Regulatory
Percent participation in clinical trials by subpopulation* for new molecular entities and therapeutic biologics approved in 2020
In 2020, CDER approved 53 novel drugs, either as new molecular entities under new drug applications or as new therapeutic biologics under biologics licence applications. Overall, 32,000 patients participated in these trials. Selected subpopulation demographics from these trials are presented in the table below.
Women Average White Black or African American Asian 56% 75% 8% 6% Hispanic Age 65 and older 11% 30%
* The percentage of all other races combined (American Indian or Alaska Native, Native Hawaiian or other Pacific Islander, other, and unknown/unreported) makes up to 100% of race category. * The percentage of Non-Hispanic and unknown/unreported ethnicity makes up to 100% of ethnicity category. * The percentage of patients from anywhere else in the world makes up to 100% of geographic category. Source: FDA
something that Diana Foster, SCRS vice-president of strategy and special projects, says is crucial in getting a diverse population on board in the first place. “[Sites need to] understand not only the demographic mix required for the potential population, but they [also] need to understand where they need to point to recruit a diverse population,” she says. “Then they are truly looking at their actual patient.”
Patient trust
Mistrust of clinical research, and medical professionals more generally, among certain populations can prevent or make them less likely to enrol. A survey by Demand Diversity, a campaign run by patient recruitment company COUCH Health, found that feelings of exploitation, dehumanisation and deep mistrust were barriers to trial participation among people of colour in the UK. Cultural and religious factors, such as concerns with ‘giving part of the body’ among those of Islam faith, also factored. Before researchers plan anything, they need to understand these patients’ challenges and what would prevent them from enrolling in a trial, says COUCH CEO Ash Rishi. This may involve physically going out to meet community groups or religious leaders, and simply asking for a chat. “We’ve gone into boroughs, mosques, synagogues […] and just asked, ‘Can we have ten or five
Guidance developments
In November 2020, the US FDA issued final guidance, ‘Enhancing the Diversity of Clinical Trial Populations – Eligibility Criteria, Enrollment Practices, and Trial Designs,’ with the agency’s recommendations on designing and executing clinical trials of drugs and biologics that include people with different demographic characteristics (for example, sex, race, ethnicity, age, location of residency) and non-demographic characteristics (for example, patients with organ dysfunction, comorbid conditions, and disabilities; those at weight range extremes; and populations with diseases or conditions with low prevalence). Also in November 2020, the Pharmaceutical Research and Manufacturers of America (PhRMA) announced industry-wide principles on clinical trial diversity. The principles focus on four main areas: building trust and acknowledging the historic mistrust of clinical trials within black and brown communities, reducing barriers to clinical trial access, using real-world data to enhance information on diverse populations beyond product approval, and enhancing information about diversity
and inclusion in clinical trial participation. In April 2021, these principles took effect. Source: US FDA and PhRMA
minutes?’ And if they’re not comfortable, we ask if we could leave our information or leaflets,” says Rishi. During these conversations, Rishi establishes understanding by asking what a person’s previous experiences with healthcare have been like. As well as building trust, these patient insights can then be used to inform inclusive trial design and to develop a culturally sensitive retention strategy. Community outreach is important, but if sites don’t continue the work once a trial is under way, only so much can be achieved, adds Foster. “We can mobilise and attempt to move the needle and enrol diverse populations, but the sites have got to be responding, and be able to be trained and knowledgeable in doing this work,” she says. Here, FDA guidance suggests that clinical investigators and research staff complete cultural competency training as a way to build patient trust and learn how to engage with participants of different backgrounds.
Doing the work
Regulators play an important role in defining clinical trial conduct and, from a UK perspective, Harris believes that the MHRA could do more to support researchers by communicating what they can expect from them as an agency. “At the end of the day, you are generating evidence for the regulator in order to get marketing, authorisation, etcetera,” she says. “[For] the next step, I think some companies would just really [like] at least a nod in the right direction.” However, too much regulation risks turning diversity into a tick-box exercise, she adds. Foster agrees, noting that while guidance like the FDA’s is helpful in moving the needle, industry professionals shouldn’t feel like they need to wait for an explicit mandate to make their work more inclusive. It’s often said that change starts on an individual level and it’s important that industry professionals accept accountability for the role they could play in promoting diversity within clinical trials. “We often talk about hard-to-reach groups,” says Harris. “But we need to put the responsibility on the research community here, it’s about them being underserved by us.” ●
10 Clinical Trials Insight /
www.worldpharmaceuticals.net US 54%
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