A framework for setting enrollment goals to ensure participant diversity in sponsored clinical trials in the United States.

BACKGROUND: Diversity in clinical trials (CTs) has the potential to improve health equity and close health disparities. Underrepresentation of historically underserved groups compromises the generalizability of trial findings to the target population, hinders innovation, and contributes to low accrual. The aim of this study was to establish a transparent and reproducible process for setting trial diversity enrollment goals informed by the disease epidemiology. METHOD: An advisory board of epidemiologists with expertise in health disparities, equity, diversity, and social determinants of health was convened to evaluate and strengthen the initial goal-setting framework. Data sources used were the epidemiologic literature, US Census, and real-world data (RWD); limitations were considered and addressed where appropriate. A framework was designed to safeguard against the underrepresentation of historically medically underserved groups. A stepwise approach was created with Y/N decisions based on empirical data. RESULTS: We compared race and ethnicity distributions in the RWD of six diseases from Pfizer's portfolio chosen to represent different therapeutic areas (multiple myeloma, fungal infections, Crohn's disease, Gaucher disease, COVID-19, and Lyme disease) to the distributions in the US Census and established trial enrollment goals. Enrollment goals for potential CTs were based on RWD for multiple myeloma, Gaucher disease, and COVID-19; enrollment goals were based on the Census for fungal infections, Crohn's disease, and Lyme disease. CONCLUSIONS: We developed a transparent and reproducible framework for setting CT diversity enrollment goals. We note how limitations due to data sources can be mitigated and consider several ethical decisions in setting equitable enrollment goals.

Disease-Specific Health Disparities: A Targeted Review Focusing on Race and Ethnicity.

BACKGROUND: Wide disparities in health status exist in the United States across race and ethnicity, broadly driven by social determinants of health-most notably race and ethnic group differences in income, education, and occupational status. However, disparities in disease frequency or severity remain underappreciated for many individual diseases whose distribution in the population varies. Such information is not readily accessible, nor emphasized in treatment guidelines or reviews used by practitioners. Specifically, a summary on disease-specific evidence of disparities from population-based studies is lacking. Our goal was to summarize the published evidence for specific disease disparities in the United States so that this knowledge becomes more widely available "at the bedside". We hope this summary stimulates health equity research at the disease level so that these disparities can be addressed effectively. METHODS: A targeted literature review of disorders in Pfizer's current pipeline was conducted. The 38 diseases included metabolic disorders, cancers, inflammatory conditions, dermatologic disorders, rare diseases, and infectious targets of vaccines under development. Online searches in Ovid and Google were performed to identify sources focused on differences in disease rates and severity between non-Hispanic Whites and Black/African Americans, and between non-Hispanic Whites and Hispanics. As a model for how this might be accomplished for all disorders, disparities in disease rates and disease severity were scored to make the results of our review most readily accessible. After primary review of each condition by one author, another undertook an independent review. Differences between reviewers were resolved through discussion. RESULTS: For Black/African Americans, 29 of the 38 disorders revealed a robust excess in incidence, prevalence, or severity. After sickle cell anemia, the largest excesses in frequency were identified for multiple myeloma and hidradenitis suppurativa. For Hispanics, there was evidence of disparity in 19 diseases. Most notable were metabolic disorders, including non-alcoholic steatohepatitis (NASH). CONCLUSIONS: This review summarized recent disease-specific evidence of disparities based on race and ethnicity across multiple diseases, to inform clinicians and health equity research. Our findings may be well known to researchers and specialists in their respective fields but may not be common knowledge to health care providers or public health and policy institutions. Our hope is that this effort spurs research into the causes of the many disease disparities that exist in the United States.