Minorities and Underserved Populations in COPD Drug Trials

The importance of clinical trial outcomes cannot be dismissed in provider evidence-based decisions when it comes to treating patients. However, the ability of a new drug to be successful in treating all possible patients cannot be accurately determined if certain key demographic data is not reported in the trial outcomes. Chronic lung disease, such as Chronic Obstructive Pulmonary Disease (COPD) has higher rates in underserved communities such as minority and rural areas. Still, there is evidence that respiratory diseases, and the therapies to treat them, are not equal in all ethnic groups in the U.S.

COPD Drug Trial Demographics Reporting

The need for the ethnic backgrounds of clinical trial participants to be included in the reported outcome is two-fold in COPD:

1. First, it has been reported that ethnic minorities, and other underserved groups, have higher instances of health issues due to lower socioeconomic status. Ethnic minorities, such as Blacks and Hispanics, have a higher risk for chronic lung disease than Whites because of lower socioeconomic status (Assari, Chalian, & Bazargan,2020). Doctors’ offices, where patients are typically funneled into clinical trials, are not prevalent in rural or minority areas. Clinical trial researchers rely on physicians to recruit patients into trials; however, this can increase the barriers. The design of the U.S. healthcare system has only amplified the disparities in these groups, including rural populations.
2. The second reason for ethnic backgrounds to be reported in clinical trial outcomes is the genetic differences in how lung disease and treatment responses are present in ethnic groups. As per a guideline published by the American Thoracic Society (ATS) and European Respiratory Society (ERS) in 2005, the standardization of spirometry encourages the use of race and ethic predictors rather than using an adjustment calculation (Braun and Wolfgang, 2013). Why does race or ethnic background matter? There are several anthropometric differences, such as height. The height, age, and gender of a person is a crucial determinant of lung size, and therefore lung capacity. In a person from an ethnic group that is considerably taller on average, lung disease would be affected quite differently than if the person was from an ethnic group of typically shorter stature. Another factor in how COPD affects people with the disease is genetics. Several environmental and genetic interactions play a part in whether a person develops COPD. This argument is strengthened because fewer than 50% of smokers develop COPD during their lifetime (Hadfield & Hess, 2020). There is little doubt that a person’s genetics influence if they are more susceptible to disease. Given that genetic variations affect disease processes and response to medications, to disregard ethnicity from trial publications is not in the spirit of providing the best care.

The NIH Revitalization Act

The National Institutes of Health Revitalization Act of 1994 set the framework for including minorities and other underrepresented groups, such as women and children, in clinical trials. The Revitalization Act created the Office of Research on Minority Health which tasked the NIH to draft guidance on recruitment of minorities. Per the policy as of 2001, it is the responsibility of the NIH Director to enforce all federally funded clinical trials to be designed in a manner to “provide for valid analysis of whether the variables being studied in the trial affect women or minority groups differently.”

FDA Guidance on Clinical Trial Demographics Reporting

Clinical trials outside the authority of the NIH are not under the same restrictions; however, a 2019 draft guidance by the FDA directly charges sponsors “to adopt practices for determining eligibility criteria that will allow clinical trial population to reflect the diversity of the patients who will be using the drug if approved” (Center for Drug Evaluation and Research, 2019, page 4). However, since the FDA guidance is voluntary, enforcement is lacking. The sponsors mentioned by the FDA guidelines are the same companies that produce many respiratory-related drugs and therapies outside of federal oversight. They routinely do not publish the ethnic backgrounds of the trial participants.

The Severe Underrepresentation in Reporting

According to a literature review completed by Burchard et al., in 2015, just over 20 years since the Revitalization Act was passed, less than 5% of industry-sponsored respiratory-related clinical trials have reported racial or ethnic backgrounds. The lack of clarity in the efficacy of respiratory drugs in ethnic minorities and underserved populations does a disservice to those disproportionately affected by chronic lung disease, such as COPD. The effect is compounded when considered that underserved populations also tend to be at a socioeconomic disadvantage, having higher incidences of chronic lung disease. Health disparities, low socioeconomic status, and higher incidences of chronic lung diseases in these racial and ethnic minorities and underserved populations with less than 5% representation in respiratory clinical trials create a homogeneity in the data that does not accurately reflect the population.

About COPD

COPD is a preventable and treatable disease of the airways, with a limitation of flow due to remodeling of the lungs, which includes abnormalities in the alveoli (Hadfield and Hess, 2020). COPD affects millions of people in the United States. The Centers for Disease Control and Prevention reports that around 16 million people in America have COPD. Due to many tobacco users in the baby boomer age group (people born in the years between 1944-1964), the number of people with COPD is growing. This chronic lung condition is on track to become the third leading cause of death in the United States and globally (Hadfield & Hess, 2020). COPD has a late-onset due to the slow progression of the disease.

Many people at risk fail to seek treatment until they are in the advanced stages of the disease state, typically around 40-50 years of age. The combination of normal progression in lung function with long-term exposure to toxic inhalants is thought to contribute to the disease. The change to the airways is not immediate, and therefore, it is seen in older adults. It is still not well understood why age seems to be a factor in the development of COPD; however, some research shows that genetic factors and environmental exposure play a part in the disease’s progression.