By Akiko Shimamura, SVP, Trial Design and Optimization, TriNetX
Earlier this year, the FDA rejected an application for a new cancer drug because it was based on a trial conducted in China, and the agency ruled it did not represent the U.S. population.[1] In 2021, the FDA’s Oncologic Drugs Advisory Committee noted in their review of another cancer drug that “members of racial minority groups relevant to who would receive the drug in the U.S. were under-represented”.[2]
These decisions are just the tip of the iceberg of what promises to be an increasing number of rejections based on lack of representation as the FDA steps up its insistence on more diversity in clinical trial participants. Recruiting diverse populations has been, and will continue to be, a massive challenge for the development of new medicines and advancing health equity. A recent analysis of 32,000 individuals who participated in new drug trials in the U.S. in 2020 revealed that only 8% were Black, 6% Asian, 11% Hispanic, and 30% over the age of 65.[3]
Diversity, Equity, and Inclusion (DEI) have become a front-and-center issue, and both the pharmaceutical and healthcare industries need to take notice. Unfortunately, current market solutions and existing data fall short—most data sources are siloed within institutions and do not have comprehensive diversity data. Additionally, diversity data is not seamlessly integrated into a clinical trial protocol feasibility process within most life science organizations.
From diversity to representation
When we talk about diversity, we are really talking about representing the underlying patient population in a particular trial. Representation means including individuals of the ethnic, racial, or social demographic group that matches the target population for the drug being tested. For example, Sanofi’s Diversity by Design approach looks at the demographics of the population impacted by the indication beforehand to set diversity targets for the trial.
Obtaining a representative cohort can also be impacted by the diversity of the research and development teams involved in the trial. A more diverse investigator group is likely to recruit a more representative cohort. When a diverse investigator group talks to participants about a potential trial opportunity, they are more likely to use language that resonates with the representative population.
Artificial Intelligence (AI) technology can be impactful in helping to ensure representation. It is time-consuming for a group of analysts to run 15 or 20 iterations to optimize a protocol. With AI, analysts can run thousands of iterations to come up with more comprehensive results more quickly.
The importance of representation in clinical trials
Recruiting a representative patient cohort for a trial is critical to examine disease and response to the therapy across different patient subgroups and to support the generalizability of the results to the broad patient population. Appropriate representation is crucial to ensure equal access to clinical trials as a treatment option, ensuring all communities share in the potential benefits of the research. The FDA now requires diversity action plans for clinical trials, building on a series of guidance released from trial design to post-marketing approaches.
While the concept of diversity as a social responsibility of pharmaceutical companies is important, achieving representation in clinical trials is also critical for robust evidence generation. Trials with representative cohorts provide important insight to sponsors, better demonstrating the efficacy and safety of the treatment in a way that is relevant for providers and payors post-launch.
Ensuring representation in a clinical trial can also help minimize the need for more studies or additional required research post-launch. In contrast, running a clinical trial with a cohort that is not representative could lead to regulatory rejection, as seen in the examples above. Failing to gain approval is extremely expensive for a pharma company that has already expended millions of dollars to get to that point. Further trials only cause delays, delaying the delivery of life-saving treatments to patients.
The greatest barrier to gaining the appropriate level of representation is obtaining access to a diverse patient database. Starting with widely diverse data allows pharma to target a representative cohort that matches the underlying patient population for the drug. Without an extensive database with patients that represent the general population, selecting the appropriate group of patients becomes nearly impossible.
How TriNetX is helping
TriNetX has established the world’s largest de-identified electronic medical record database, with over 250 million patients worldwide from more than 30 countries and 250 healthcare organizations. Our diversity data is comprehensive — encompassing 82% of known race and 73% known ethnicity values for patients who had a visit in the last year. This ensures that pharmaceutical companies and CROs using TriNetX data can be sure to find and select both depth in clinical and diversity data for protocol optimization, site identification, and patient recruitment.
The TriNetX platform is a critical part of the machinery of clinical trials, with 14 of the top 15 large pharmaceutical companies leveraging the platform and Diversity Lens feature to better evaluate the impact of inclusion and exclusion criteria on target and underrepresented populations.
Our mission at TriNetX is to ensure that clinical trials represent the patients the therapy is intended to serve and for our clients to generate the most robust evidence possible to support regulatory and post-launch activities. TriNetX helps accomplish these goals by optimizing protocol queries and designs to meet diversity targets and identify diverse populations.
Explore how TriNetX is pioneering the advancement of Diversity, Equity, and Inclusion in clinical trials with data at TriNetX | Advancing Diversity, Equity, and Inclusion with Data – TriNetX
[1] FDA New Diversity Plan Guidance, And What It Means for Sponsors Developing Cancer Drugs, Applied Clinical Trials. Click Here.
[2] 3 Ways the FDA is Taking Action On Lack Of Diversity In Cancer, Clinical Trials, Clinical Leader. Click Here.
[3] Embracing Diversity: The Imperative for Inclusive Clinical Trials, Harvard Medical School, Trends in Medicine. Click Here.