What I Wish I Knew: Diversity in Clinical Trials

Clinical trials are a testing phase for drugs, devices, and procedures. These research studies evaluate the effectiveness and safety of a medical intervention in humans.

Clinical trials are essential to advancing medical treatments, including those for cancer. Historically, the trials have largely been conducted on white males. Now, medical professionals are examining how gaps in representation within these trials may affect treatment approaches and patient outcomes in different groups, such as females and ethnic minorities.

Health spoke to Anita Phung, a general practitioner who helps oversee clinical trials conducted with Lindus Health, an England-based medical research organization. Experts like Phung advocate for wider representation in clinical trials to expand treatment options and provide more individualized care. Understanding genetic differences could be particularly crucial for advancing cancer prevention and treatment.

“Cancer is complex, and genetic variations across different people can further influence treatment outcomes. Researchers are just scraping the surface of the importance of diversity in understanding health risks and medication effects across different populations,” said Phung.

What do you wish you had known sooner about the role of diversity in medical research?

Anita Phung: Previous clinical trials were basically done on a homogeneous population, meaning people of the same background. This was typically white males.

Looking back, it means that many of the medications I’m prescribing and treatments I’m recommending to my patients are based on very biased and potentially inaccurate data. For certain drugs, the research available hasn’t taken into consideration differences in sex or ethnicity.

Now that I know that, I’m hoping that I can do more to facilitate a balancing of representation in medical research. There is no one-size-fits-all medication. Different individuals can have different responses to the same medication, which is why multiple types of similar medications—and the research behind them—are necessary.

How have clinical trials evolved to be more inclusive?

Phung: In the medical community, there has been a growing interest and focus on pharmacogenomics. This is the study of how genetic variations, or differences in a person’s biological makeup, can influence how the body responds to drugs.

Your genetics may affect how your body metabolizes a drug, how effective a drug might be for you, and the side effects of the drug you experience. This all plays into how well you respond to a medication.

In general, I think people are taking more interest in addressing inequalities and trying to involve underrepresented populations. I’m seeing more and more studies focused on ethnic minorities.

What do you wish you had known earlier about how adequate representation in cancer clinical trials can affect people’s disease outcomes?

Phung: Research focused on specific populations has been able to determine which medications are most effective for treating conditions common in these groups.

For example, my grandma is of Southeast Asian, Chinese background, is a non-smoker, and was diagnosed with lung cancer. The hospital she went to did genetic testing on her. At the time, I wasn’t familiar with lung cancer and genetic testing, so I didn’t understand why. But they were testing to see which medication she would respond to most effectively and would give her the best outcomes.

Her lab results came back positive for an EGFR mutation, which is a change in the epidermal growth factor receptor gene that affects how cells grow and divide and may increase cancer spread in the body. This mutation, as well as non-smoking-related lung cancer, is more common among East Asian populations.

If you test positive for this mutation, you are prescribed a different medication than if you test negative. Clinical trials have shown that the medication Gilotrif (afatinib) is most effective for treating non-small cell lung cancer in East Asian populations with an EGFR mutation, which tells healthcare providers this drug should be a first-line treatment for this group.

As a result, my grandma received the best treatment available for her, for someone of her background. If she had been prescribed a different medication, she may have had a poorer prognosis or experienced more side effects. I’m grateful that this expanded research was done.

What conversations should people be having with their oncologist about personalized care?

Phung: It’s important to be curious. Read up on the treatment your doctor recommends, and ask questions. For example, has this treatment been tested with people of my background—people of the same sex, ethnicity, or health conditions? Are there differences in how people might respond to this treatment? What are the other treatment options?

Your doctor might not know all the answers, but they can find out. Too much information can be overwhelming, especially when approaching cancer treatment, but it’s your doctor’s job to help you understand the information you need to make an informed decision.

For doctors, it’s important not to make assumptions about how a person wants to approach their cancer treatment. They should give their patients all the options. Some people might want to participate in a clinical trial for the greater good, to contribute to science and help develop better treatments, and that’s okay, too. Joining a clinical trial has potential risks and benefits, and those should be weighed.

Knowing what you know now about the lack of representation in medical research, what would you tell your younger self?

Phung: Think about the bigger picture. Just because guidelines tell you what you’re supposed to do, you don’t necessarily need to follow them. Guidelines are guidelines, and many were written based on a single population. Be critical and really appraise the profile, safety, and research behind the drug, especially if the risks and benefits have significant outcomes for the patient.

Having read more work on diversity in medical research has made me reflect on my practice and examine the data on even the medications I’m taking and my relatives are taking.