Clinical Trials Can Make Anyone a Hero

For the first study I spent two hours in an MRI machine watching videos, some funny, others scary, while researchers scanned me to determine which parts of my brain were most active when I expressed different emotions to what I was watching.

The next day, when I returned to the lab, a colleague showed me her arm that was wrapped in cellophane to protect the injection sites from doses of an investigational intradermal drug. She was enrolled in a study assessing a new, and potentially safer, method of administering an old drug.

At the time, neither of us thought about the profound impact of what we were doing, but as I pursued my career into the world of clinical research and medicine I realized the enormity of my participation. Without human clinical trials like those, researchers could never develop newer, safer, or more effective ways to treat disease–and save lives.

What would a pharmacy look like without clinical trial volunteers? This public service announcement is a collaboration between Sanofi and the Center for Information and Study on Clinical Research Participation (CISCRP).

Respecting the volunteers

Now more than ever, human participation in clinical research is essential for advances in medicine, as there is an urgent need for more clinical trial participants during the COVID-19 pandemic. Over the years, however, clinical trials have faced serious challenges, some of which are still with us.

A history of racism and fear of maltreatment in clinical research have led to hesitancy in participation.¹ The publication of the Belmont Report² in 1979 was the culmination of more than four years of research by the National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research, and a landmark moment in clinical research. The commission was formed in part due to the results of the Tuskegee experiment: the infamous, cruel syphilis experiment performed without full consent on African-American men in the southern United States.³

In addition, patient volunteers are often consulted on the design of a clinical trial, forming a “patient panel” of experts. Clinical research is conducted under international rules and regulations to protect the safety and wellbeing of trial participants, irrespective of where the trials are conducted.

A key moment for clinical trial participants

The Belmont Report² changed how human research would be performed by establishing three ethical principles:

• Respect for Persons–Acknowledging autonomy and protecting those with diminished autonomy
• Beneficence–Do no harm; maximize possible benefits and minimize possible harms
• Justice–Fair treatment and distribution of benefits

The result was increased oversight of clinical trials: today, volunteers are viewed as equal partners in research. They are fully informed of their rights, the potential benefits of participating and, importantly, the potential risks. 

Diversity in clinical trials

However, the third principle from the Belmont Report, “Justice”, has yet to be truly achieved. There is still a troubling lack of diversity among clinical trial participants with far too few people like me taking part. People of color and members of minority communities are underrepresented in research studies, many of which disproportionally affect those same communities.

We need to actively encourage more participation in trials by minorities, particularly women. The research community is actively reaching out to these groups to let them know how their participation could contribute to fight diseases that affect their communities, but those efforts must be intensified as most people don’t even know that they have the chance to be a hero.

Under-representation in research

A 2020 report from the United States Food and Drug Administration (FDA) estimates that globally, 76% of all clinical trial participants are white, while Asians represent 11% and black participants of African descent 7%.⁴ This skewed distribution fails to capture the diversity of human populations. The US FDA also reported⁵ that in 2019, 48 new medicines were approved thanks to 46,931 volunteer participants in clinical trials. Of those participants, 9% were Black or African American, and in oncology trials only 4% were Black or African Americans.⁵  Yet, this group represents 13.4% of the population.⁶

This has enormous implications, given the prevalence of aggressive cancers among African Americans. For example, lung cancer occurrence is 15% higher in black men compared to their white counterparts, and multiple myeloma is 2.5 to 3.5 times higher in black men and women under age 50.⁷ We also know that women and people of color may respond differently to the same medication due to genetic variances.⁸

Diversity in clinical trials can help save lives

Without broadly diverse participation in clinical trials, we are unlikely to understand the impact of race, ethnicity, and gender-related differences in how the human body responds to drugs–and those differences could save lives.

For example, drugs against enzymes called "tyrosine kinase inhibitors" can have a positive impact in Asian females with certain types of lung cancer. These drugs have led to a 26% reduction in premature death for Asian females, compared to about 16% for non-Asian males.⁹ That life-saving knowledge is thanks to the heroes who volunteered to participate in studies on those drugs.

The path to becoming a clinical trial hero

On International Clinical Trials Awareness Day, and every day, I hope you will connect with someone you know who has participated in research, ask them about their experience, and thank them for advancing medicine. I was fortunate: Working in a research laboratory setting, my opportunity was right in front of me on that bulletin board, but we can’t always rely on such happy accidents to bring the diverse participation that science and society so desperately need. We have to be actively engaged to meet that third goal of justice and ensure human clinical trials benefit everyone.