Big data analysis of more than half a million patients highlights severe diversity issues in US clinical development design and recruitment
Boston, USA, 2022: Significant new analysis from Phesi Inc., a data-driven provider of AI-powered clinical development analytics products and services, has found that 42% of US cancer trial cohorts do not include African American patients and 48% have no Hispanic American patients. Phesi analyzed 589,295 patients participating in 6,372 US-recruiting cancer clinical trials over the past 15 years (Figure). In April 2022, the FDA issued a draft guidance to improve enrolment of underrepresented racial and ethnic populations in clinical trials, encouraging trial sponsors to define enrolment goals for underrepresented groups as early as practicable in clinical development, highlighting cancer as a priority area for improvement.
“It is clear why the FDA has issued the recent guidance on clinical trial diversity, as 1/3 of the population is not being represented in clinical trials, creating a problem in ensuring that treatments are beneficial for all,” explained Dr Gen Li, President, Phesi. “Clearly, this guidance does not go far enough to deliver real change to achieve patient diversity in clinical trials, mainly because there are no incentives and no consequences.”
According to the 2020 US Census 18.7% of the US population identify as Hispanic and Latino, and 12.1% are Black or African American, and according to the American Cancer Society, Black people have the highest death rate and shortest survival rate for cancer of any ethnic group in the US, and are at a greater risk of developing stomach, liver and cervical cancers.
This lack of patient diversity is likely in part driven by lack of equitable access to healthcare in the US, according to Dr Li: “Clinical trial recruitment sites are typically selected based on quality of facilities and equipment – there is potential that the lack of equal access to healthcare in the US is further driving health disparities all the way to drug development.”
“Data is available to support clinical development organizations with identifying appropriate recruitment sites and selecting patients that represent the population. Clinical development companies need to use it when planning trials. By taking steps such as considering recruitment sites earlier in trial design, and assessing patient demographics early on, clinical development companies can achieve appropriate representation of all ethnic groups in clinical trials.”
Drugs that come from trials conducted in a cohort that is not representative of the population could be less effective in other ethnic groups or even have adverse effects. Biological differences exist in how people respond to certain therapies. For example, variations in genetic coding can make a treatment more (or less) toxic for one racial or ethnic group than another.
“To see entire racial and ethnic groups excluded from clinical trials in important disease areas such as cancer highlights significant failings in clinical trial design and recruitment. It underlines the need to use data analytics in protocol design,” explained Otis Johnson, Chief Diversity, Inclusion & Sustainability Officer, Clario. “As new medicines are being developed for the benefit of the entire population, it is dangerous that the population is not being truly reflected in pivotal clinical trials and further entrenches existing healthcare inequalities.”
“Data generated from clinical trials without diverse enrolment should not be considered good quality. It is essential that patient diversity becomes a part of the definition of data quality because without this, there is no guarantee of quality for those not represented during the clinical development process,” continued Johnson. “Representation of all racial groups in the clinical development process is essential in ensuring that new therapies are effective for all and not just the privileged few.”
Figure: Ethnic composition of cancer clinical trial cohorts in the USA.