By Cindy Dubin, Contributor
This year marks the 10th anniversary of Clinical Trials Day—a day meant to champion and celebrate clinical research professionals in their relentless pursuit of knowledge and commitment toward patient well-being. Clinical Trials Day was founded by the Association of Clinical Research Professionals, which has made transforming the clinical trials landscape one of its top goals.
Yet, an Iqvia study reveals that despite increasing sponsor focus on diversity in clinical trials and diversity data reporting, and recent Food & Drug Administration (FDA) guidance on diversity data reporting and clinical program diversity planning, Black/African American and Hispanic patient inclusion failed to reach US demographic levels on average across interventional trials, including U.S. sites in the past decade.
This is supported from the results of a 27-year study by Johns Hopkins, which found that only 40% of clinical trials included data on patient race or ethnicity, and of that 40%, 94% were white, 2.1% were black, and .6% were Hispanic. Upon review of the data, the study researchers said: “Racial and ethnic minorities have been vastly underrepresented in clinical research and there is a need to enroll these populations in future trials to address potential differences in clinical trials.”
Additionally, a recent analysis in Health Affairs revealed that a five-year action plan launched by the FDA in 2015 aimed at improving diversity in, and transparency of, pivotal clinical trials. The journal reported that there was little evidence to suggest he action plan did anything to improve representation of Black trial participants, and that fewer than 20% of drugs had data regarding treatment benefits or side effects reported for Black patients.
Financially and socially, the lack of diversity in trials will cost the US “billions of dollars” over the next three decades, according to a 2022 report by the National Academies of Sciences, Engineering and Medicine. The report also said trials that aren’t inclusive hinder innovation, fail because of low enrollment rates, undermine trust, and worsen health disparities.
Regulatory agencies, clinical trial patient advocacy groups, and Big Pharm continue to take steps to reduce and possibly eliminate disparities. To that end, this month, a National Action Plan for Achieving Diversity in Clinical Trials was launched by a consortium that supports coordination, learning, and accountability for transformation across the clinical trials enterprise. This call to action will reach out to community leaders and government agencies aimed at achieving diversity in clinical trials.
Big Pharma’s Diversity Efforts
Big Pharma is also doing its part to transform and diversify clinical trials. For example, Pfizer spent nine years assessing its own efforts of enrolling diverse clinical trial participants and determine how many of its trials actually achieved census level diversity. In fact, representation was not uniform across 213 trials. This led Pfizer to take steps to increase diversity among research participants and publicly commit to achieving racially and ethnically diverse participation at or above U.S. census or disease prevalence levels, as appropriate, in all of its clinical trials.
In addition, Pfizer has evolved its approach to set recruitment and site selection goals for each therapeutic area. In doing so, Pfizer claims it draws on criteria such as the prevalence of a particular disease across demographic groups and census data for various groups’ representation in the population. Pfizer’s clinical trial teams develop recruiting strategies to meet the objective and to ensure that the enrollment criteria do not unduly prevent individuals from particular racial or ethnic groups from participating. For example, if a certain disease that is prevalent among a particular racial group is listed as an exclusion criteria (e.g., individuals with that disease are not eligible to participate in the clinical trial), the clinical trial team will determine whether that exclusion criteria is necessary or whether it can be safely altered or eliminated.
Similarly, Bristol Myers Squibb has been focused on its own global efforts to increase diversity in clinical trials for almost 10 years. The company’s Diversity in Clinical Trials Career Development Program has been recognized as a unique program — the first that will train scholars to be community-oriented clinical trialists.
Using AI to Increase Participation Rates
Other leading drug makers are using artificial intelligence to facilitate clinical trial diversity. This includes finding patients for clinical trials quickly or reducing the number of people needed to test medicines—both aimed at accelerating drug development and potentially saving millions of dollars. Reuters reported that the FDA received about 300 applications that incorporate AI or machine learning in drug development between 2016 and 2022. More than 90% of those applications came in the past two years and most were for the use of AI at some point in the clinical development stage.
The complexity of clinical trials has required pharma companies to rely on academic medical centers, but these populations may not be as diverse. Computer algorithms can help researchers quickly review data on past medical studies, search through medical records from around the world, and assess the distribution of disease in a population. That data can help drug makers find new networks of doctors and clinics with access to more diverse patients who fit into their clinical trials—sometimes months faster and much more cheaply than if humans were reviewing the data.
J&J, for example, is using AI to increase participation rates of Black Americans in clinical trials. Algorithms helped J&J pinpoint community centers where Black patients with multiple myeloma (which is more likely to affect Black Americans) might seek treatment. The machine-learning algorithm computes and creates a heat map for you as to where the patients eligible for that trial are. That information actually helped lift the Black enrollment rate in five ongoing studies to about 10%, the company says. J&J is now using AI to increase diversity in 50 trials and plans to take that number to 100 this year.
Elsewhere, Takeda Pharmaceutical Co. uses AI to help attract and retain diverse populations in its clinical trials by personalizing letters of consent to patients in minority groups. The technology can adjust wording to correspond to how people identify themselves by gender and sexual orientation.
Additionally, Walgreens Boots Alliance Inc.—which began running clinical trials for drug makers in 2022—uses AI to locate eligible patients from diverse groups, and relies on local pharmacists at its almost 9,000 US stores to recruit individuals from underrepresented groups. According to the company, this actually improved participation of Black patients in one cardiovascular study to 15%, a number that exceeds the percentage of Black people in the general population.
AI does pose challenges for drug makers, however, because the technology carries the risk of making things worse than they already are by introducing what’s known as algorithmic bias. In 2019, for instance, academics said they uncovered unintentional racial bias in one software product sold by Optum Inc., a health services company, which health centers across the country used to predict which patients needed high-risk care. Bloomberg reported that algorithm based its predictions on patients’ health-care spending, rather than the severity or the needs of their illness. Only 18% of Black patients ended up getting additional help, rather than the 47% who needed it, according to a study of the algorithm’s effects at one institution that was published in the journal Science. Its authors say that skew is typical of risk prediction tools that medical centers and government agencies use to service 200 million people nationwide, and that such bias likely operates in other software as well. Optum maintains that the algorithm is not racially biased.
Global Regulatory Agencies Focus on Diversity
In November, Bloomberg reported that the FDA is considering drafting recommendations for companies that are submitting AI applications for drug development to ensure their models don’t inadvertently discriminate against underserved patients. In the meantime, regulators are increasingly want drug makers to consider such disparities when vetting new treatments. In 2014, the European Medicines Agency introduced guidance requiring drug makers to justify non-representative clinical trials. Australia’s Therapeutic Goods Administration’s 2022 guidelines say drug study populations should represent the makeup of the broad population. And, the Food and Drug Omnibus Reform Act (FDORA) of 2023 established legislative mandates for increasing clinical trial diversity and demand prioritization of representation in clinical trials. FDORA will require trial sponsors to include diversity action plans for Phase 3 and other pivotal studies of new drugs. Companies that fall short will face repercussions.
Decentralized Clinical Trials and use of digital technologies are additional components of FDORA. Digital health technologies have greatly expanded the types of trial-related activities that can be conducted remotely and the scope of data that can be collected. And, the use of decentralized elements, such as obtaining laboratory tests at a local facility (rather than requiring a participant to travel to an academic medical center) or conducting follow-up visits in a participant’s home or via telemedicine, increases the convenience of such activities for research participants and can assist in diversity efforts.