Contributed Commentary by Ryan Bailey

September 28, 2018 | Clinical research hinges on the principle of the representative sample. Using a small, statistically-derived sampling of data, we determine if a treatment is safe and efficacious for the broader patient population. Hence, one of our driving goals should be to enroll subjects that represent, as closely as possible, our ultimate patient demographic. Unfortunately, our industry has long struggled with underrepresentation of important demographic groups. Whether it’s age diversity, socio-economic diversity, or racial and ethnic diversity, many populations have been marginalized in the clinical trials recruitment process.

Yet, we know diseases differ between different demographic subgroups, and we know treatments can likewise vary by factors like age, race, and gender. This poses both a scientific and an ethical concern for us.  If our study subjects aren’t reflective of the patients we intend to treat, how can we be sure our products and devices work properly and safely? 

This dilemma is not new, but it is one that continues to hamper our industry and it is drawing increased scrutiny from regulatory agencies. In recent years, the FDA has been focused on improving trial diversity through several forums. In 2010, they created the Office of Minority Health.  In 2014, following the passage of FDASIA, the agency developed an action plan to encourage greater participation among diverse patient subgroups. This was followed two years later by FDA Commissioner Robert Califf declaring 2016 to be the Year of Diversity in Clinical Trials. Earlier this year, the FDA conducted a public workshop to evaluate inclusion and exclusion criteria in clinical trials to address whether they are too stringent and understand how they unintentionally discourage needed diversity in clinical trial populations.      

With this rising emphasis on enrolling subjects that more realistically represent the patients we intend to treat, we may soon hear a new twist on an old slogan: “No medication without representation.” 

No one group has all the answers, but Rho team members have conducted trials in different populations, and the projects and teams I’ve worked on have begun to establish our own internal best practices for recruiting and enrolling more diverse populations. Below are just a few ideas based on strategies we have implemented successfully in our trials.

Employ Culturally Competent Staff

Successful recruitment and retention of trial subjects depends on our ability to build trust with them.  Trust requires that we understand and value what our patients value.

• Hire staff from the racial and socio-economic population you are trying to reach.  Such employees are invaluable at speaking the “cultural language” and building instant rapport with patients.
• Speak the language.  If you are recruiting patients who speak English as a second language, and you have no one on your staff who can speak their native tongue, you may face difficulty establishing clear communication and trust.
• Learn and honor the culture and customs of your patients.  Don’t be afraid to ask your patients if you don’t understand, and seek out knowledge from those with more information. Few things will discourage participation like misunderstanding and offending your patients.

Learn About The Needs Of Diverse Patient Groups

Do you know what limitations and barriers to participation face your patients?  How will you work to overcome these challenges?

• Reach out to patient advocacy groups to learn about patient needs and develop collaborative partnerships.
• Consider potential barriers to participation among your demographic subgroups.  For example, how will your patients reach the clinic site?  Some elderly may not be able to drive.  Some in lower socioeconomic status may not have access to a car.  Some may not have easy access to public transit.  Once you understand their challenges, consider how you can help them overcome these barriers (e.g., reimbursing for transit costs, providing complementary transportation, having a mobile clinic that can go to their home.)

Re-evaluate Protocol Design With The Patient In Mind

A protocol is often designed by scientists, statisticians, and investigators to maximize scientific output, but if we do not consider the patient perspective, we risk overlooking the most basic needs of our most important asset.

• Invite patients to contribute to the protocol design or conduct protocol focus groups and listen to patient feedback.  In one project with pediatric patients, we learned that we needed to offer clinic hours at night and on weekends to accommodate single working parents who were unable to take extended leave from their jobs to make visits during traditional business hours.
• Evaluate your inclusion and exclusion criteria. A well-meaning criterion may inadvertently have the effect of preventing an important subgroup from participating.  For example, many trials establish age limits for participation that are mostly arbitrary. If you are researching a medication for heart disease but you cap enrollment at age 59, you are excluding a large portion of the population who are most at risk for heart disease: those 60 and older.

These strategies call for a patient-centric mindset: a way of thinking about the patient, for the patient, and from the patient’s perspective, from the planning stages of your trial all the way through implementation.

Ryan Bailey, MA is a Senior Clinical Researcher at Rho. He has over 10 years of experience conducting multicenter asthma research studies, including the Inner City Asthma Consortium (ICAC) and the Community Healthcare for Asthma Management and Prevention of Symptoms (CHAMPS) project. Ryan also coordinates Rho’s Center for Applied Data Visualization, which develops novel data visualizations and statistical graphics for use in clinical trials. He can be reached at ryan_bailey@rhoworld.com.