This blog was originally published on HYPER’s LinkedIn on 20 May 2023.

This Clinical Trials Day, Mümün Gencoglu reflects on existing challenges to clinical trials diversity and how the role of ethics and scientific innovation can nurture a better research environment – not only today but for future generations. More information about this topic can be found in this HYPER webinar on diversity, equity, and inclusion in clinical trials.

Clinical trials are essential in the pharmaceutical industry’s drug development process to ensure the safety and efficacy of new drugs before they reach the market. The history of clinical trials dates back to the late 18th century when Scottish physician James Lind conducted the first recorded controlled trial on the effects of citrus fruits in treating scurvy. Since then, clinical trials have become a cornerstone of medical research, with randomized controlled trials becoming the gold standard for evaluating the safety and efficacy of new drugs^1,2.

However, despite their long history and essential role in drug development, clinical trials have been subject to persistent issues of inequity and inequality, raising significant ethical, social, and economic concerns. Certain populations, such as racial and ethnic minorities, women, the elderly, and individuals from low-income backgrounds, are underrepresented in clinical trials. This poses significant challenges in understanding how drugs will work in these populations and may lead to serious health disparities in accessing and benefiting from new drugs. These populations may also face additional barriers to accessing healthcare services and may be disproportionately affected by the adverse effects of drugs ^1,3.

Overcoming historical hurdles through a more inclusive approach

Regulators and policymakers, together with drug manufacturers, have recognized the need for greater equity and diversity in clinical trials and are taking steps to address this issue. For example, the US FDA has encouraged the inclusion of underrepresented populations in clinical trials through its guidance documents and has established the Office of Minority Health and Health Equity to promote health equity and eliminate health disparities ^3,4. Similarly, the European Medicines Agency (EMA) has established policies to encourage greater diversity in clinical trials and has recognized the need for increased participation of underrepresented populations ⁵.

Despite these efforts, significant challenges remain in achieving greater equity and equality in clinical trials. The issue of clinical trial participation is complex, with numerous factors contributing to underrepresentation, including socioeconomic status, language barriers, cultural beliefs, and mistrust of the medical establishment. Addressing these challenges requires a multifaceted approach that involves engaging with underrepresented communities, increasing awareness and education, and building trust between researchers, regulators, and the public ^5,6.

Recognizing innovation as an indispensable tool for better representation – and better results

The issue of clinical trial equity and equality is not only a matter of social justice but also of scientific necessity. The lack of diversity in clinical trials undermines the validity and generalizability of study results and may lead to unforeseen adverse effects when drugs are approved for wider use. Failure to include underrepresented populations in clinical trials may limit the development of innovative drugs that address unmet medical needs in these populations ^6,7.

The consequences of clinical trial inequity and inequality extend beyond healthcare and scientific fields. It also has economic implications, as healthcare costs can increase due to the lack of effective treatments for certain populations ⁷. It can impact the pharmaceutical industry’s bottom line, as drugs may not be as effective as they could be if they are not adequately tested on a diverse population.

Innovative approaches to clinical trial design are being explored to address some of the challenges posed by traditional randomized controlled trials. For example, adaptive trials allow for changes in the study design based on ongoing data analysis, potentially reducing the number of participants needed and increasing the efficiency of the study ¹⁰. Real-world evidence studies, which gather data from sources such as electronic health records and insurance claims, can provide valuable information on how drugs work in different populations outside of a traditional clinical trial setting.

Altering the clinical trials’ landscape for the benefit of patients

Clinical trials should include patient populations that mirror the epidemiology and demographics of those who would benefit from the treatment being tested. The inclusion of diverse participants in clinical trials is important for both equity and scientific accuracy. Every individual should have an equal chance to participate in relevant clinical trials ⁸.

To address these challenges, various organizations, including the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), are working to increase patient and community engagement in clinical trials, as well as address structural barriers that prevent underrepresented groups from participating. This includes initiatives to increase awareness and education about clinical trials, provide language and cultural support, and engage with underrepresented communities to build trust and understanding ⁹.

Clinical trials are a vital component of drug development, but the persistent issues of inequity and inequality pose significant ethical, social, and economic challenges. The lack of diversity in clinical trials undermines the validity and generalizability of study results, perpetuates health disparities, and hinders scientific progress ^1,2,4,10.

Young people in the healthcare industry are already proactively advocating for and participating in initiatives that promote diversity in clinical trials. By amplifying their voices and pushing for greater equity and equality in clinical trials, the next generation of healthcare leaders can contribute to a more just and effective healthcare system for all.

References:

1. Eichler, H.-G., Bloechl-Daum, B., & Abadie, E. (2013). The risks of risk aversion in drug regulation. Nature Reviews Drug Discovery, 12(12), 907-916. https://doi.org/10.1038/nrd4090
2. Freedman, B. (1987). Equipoise and the ethics of clinical research. New England Journal of Medicine, 317(3), 141-145. https://doi.org/10.1056/NEJM198707163170304
3. Grover, A., & DeMets, D. L. (2018). More equitable clinical trials and the future of research. Journal of the National Cancer Institute, 110(8), 801-803. https://doi.org/10.1093/jnci/djy077
4. Hellström, L., & Höglund, A. T. (2013). Inclusion and exclusion criteria in clinical trials: A review of the literature. Applied Nursing Research, 26(4), 197-201. https://doi.org/10.1016/j.apnr.2013.08.004
5. Kimmelman, J., & Federico, C. (2017). Consider drug efficacy before first-in-human trials. Nature, 542(7640), 25-27. https://doi.org/10.1038/542025a
6. Largent, E. A., Lynch, H. F., & McCabe, M. S. (2018). Clinical trial participation as part of a long-term care insurance benefit: Perceived benefits and concerns. Journal of Medical Ethics, 44(1), 33-37. https://doi.org/10.1136/medethics-2016-103411
7. Mills, E. J., Seely, D., Rachlis, B., Griffith, L., Wu, P., Wilson, K., Ellis, P., Wright, J. R., & Gafni, A. (2006). Barriers to participation in clinical trials of cancer: A meta-analysis and systematic review of patient-reported factors. The Lancet Oncology, 7(2), 141-148. https://doi.org/10.1016/S1470-2045(06)70576-9
8. https://www.ifpma.org/publications/diversity-and-inclusion-in-clinical-trials-bioethical-perspective-and-principles/
9. Pocock, S. J. (1983). Clinical trials: A practical approach. John Wiley & Sons.
10. Sankar, P., Cho, M. K., Condit, C. M., Hunt, L. M., Koenig, B. A., Marshall, P., Lee, S. S.-J., & Spicer, P. (2004). Genetic research and health disparities. Journal of the American Medical Association, 291(24), 2985-2989. https://doi.org/10.1001/jama.291.24.2985

Author

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  Mümün Gencoglu Associate Director, Innovation Research and Analytics m.gencoglu@ifpma.org