By Andrea Wilkinson, head of patient engagement in neurological and rare syndromes, UCB
The landscape of neurological and rare disorders is as diverse as the human population it affects. These conditions do not discriminate, impacting individuals of every race, ethnicity, and socioeconomic status. Yet, the clinical trials that seek to find treatments for these conditions have not always mirrored this diversity.
The Imperative Need For Patient Diversity
Neurological disorders, such as epilepsy, often present and progress differently among various demographics. This can be influenced by genetics, lifestyle, and environmental factors. Because of this, diversity in clinical research isn’t just a “nice to have” but a scientific necessity.
Without diverse patient populations, we risk missing critical nuances, potentially leading to treatments that are less effective or have unknown effectiveness in certain populations. At UCB, we focus on areas of unmet need and, in particular, strive to achieve inclusive representation of patients living with refractory epilepsy and rare syndromes.
To do that, we secure insights from patients and their caregivers, whether a spouse or another relationship, on varying ethnic and socioeconomic backgrounds to provide a better understanding of the logistical challenges of mobility and safety in daily life.1 We also can see that these patients face many challenges accessing healthcare due to transportation issues, financial constraints, and lack of equipment and support.
Achieving Diversity By Improving Accessibility
For people who live with a range of disabilities, we need to ensure accessibility by leveraging healthcare technology and AI to boost virtual patient engagement efforts. AI can help target the hard-to-reach patient populations at a faster pace. It can analyze data to identify at-risk patients, and by identifying these patients early, we can try to prevent neurological conditions from developing and improve patient outcomes. It also allows us to increase our understanding of these disorders and how we can integrate patient voices and these findings into our research.
The use of wearable technology to monitor symptoms and collect data in real time and remotely has bolstered efforts for increased accessibility. Wearables drive our understanding of how to move forward with clinical trials without disrupting patients’ daily routines or their quality of life. For instance, in-home electroencephalograms (EEGs) are increasingly viable for routine use. They are being tested in patients to determine whether the data quality is scientifically rigorous enough to be included as clinical trial outcome measures. However, additional research is needed to evaluate how digital health technologies for patients with epilepsy could improve their health outcomes, support self-management, and increase access to cost-effective care.
In addition, we aim to increase patient participation in clinical trials by integrating lab data collections into routine care. So, instead of patients attending frequent visits with their healthcare professional and participating in ongoing face-to-face routine lab tests, they can now undergo these assessments at home, which saves time and resources for patients and families, especially those in rural or low-income communities. This patient-informed trial design allows for remote participation to alleviate these challenges while also securing efficient data collection.
However, accessibility pertains not just to physical access but access to information about our medicines, which must be easy to find and written in plain language. After decades of learning across all our clinical trials, we now understand that we need to constantly adapt our educational offerings to make sure they are suitable for all. In 2014, UCB began publicly posting lay summaries of key clinical study results of our approved and/or withdrawn medicines to our external portals. Each published clinical trial summary has received feedback and input from a patient, caregiver, medical professional, lay expert, and member of the public to make sure it is understandable to all people, regardless of education level.
Understanding Unique Needs Of Neurology Patients
Neurological patients often require a level of support that goes beyond the standard protocols, such as those who are pregnant. Pregnancy is often overlooked in those living with a neurological condition and who still need to take their medication throughout their pregnancy. Research suggests that up to 70% of pregnant women take at least one prescription drug.2 However, it is estimated that only 5% of these drugs have been researched in pregnant women and include information in the label to guide use during pregnancy and breastfeeding.3
Pregnant women are often excluded from clinical trials due to safety risks, particularly to unborn babies, and as a result of the pregnant women’s exclusion, they often lack crucial information relating to the risks of drugs they may be taking. To address this issue, UCB is broadening its research objectives to include benefits and risks to mothers from treatment or non-treatment. To achieve this, UCB is pursuing the generation of better-standardized data through global partnerships with pregnancy data registries. We are also working with a new global commission to improve understanding about pregnancy and breastfeeding medication decisions, ensuring women can make fact-based decisions.
Our approach is to learn from the past and address current gaps in research, not only for women but also for underserved communities. We are looking at traditionally underserved patients and those living with a neurological condition not addressed by the medicines currently on the market. For example, UCB published findings from a U.S. survey of neurologists on health disparities in Black patients living with epilepsy, concluding that increasing neurologist awareness of situations that have negative health outcomes in these patients may help to address racial health disparities in this patient population.
Securing Diverse Patient Representation
Securing a representative pool of patients needs to be done at the earliest stages of translational research and begins with the recognition that diversity and accessibility are not just end goals but are integral to the entire clinical trial process.
There’s a natural selection of advocates who have the bandwidth and interest to engage with clinical trials, and they tend to be at a higher income level and are more educated, both of which can bias research. This means we must intentionally engage with diverse patient populations at the very beginning of a clinical trial’s conception, incorporating their insights into study design and continuing these insights gathering throughout the trial.
The path to equitable neurological patient engagement is complex and requires a multifaceted approach. This includes diversifying clinical trial locations and decentralizing the clinical trial data collection so that we not only invite diverse populations to participate in clinical trial design and recruitment efforts but that we also remove the barriers that prevent their participation.
- Taken from PRC approved dated April 2022, LGS Caregiver Advisory Board Meeting Presentation at 2022 LGS Foundation Family & Professional Conference, June 17-19th, 2022.
- Hudson RE, Metz TD, Ward RM, McKnite AM, Enioutina EY, Sherwin CM, Watt KM, Job KM. Drug exposure during pregnancy: Current understanding and approaches to measure maternal-fetal drug exposure. 2023
- Pharmacotherapy during Pregnancy, Childbirth, and Lactation - PMC (nih.gov)
About The Author:
Andrea Wilkinson has had a successful career of problem solving by tailoring strategic product launch solutions for 30+ complex, innovative, and breakthrough biopharmaceutical therapeutic interventions. Over her career, she has addressed challenging global government, regulatory, public policy, and patient barriers. Today, she serves as UCB’s head of global patient engagement in the epilepsy & rare syndromes portfolio strategy team, responsible for integrating patient and caregiver voices throughout the company’s pipeline and portfolio life cycle and delivering patient-defined value that results in improved care and outcomes with and for patients, health systems, and UCB.