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The pursuit of patient diversity in clinical trials

When all-inclusive isn’t an option, it’s an obligation

Everyone knows that increasing the diversity of clinical trial populations is a top priority for global pharma.

But the fact remains that there’s a long way to go before the reality matches the rhetoric. The numbers tell the story. Despite the pandemic, 2020 was a banner year for pharma innovation, with 53 novel medicines approved by the FDA – the second highest on record. However, of the 32,000 patients who took part in trials for these medicines, 75% were white.

Just 8% were black or African American and even fewer (6%) were Asian. These damning figures, the latest in a series of drug trial snapshots published by the FDA, aren’t unusual. A five-year summary of clinical trial participation reveals a familiar pattern: of 292,766 trial participants from 231 snapshots between 2015 and 2019, just 7% were black or African American and 76% were white. This, of course, is crude analysis.

Patient diversity isn’t limited to race and ethnicity – it encompasses everything from age, gender and sexual orientation, to socio-economic status, religion, literacy, culture, location…. and much more beyond. However, if the goal of clinical research is to conduct trials that reflect real life, it’s clear the industry is falling short. Despite a renewed focus on patient diversity in clinical trials, the needle has barely shifted. There’s much to do.

Diversity is diverse

The FDA has been addressing the broad issue of diversity in clinical trials since the 1980s, when it issued guidance on the importance of studying the effects of drugs in elderly populations. Since then, it’s published guidelines around gender and sex differences in the clinical evaluation of drugs, and developed measures to address the underrepresentation of racial and ethnic minorities.

In recent years, the focus on race and ethnicity has intensified, resulting in a global effort to promote the benefits of clinical trial participation and increase inclusion from diverse populations. The rationale is well understood. Clinical trial populations must reflect the real-world population most likely to use an approved drug, and since few diseases are specific to a single demographic, trial populations must also be broad and representative.

Unfortunately, however, the diversity gap that’s long bedevilled trial participation remains wide, and certain groups are still greatly under-represented. It’s a huge issue. The struggle for inclusion plays into – and indeed exacerbates – wider narratives around health disparities and inequalities, and the social determinants of health.

In November 2020, the FDA issued new guidance to help industry enhance the diversity of clinical trial populations through broadening eligibility criteria and adapting trial designs. The report is an attempt to shift the conversation from ‘why’ to ‘how’ – offering a series of recommendations to help companies develop more inclusive trial practices and drive more proportional representation from marginalised populations.

However, making the leap from good intentions to authentic achievement requires significant change. So how is industry faring? And what do companies need to do to improve performance?

The COVID exemplar

At the headline level, the signs are encouraging. “Pharma is increasingly committed to ensuring trial populations are more diverse than they perhaps were in the past,” said Nuala Murphy, President, ICON Clinical Research Services. “A good example is Pfizer’s COVID-19 vaccine trial, which was conducted at around 150 trial sites across the US, Argentina, Brazil, South Africa, Germany and Turkey. The trial not only targeted geographies with high infection rates, but it was also designed to ensure the vaccine was developed in countries where the patient population was adequately represented. Over 42% of overall trial participants came from diverse ethnic backgrounds.”

Certainly, the rapid development of COVID vaccines was a phenomenal achievement made possible through unprecedented global collaboration. Similarly, the diversity seen in trial populations was no doubt driven by a worldwide determination to find a way out of the pandemic.These were exceptional circumstances.

The challenge, moving forward, is to build on the learnings to ensure the successful approaches companies adopted aren’t the exception, they’re the rule. Mark Evans, Managing Director, Havas Lynx Faze, thinks a wholesale change in approach is required. “With 70% of trials failing to recruit, the focus right now is often on finding the easiest patients to recruit – without realising that using the same old approaches risks missing out on a huge group of underserved people. ‘Site burden’ continues to be an unacceptable reason for failing to recruit patients when the burden patients face is far greater and happens every day.

“We need to shift from the old model of catering to the needs of sites and focusing only on site burden, to the unique needs of the patients we are trying to serve. If we do this effectively and authentically, trials will need to start considering the real people they are intended for rather than the ‘echo chamber’ at which they are often designed without a representative group of patients involved.”

All about patients

The answer, as always, is to focus studies around the needs of patients. But companies must make sure their attempts to be patient-centred are reflected in behaviours not buzzwords.

“Patient-centricity was a conversation about good intentions rather than business critical,” said Mark Evans. “As such, many companies haven’t moved it high enough up on their agendas, often restricting it to a few specific roles and a PR story or two. It’s important that the current focus on diversity in trials doesn’t go down the same route. We need to understand it’s not just the right thing to include all people, but that the long-term success of trials and products depends on understanding and serving the real people we’re trying to help and make real change to address the fundamental challenges faced.”

Jo Fearnhead-Wymbs, Vice President, Patient Engagement, Ashfield MedComms, said early patient engagement is critical when it comes to developing new medicines. “We cannot make assumptions, we need to talk to the patients who’ve got experience of the condition we’re targeting to identify what’s important to them in the real world. Diversity and inclusion in that engagement process is essential. If we want to design studies around endpoints that are relevant to patients – rather than just focusing on clinical markers identified by expert medics – we need the broadest possible exposure to the target population.

"Ultimately, we have to ensure that clinical trials sample the people who are going to get the most value from a medicine – because if we’re not sampling typical patients, we’re not being realistic.”

There are some key challenges surrounding diversity in clinical trials, particularly around achieving broader representation from minority ethnic populations. “When you look at the reasons why people don’t want to participate in clinical trials, a long-standing legacy of mistrust in industry is obviously a huge and well-known issue – but it’s not the only one,” said Jo.

“Real-life challenges present major obstacles too. Factors like childcare, sick pay coverage, site location and transportation are all barriers to participation – and, sadly, some of these challenges appear to disproportionately affect minority ethnic communities. Once again, this highlights the importance of early patient engagement to understand those barriers and design studies that help overcome them. Companies need to think proactively about the burden that taking part in a study heaps on the shoulders of those participating – and adapt protocols so they encourage enrolment and representation from diverse groups.”

Location, location, location

So how do you increase diversity? “There’s increased sensitivity to ensuring eligibility criteria really drives diversity into clinical trials,” said Nuala Murphy. “Location plays a key role here. It’s important to select investigators that are in areas that really do provide access to diverse patient populations. We do a lot of work upfront to assess the diversity of the communities in the sites we choose to work with.

“There’s also a piece around trial design. Are you making it easy for people to be in clinical trials? For example, if you’re working in rare diseases, or with severely disabled populations, is it better to bring the trial to the patient, rather than asking the patient to come to the trial? There’s an increased focus on ‘decentralised’ trials – shifting trials more towards the home setting. If you remove the burden on patients having to be on site every second week – spending hours with clinicians in busy clinics, when in fact they could be monitored remotely and visited by a nurse when necessary – it lends itself to greater participation and greater diversity.”

Mark Evans agreed: “It’s not just about widening the criteria; it’s about understanding the inherent bias inbuilt into that criteria, and understanding that the trial design needs to be built around the needs of real patients. Are we making site visits a burden to those with jobs – those without sufficient income to pay their own travel expenses? Fundamentally this rules out those of a lower socioeconomic group, which in turn creates bias against a specific group of people.”

Simulating success

So how do you get it right? “Companies need to redouble their efforts in understanding the real-world challenges facing particular populations when it comes to taking part in clinical trials,” said Jo Fearnhead-Wymbs.

“One way of doing this is through the use of Clinical Trial Simulations (CTS), which allow sponsor companies to test different trial designs with target cohorts before exposing patients to any treatments. The process generates qualitative data on the engagement and experiences of patients and can be used to inform protocol design.”

A great example of this is a CTS from AstraZeneca which looked at patient engagement factors for diverse populations in lupus. “The study simulated clinic visits with patients in two distinct groups – African American females in Atlanta, GA, and white females in Altoona, PA,” said Jo.

“It showed that Atlanta study patients had very different requirements – including a need for strong community support, emphasis on transportation and childcare, and placed greater importance on time commitment and understanding the personal benefits of participation. These insights were used to improve patient recruitment, retention, compliance and advocacy.

“Use of CTS is growing across the industry – although often in very specific target patient groups. Their application in diverse populations, particularly among minority ethnicity groups, can only help improve representation in clinical trials.”

The remote possibility

Another measure that has the potential to increase trial enrolment is the use of remote monitoring. “If you can set up your trials so that you can access electronic medical records and review data remotely, you’re often able to speed up the trial quite significantly,” said Nuala Murphy.

“This takes away the need to have resources on site, and the need for patients to travel. We’re now seeing trials being set up with a greater component of remote monitoring, even up to maybe 90%. COVID-19 has proven that it can be done on a large scale.

“We’re also seeing more ‘adaptive and seamless trials’, ie, trials that include pre-specified criteria for adding or removing vaccine candidates and protocol specifications for advancing from one phase to the next while broadening the patient population.” Adaptive techniques can make it easier for people to participate, encouraging enrolment and enhancing diversity.

A culture strategy

There’s a wide range of measures that can be deployed to drive inclusion in clinical studies but, fundamentally, long-term success will have a strategic anchor.

“Diversity isn’t about tactics, it’s about strategy,” said Amber Chenevert, Group Director, Strategy & Insights, and culture studio lead at VMLY&R. “If we want life-changing innovation to reach the people who need it most, we have to ensure all potential patient populations are well represented in the research. That’s a business strategy challenge and it needs to be considered from the get-go – when companies are deciding where they want to play, the problems they want to solve, and the miracles they want to create. Making sure your purpose is in tune with the needs of people and populations is critical.

“From there, a deep understanding of cultural nuances in the communities we want to engage is essential. It’s a big challenge; culture is more than just a word, it’s a legacy. Our cultural foundations are dug deep – and they exist in every community, no matter their race, background or location in the world. Culture’s a broad church too. It’s made up of everything from language, customs, rituals and faith to art, food, music, jobs, ideology. Those foundational pillars of culture are embedded in our behaviours. Understanding them – really understanding them – is key to driving diversity in clinical trials.” So how do we do it?

“We need to get down to specifics and work out which aspects of culture are getting in the way of trial participation for specific communities or populations,” said Amber. “Whatever your race, religion, ideology or otherwise, there’s often a disconnect between the desired behaviour a company wants – in this case, trial participation – and a belief rooted in our cultural values. It’s only once companies have identified and dissected that disconnect that they can begin to design trials – and health communications – that attract and engage diverse populations.”

The diverse approach

Fittingly, the challenge of securing balanced representation in clinical trials requires a diverse but integrated approach. Developing the right strategy is as much about communications – through patient engagement, community outreach, advocacy and education – as it is about study design, site selection and research methodology.

To succeed, companies need to bring everything together and align their entire operations around the real-life needs of the diverse populations they service. Because if we’re going to reduce health inequalities, an all-inclusive strategy is the only way to go.

Chris Ross is a freelance writer specialising in the pharmaceutical and healthcare industries

10th June 2021

Chris Ross is a freelance writer specialising in the pharmaceutical and healthcare industries

10th June 2021

From: Research, Marketing



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