Livia Ng, Founder and CEO of Neuroute explains the significance of diversity in clinical research and why it is a non-negotiable for science, for medicine and for patients.
Homogeny is no longer an acceptable status quo, and rightly so. Diversity matters, and tough questions are being asked across every industry about how diversity can be embedded into everyday practice. But what does that mean for healthcare and clinical research?
When diversity is absent from patient cohorts, it is recognised as a serious scientific, medical and ethical issue that not only slows progress, but puts lives at risk too. It is well known that there are significant and nuanced physiological differences across genders, age groups, ethnicities, lifestyles, and socioeconomic and cultural groups that influence prevalence, severity and presentation of disease, as well as the safety and efficacy of treatments. Yet little has changed in centuries of clinical research to truly understand and address the extent of these variations, and what they could mean for patient outcomes.
We’ve known about this healthcare disparity for 2000 years
The examples are infinite. Historically, women have been largely excluded from participating in clinical research, and conclusions from male-only cohorts applied to the treatment of both sexes[1] But we know that female bodies are not simply small male bodies, not only having varied physiology, but also entirely different conditions and health experiences including, but not limited to menstruation, pregnancy, birth and menopause. What’s more, data shows that immune responses are generally higher in females than males, which likely contributes to women experiencing higher incidence and severity of many autoimmune conditions than men.[2]
Even as far back as 2000 years ago, Hippocrates noticed the difference in responses between men and women during an influenza outbreak. Yet, it wasn’t until 1993 that US law mandated inclusion of women and minority groups in clinical research, to ensure these variances could be investigated and understood scientifically, biologically and medically.[3]
Although the number of countries participating in pivotal trials has nearly doubled over the past 25 years, there has not been a substantial increase in the diversity of clinical trial populations. Recruiting and enrolling eligible patients in studies can be slow and expensive, meaning that building diverse cohorts is often bottom of the agenda. But ultimately, omission of diverse study participants is skewing data, slowing progress, and leaving minority groups at risk from under-tested treatments.
Signifying the extent of the problem, eltrombopag, a medicine for thrombocytopenia and severe aplastic anaemia, is the only treatment since 2016 to have received first time approval that includes a different dosage recommendation for a specific ethnic group. We already know that prevalence and outcomes for ethnic minority groups in certain conditions are significantly worse. For example, even as recently as the COVID pandemic, data demonstrated that mortality was 3x higher in black men than in white men.[4] A recent US study showed that while 12·4% of pancreatic cancer diagnoses are among Black people, they only accounted for 8·2% of participants in pancreatic cancer clinical trials.[5] We also know that not only are black women more likely to die of breast cancer than white women despite similar incidence rates, they’re also 4x more likely to die in childbirth.[6],[7] It just isn’t good enough.
So diversity in clinical research is not simply a ‘nice-to-have’, it’s critical to achieving better health for individuals and at population level.
Finally, 2000 years after Hippocrates’ observations, stakeholders from across healthcare are beginning to take notice – just this year GSK published an ambition to ensure over 75% of their interventional clinical trials to have a clear demographic plan aligned with disease epidemiology.[8] But do plans go far enough? What action can and should be taking place to practically address these issues?
Data is the great equaliser
With this growing awareness and acknowledgment, alongside greater access to higher quality data sets and real-world data, the quantity and quality of accessible patient data can now increase. Using vast datasets of real-world data, we now have the chance to use specially-built algorithms to identify, select and prioritise perfectly diverse cohorts for clinical research. This means identifying the right demographic mix for the study cohort, or indeed identifying the right specific demographic for targeted clinical research. For us at Neuroute, it’s very much the foundation for everything we do; In a nationwide anxiety and depression study, we facilitated a 670% increase in BME patient participation, proving that proportionate representation is possible and doesn’t have to be difficult nor expensive.[9] Not only will study cohorts become inherently more valuable with better, more accurate and representative results, but we’ll also be able to more easily dedicate research resources to clinical research personalised for minority groups, to fully understand their physiological needs and responses.
Inclusion and accessibility should be factored into clinical trial design. Movements towards adaptive clinical trials and the adoption of new technology in clinical operations provides the opportunity to build protocols that enable diverse representation in study cohorts.
From ‘not the norm’ to ‘non-negotiable’
Building on this awareness and early action, now is the time to drive real, meaningful progress. As an industry we must collectively build a set of non-negotiable, gold standards, that put diversity front and centre in clinical research for the benefit of science, healthcare and the lives of patients. This will require collaboration from experts and advocates from across the sector – from regulators and policy makers, to patient groups, innovators, academic institutes, clinical research specialists and commercial enterprises – to design a framework that can confidently deliver on making the vision for a diverse future of clinical research a reality.
In doing this, everyone wins; with greater diversity in clinical research we can uncover and bring novel treatments to patients faster, transforming health and healthcare globally.
References
[1] Well + Good https://www.wellandgood.com/women-clinical-trials/
[2] Nature https://www.nature.com/articles/nri.2016.90#:~:text=Generally%2C%20adult%20females%20mount%20stronger,to%20inflammatory%20and%20autoimmune%20diseases.
[3] National Institute of Minority Health and Health Disparities, US https://www.nimhd.nih.gov/resources/understanding-health-disparities/diversity-and-inclusion-in-clinical-trials.html
[4] The Office for National Statistics https://www.ons.gov.uk/peoplepopulationandcommunity/birthsdeathsandmarriages/deaths/articles/coronaviruscovid19relateddeathsbyethnicgroupenglandandwales/2march2020to15may2020
[5] The Lancet https://www.thelancet.com/journals/langas/article/PIIS2468-1253(21)00228-4/fulltext
[6] The Cancer Institute
https://www.cancer.gov/about-cancer/understanding/disparities
[7] MBRRACE – UK https://npeu.ox.ac.uk/assets/downloads/mbrrace-uk/reports/maternal-report-2020/MBRRACE-UK_Maternal_Report_Dec_2020_v10.pdf
[8] GSK https://www.gsk.com/en-gb/innovation/trials/diversity-in-clinical-trials/#:~:text=The%20safety%20and%20efficacy%20of,broadly%20across%20all%20patient%20demographics.
[9] Neuroute https://docsend.com/view/me74rh6sk9z4u3gh
