NATIONAL HARBOR, Maryland — The “highest quality science is inclusive,” Nadia Bodkin, PharmD, founder of the Rare Advocacy Movement (RAM), told participants of a roundtable discussion at the 2021 World Orphan Drug Congress. RAM sponsors and encourages patient activism and collaboration at a grassroots level.

One such collaborative effort was the Our Lives Matter (OLM) Diversity, Equity, and Inclusion (DEI) Initiative, which began in May of 2020 following the events surrounding the death of George Floyd. It delved into research on inherent bias in the medical system.

“Controls aren’t inclusive,” Dr. Bodkin said. “They are based on white, European, 40-year-old men, so what does this mean for women, older or younger individuals, or people of color?”

Another insight explored during the OLM initiative was related to what Dr. Bodkin called “race-based pseudoscience.” This occurs when clinical trials recruit members of only one race. Therefore, these studies automatically are no longer generalizable to the public, rather only a small subset of patients. The social construct of race counters the concept of “highest quality science,” she said.

Dr. Bodkin and her RAM colleague, Kay-Diene Robinson, also an OLM DEI expert, argued that such race-based classification for clinical trials should be eliminated entirely for true inclusivity during the scientific process. Robinson also brought up the topic of tokenism and questioned how companies can move away from empty gestures and instead achieve meaningful action.

Rare disease patient advocacy groups have the power to challenge the existing system by collecting data representative of all races, genders, and age groups to guide and inform the highest quality science and research, Dr. Bodkin said. A strong, patient-centric foundation guided by patients’ lived experiences in turn may prevent the failure of clinical trials that are not relevant to the patient experience — saving time, effort, and money.

One barrier to accessing such valuable data within underrepresented communities involves a lack of established relationships built upon trust, which take effort and time to develop and nurture. If the medical community and industry “communicate authentically to establish trust with patients, then patients will genuinely want to help with these studies,” Dr. Bodkin said.

Rural and remote communities pose another challenge as social isolation prohibits easy access to quality health care, pharmacies, foods, the Internet, and essential resources easily obtained in cities. The nearest hospital may be miles away, and questions remain about how individuals in these communities can consistently obtain the same quality of care commonly administered elsewhere.

Many questions still need to be addressed and there is no single source to determine the best path forward, Dr. Bodkin and roundtable participants agreed. Both Dr. Bodkin and Robinson admitted they would “rather not be experts in DEI,” but would “prefer to be experts in the highest quality science,” and they encourage others to be as well.


Diversity, equity, and inclusion (DEI): insights and lessons learned from the Our Lives Matter DEI Initiative. Presented at: World Orphan Drug Congress USA 2021: August 26, 2021; National Harbor, MD.