Original Research by Rama Bhagwat, 2020-2021
Research I conducted between August 2020 and March 2021 highlights the need to address both clinical trial representation, as well as medicine effectiveness based on race and ethnicity. Good progress has recently been made by academic research institutions and companies to begin addressing disparities in clinical trial representation. However, addressing medicine effectiveness based on race and ethnicity continues to be overlooked as a potentially important bioethical issue.
While limited in its scope by my resources and the lack of access to proprietary data on real world outcomes, I believe my research, conclusions and implications create a compelling case for additional comprehensive study and analysis of these issues. Additionally, this research makes the case for better – more comprehensive and standardized – reporting requirements from the FDA for medical effectiveness and adverse events data by race and ethnicity. We can not improve what we do not measure!
I focused my research on three diseases – Diabetes, Prostate Cancer, and Lung Cancer and eight approved medicines / therapies and their publicly available registration clinical trial data. My research conclusions included:
- For the disease – medicine combinations I researched there was under representation of the racial and ethnic minorities in the clinical trials used to approve the medicines
- Five of the eight medicines demonstrated significantly lower effectiveness, as measured by the Hazard Ratio, for Black patients as compared to White patients.
- Lack of adequate representation of Blacks in the registration clinical trials may be contributing to approved medicines that may be less effective in Black patients as compared to White patients.
There are three potentially far reaching and significant implications of this research:
- Expand the public availability and consistent reporting of data that is currently available for pre-approval clinical trials, and expand it to post-approval trials, adverse event reporting and medicine effectiveness. One of the challenges of conducting this research was that so much of the medicine effectiveness data are proprietary to the pharmaceutical companies. While the FDA website provides good transparency into the racial and ethic makeup of clinical trial participants, this same transparency does not carry over to the reporting of medicine effectiveness data or reporting of the adverse events by race and ethnicity, post approval of the medicine. Just as the FDA has begun efforts to provide draft guidelines to ensure that clinical trial participants represent the racial and ethinc diversity of the patient population, I believe it is imperative, based on this research, that these guidelines be extended to medicine effectiveness measures and adverse event reporting.
- Efforts must be made to systematically study, understand and address the complex non-medical factors – such as ethical and socio-economic factors – that contribute to disproportionately low representation of Blacks in clinical trials. Solutions must include addressing race and ethinic group specific factors such as access to healthcare, community outreach and cultivating trusted advocates and spokespersons in local communities.
- In addition to informing better data reporting standards and guidelines, another potentially important implication of this research is that – approving medicines because they are effective on an average, or based on registration trials that are not representative of disease demographics, should no longer be good-enough. Certainly not in this age of improving healthcare equality, mega-data, AI and ML enabled precision medicine. Patients should not be given medicines that are far less effective for them, especially when more effective alternatives may exist. The data on Hazard Ratio for Prostate Cancer in Black men as compared to White men, for approved medicines was eye opening!
Summary Slides
