By James Gillespie and Kathleen Drennan
For an industry committed to delivering innovative healthcare, and where science outpaces antiquated trial planning and processes, there is no better time than now to take stock of the current burgeoning shift in population demographics and how it will affect the business of new drug development. But could it take another few decades to bridge the disparities gap of underrepresented patient populations in clinical trials compared to disease incidence — specifically Black/African Americans and Hispanic/Latinos, who will be the largest consumers of healthcare (2010 U.S. Census)? It took until the middle of the 1990s to acknowledge the lack of inclusion of women in clinical trials in any significant numbers, leaving a legacy of medicines extrapolated from studies done only in men. The industry missed opportunities to recognize critical scientific and gender issues affecting optimum treatments and outcomes in women’s health, and it missed the biggest opportunity of all: Women are the largest buyers and decision makers of healthcare purchases.
A Complex Challenge And Strong Call To Action
The FDA is rapidly pushing the current diversity agenda of reducing disparity between disease incidence and prevalence in minority populations and the sample size represented in clinical trials. The FDA established the Office of Minority Health in 2010 to advance the Agency’s regulatory oversight in achieving the highest standard of health for all.
Is industry, including CROs, giving adequate attention and proactive planning to guidelines and suggestions, which will surely become an issue at the time of FDA scrutiny, of their clinical trials and new drug approval? How many companies are addressing and including “multicultural competency” across their research endeavors necessary to make progress?
What is cultural competence as it relates to developing new medicines through clinical trials for the three primary stakeholders as guided by the FDA?
For industry, it is a set of congruent behaviors, attitudes, and policies coming together in a system, agency, or among professionals enabling those entities to understand and work effectively in cross-cultural situations.
For clinical sites, it is culturally competent physicians and study coordinators providing patient-centered care by adjusting attitudes and behaviors to account for the impact of emotional, cultural, social, and psychological issues on the disease.
For patients, culture is a set of behaviors taught and inherited as members of a particular group, guiding how to view the world and healthcare, how to experience it emotionally, and how to behave in relation to other people and their disease.
Racial and ethnic segmentation strategies demand a departure from business-as-usual in the development of clinical trials, especially when envisioning trial-site communications, patient education materials, and doctor/patient relationships. New mindsets and skills are needed to address long-entrenched patient skepticism and deeply rooted mistrust about the accuracy of healthcare information, medical institutions, and especially the industry itself.
The Stakes Are High
In 2011, the FDA approved the first new drug to treat lupus in 56 years. Lupus disproportionately affects women, and usually develops between ages 15 and 44. Estimates vary on the number of lupus sufferers in the United States, ranging from approximately 300,000 to 1.5 million people. People of all races can have the disease; however, African American women have a three times higher incidence (number of new cases) than Caucasian women. Two clinical studies with lupus demonstrated the safety and effectiveness of the drug, but patients of African heritage participating in the two studies did not appear to respond to treatment. The studies lacked sufficient numbers to establish a definite conclusion, so the sponsor agreed to conduct an additional study of people with those backgrounds to further evaluate the safety and effectiveness of this lupus drug.
This is a recurring story suggesting that proactive planning of such protocols could eliminate having to go back and do additional studies. The most dramatic of examples is the incidence of diabetes in the United States versus adequate study enrollment representation. Looking at published data readily available from many sources on the Internet, diabetes affects over 60% underrepresented populations, whereas enrollment of such populations in clinical trials averages about 11% or less.
The ability to reach and motivate patient communities representing populations needed to participate in today’s clinical trials is not insurmountable; however, it is challenging. Progressive and enlightened companies that embrace clinical trial diversity as an integral part of their research planning and processes will build new bridges with the patient and physician communities. Adopting a multicultural competence methodology at the clinical-team level must include aggressive strategy and investment deviating from the current norm.
James Gillespie, Ph.D., J.D., M.P.A., is president of the Center for Healthcare Innovation, president of the National Biotechnology & Pharmaceutical Association, and president of the National Hispanic Life Science Society.
Kathleen Drennan is founding president and CEO of TrialAdvance, Inc., established to bring real-world insights of patients and trial sites to the relevant planning, execution, and management of clinical trials.