With a variety of sources available, including electronic health records and devices to collect data directly from patients, real-world data (RWD) is rapidly becoming the latest frontier for life sciences companies in supporting regulatory filings and conducting drug safety surveillance. In a conversation with Bill Row, Bruce Capobianco and Maggie Adamski from ICON plc, we explore the intersection between real-word studies and patient participation in drug development. How can patient involvement be encouraged?
What is the role that real-world studies are now playing in clinical development?
ICON: Randomised clinical trials (RCTs) aren’t about to be replaced by studies based on real-world evidence (RWE), although RWE certainly is a disrupter that’s being used in many ways – from continuous monitoring and studies of drug utilisation to routine safety monitoring and filling evidence gaps.
The expenses associated with enrolling patients in RCTs, compensating providers and following patients over several years can amount to millions of dollars each year. The availability of RWD, driven by the wide adoption of Electronic Health Records (EHRs), presents an alternative approach to observational research and is suitable for prospective, retrospective and hybrid studies.
Already, there are several instances across therapeutic areas where the US Food & Drug Administration (FDA) has been receptive to examining historical, real-world cohorts of patients treated under the standard of care for safety and efficacy, particularly in cases where a control arm is not possible.
And, as the integrity, quality and timelines of the data collected improves, more and more clinical trials will employ electronic media, such as smartphones and tablets, to collect patient-reported outcomes.
With big data solutions advancing to the forefront of the healthcare ecosystem, companies must build – or have access to – a technology platform capable of generating a continuum of insights from EHRs and other RWD sources.
How can companies encourage patient participation in providing real-world data?
ICON: The key to patient participation in clinical studies of any kind is to use patient feedback to inform the earliest stages of development to ensure that the treatment will address patient needs and reflect their preferences. Fortunately, companies are now routinely bringing patients into the conversation to understand how their disease affects them, day-to-day. It must go beyond understanding side effects and modes of action to include their ‘lived experience’ with the disease. To ensure diversity that reflects the patient population, there are many factors to consider, such as cultural perceptions, ethnicity, socio-economic status and demographics. Listening to patients should always be part of protocol design to minimise the patient burden, increase enrolment and bolster retention.
Truly understanding patient needs and priorities means engaging directly with patients, families and patient advocates to develop high-value, trusting relationships. A variety of methodologies – ranging from interviews, surveys, social listening and co-design workshops to study visit simulations – can be used to gain a holistic view of patients’ ecosystem and how it influences their decisions and behaviours.
How can patients inform selection and development of patient reported outcomes (PROs)?
ICON: The key to successful data collection is ensuring that the design, method and frequency of the PRO fits into the daily lives of patients as well as their care partners. The ideal PRO cannot be burdensome nor cumbersome and must consider multiple factors such as the patient’s condition, age and disease literacy. By uncovering the person behind the patient and the many factors that influence the individual healthcare journey, we can understand what is important – and ultimately what endpoints are of value to the patient to encourage adherence and completion.
What’s the role of patient advocacy groups in encouraging patient participation?
ICON: With patients and their families increasingly looking beyond their healthcare professional for disease information, management and a sense of community, advocacy groups have an ever-expanding role in influencing treatment decisions – including participation in clinical trials and registries. Beyond rare disease areas, disease-specific groups are forming at the international and local level, and they’re often led by patients or family members themselves.
These groups are an increasingly trusted and valuable resource for disseminating information about the importance of trials and their availability to eligible patients who otherwise may not have access to this information. Patient advocacy organisations are emerging as major contributors to the drug discovery process and can often determine product evolution, influence protocol design and advocate for the approval of beneficial treatments that meet the unique needs of specific populations. Relationships with these groups can help drug developers access patients and earn the support needed to conduct trials.
Bill Row is Divisional Principal, Real-World Evidence Strategy & Analytics, Bruce Capobianco is Senior Director, AI Strategy and Analytics and Maggie Adamski is Senior Director, Global Patient Insights and Engagement, all at ICON plc
