifications are not consistent with OMB definitions (and also must take into account that data is often being generated globally) There are significant issues about the constructs of race, ethnicity and gender What is meaningful participation and clinical trial design? Hypothesis generation: which drugs need to be identified early for special consideration for subgroups? Estimating the treatment effect within a group or the heterogeneity of treatment effect across groups What steps can FDA and NIH take in working with academia and industry?

Office of Minority Health (OMH) at Work in Indian Country

Office of Minority Health (OMH) at Work in Indian Country J. Nadine Gracia, MD, MSCE Deputy Assistant Secretary for Minority Health Director, Office of Minority Health U.S. Department of Health and Human